Trial Outcomes & Findings for A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) (NCT NCT02760264)
NCT ID: NCT02760264
Last Updated: 2019-01-02
Results Overview
Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug. Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
48 participants
Primary outcome timeframe
Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
Results posted on
2019-01-02
Participant Flow
Participant milestones
| Measure |
Vamorolone 0.25 mg/kg/Day
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Vamorolone 0.75 mg/kg/Day
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Vamorolone 2.0 mg/kg/Day
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Vamorolone 6.0 mg/kg/Day
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
0.25 mg/kg/Day
STARTED
|
12
|
0
|
0
|
0
|
|
0.25 mg/kg/Day
COMPLETED
|
12
|
0
|
0
|
0
|
|
0.25 mg/kg/Day
NOT COMPLETED
|
0
|
0
|
0
|
0
|
|
0.75 mg/kg/Day
STARTED
|
0
|
12
|
0
|
0
|
|
0.75 mg/kg/Day
COMPLETED
|
0
|
12
|
0
|
0
|
|
0.75 mg/kg/Day
NOT COMPLETED
|
0
|
0
|
0
|
0
|
|
2.0 mg/kg/Day
STARTED
|
0
|
0
|
12
|
0
|
|
2.0 mg/kg/Day
COMPLETED
|
0
|
0
|
12
|
0
|
|
2.0 mg/kg/Day
NOT COMPLETED
|
0
|
0
|
0
|
0
|
|
6.0 mg/kg/Day
STARTED
|
0
|
0
|
0
|
12
|
|
6.0 mg/kg/Day
COMPLETED
|
0
|
0
|
0
|
12
|
|
6.0 mg/kg/Day
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Baseline characteristics by cohort
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
Total
n=48 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Customized
Age
|
5.2 years
STANDARD_DEVIATION 1.03 • n=99 Participants
|
4.8 years
STANDARD_DEVIATION 0.83 • n=107 Participants
|
4.7 years
STANDARD_DEVIATION 0.89 • n=206 Participants
|
4.8 years
STANDARD_DEVIATION 0.75 • n=157 Participants
|
4.9 years
STANDARD_DEVIATION 0.87 • n=390 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
0 Participants
n=390 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
12 Participants
n=157 Participants
|
48 Participants
n=390 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
3 Participants
n=157 Participants
|
4 Participants
n=390 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
9 Participants
n=157 Participants
|
44 Participants
n=390 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
0 Participants
n=390 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
0 Participants
n=390 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
1 Participants
n=390 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
0 Participants
n=390 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
1 Participants
n=390 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
12 Participants
n=157 Participants
|
45 Participants
n=390 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
0 Participants
n=390 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
1 Participants
n=390 Participants
|
|
Region of Enrollment
Canada
|
3 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
2 Participants
n=157 Participants
|
7 Participants
n=390 Participants
|
|
Region of Enrollment
Sweden
|
0 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
4 Participants
n=390 Participants
|
|
Region of Enrollment
United States
|
9 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
6 Participants
n=157 Participants
|
20 Participants
n=390 Participants
|
|
Region of Enrollment
United Kingdom
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
2 Participants
n=157 Participants
|
6 Participants
n=390 Participants
|
|
Region of Enrollment
Israel
|
0 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
0 Participants
n=157 Participants
|
5 Participants
n=390 Participants
|
|
Region of Enrollment
Australia
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
2 Participants
n=157 Participants
|
6 Participants
n=390 Participants
|
PRIMARY outcome
Timeframe: Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in safety population.
Treatment-emergent adverse events (TEAEs) are defined as any adverse event or worsening of an existing conditions after initiation of the investigational product and through the subject's last study visit (study completion or early termination). Serious adverse events were recorded for up to 30 days after the final administration of study drug. Note: Total Number of Treatment Emergent Adverse Events: The total incidences of TEAEs experienced in study; Any Treatment Emergent Adverse Event: TEAEs reported at least once per dose group
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Total Number of Treatment Emergent Adverse Events
|
13 Number of Events
|
13 Number of Events
|
11 Number of Events
|
9 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Total Number of Adverse Events
|
16 Number of Events
|
18 Number of Events
|
13 Number of Events
|
11 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Any Treatment Emergent Adverse Events
|
7 Number of Events
|
6 Number of Events
|
8 Number of Events
|
7 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Any Drug Related Treatment Emergent Adverse Events
|
1 Number of Events
|
2 Number of Events
|
2 Number of Events
|
3 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Any CTCAE Grade 3 or Higher TEAE
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Discontinuation of Study Drug due to TEAE
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Any Serious Treatment Emergent Adverse Events
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
|
Overall Summary of Adverse Events as Assessed by CTCAE Version 4.03
Death
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
0 Number of Events
|
SECONDARY outcome
Timeframe: Baseline, Week 2Population: Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Week 2
|
85.3 mg/ dL
Standard Deviation 9.29
|
83.1 mg/ dL
Standard Deviation 6.69
|
89.5 mg/ dL
Standard Deviation 5.20
|
89.2 mg/ dL
Standard Deviation 11.12
|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Week 2 Change from Baseline
|
-2.2 mg/ dL
Standard Deviation 10.46
|
-5.8 mg/ dL
Standard Deviation 18.92
|
0.2 mg/ dL
Standard Deviation 8.79
|
-1.3 mg/ dL
Standard Deviation 9.41
|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Fasting Glucose
Baseline
|
87.5 mg/ dL
Standard Deviation 9.44
|
88.9 mg/ dL
Standard Deviation 18.71
|
89.3 mg/ dL
Standard Deviation 7.91
|
92.3 mg/ dL
Standard Deviation 8.19
|
SECONDARY outcome
Timeframe: Baseline, Week 2Population: Safety Population: All subject who receive at least dose of vamorolone study medication will be included in the safety population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=10 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance) -Fasting Glucose
|
-1.8 Week 2 % change from Baseline
Standard Deviation 13.07
|
-4.2 Week 2 % change from Baseline
Standard Deviation 13.98
|
0.8 Week 2 % change from Baseline
Standard Deviation 9.76
|
-1.2 Week 2 % change from Baseline
Standard Deviation 9.80
|
SECONDARY outcome
Timeframe: Baseline , Week 2Population: Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Baseline
|
5.54 µIU/mL
Standard Deviation 3.651
|
3.09 µIU/mL
Standard Deviation 2.033
|
3.40 µIU/mL
Standard Deviation 1.548
|
3.96 µIU/mL
Standard Deviation 2.027
|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Week 2
|
5.29 µIU/mL
Standard Deviation 2.671
|
3.22 µIU/mL
Standard Deviation 1.924
|
3.87 µIU/mL
Standard Deviation 2.118
|
6.73 µIU/mL
Standard Deviation 4.599
|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
Week 2 Change from Baseline
|
-0.65 µIU/mL
Standard Deviation 2.913
|
0.34 µIU/mL
Standard Deviation 1.289
|
0.47 µIU/mL
Standard Deviation 2.777
|
2.78 µIU/mL
Standard Deviation 4.651
|
SECONDARY outcome
Timeframe: Baseline, Week 2Population: Safety Population All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=10 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=8 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Insulin Resistance)- Insulin
|
-5.54 Week 2 % change from Baseline
Standard Deviation 32.622
|
26.07 Week 2 % change from Baseline
Standard Deviation 76.483
|
42.85 Week 2 % change from Baseline
Standard Deviation 107.337
|
83.55 Week 2 % change from Baseline
Standard Deviation 117.064
|
SECONDARY outcome
Timeframe: Week 2 (pre-dose)Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=11 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=11 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=10 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Adrenal Axis Suppression)- First in Morning Cortisol
|
10.425 mcg/dL
Standard Deviation 1.7358
|
9.755 mcg/dL
Standard Deviation 2.7614
|
7.321 mcg/dL
Standard Deviation 3.0322
|
3.010 mcg/dL
Standard Deviation 1.0141
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Week 2, Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Baseline
|
37.94 ng/mL
Standard Deviation 11.622
|
35.66 ng/mL
Standard Deviation 6.800
|
41.17 ng/mL
Standard Deviation 5.617
|
44.36 ng/mL
Standard Deviation 5.979
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Day 1
|
39.37 ng/mL
Standard Deviation 14.189
|
35.89 ng/mL
Standard Deviation 7.526
|
34.93 ng/mL
Standard Deviation 6.881
|
33.52 ng/mL
Standard Deviation 9.135
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Day 1 Change from Baseline
|
1.43 ng/mL
Standard Deviation 8.197
|
0.23 ng/mL
Standard Deviation 5.304
|
-6.23 ng/mL
Standard Deviation 10.930
|
-11.37 ng/mL
Standard Deviation 9.137
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 2
|
38.53 ng/mL
Standard Deviation 12.025
|
35.10 ng/mL
Standard Deviation 8.238
|
37.51 ng/mL
Standard Deviation 8.624
|
29.04 ng/mL
Standard Deviation 4.853
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 2 Change from Baseline
|
0.58 ng/mL
Standard Deviation 5.986
|
-0.56 ng/mL
Standard Deviation 7.934
|
-3.66 ng/mL
Standard Deviation 9.818
|
-15.32 ng/mL
Standard Deviation 6.450
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 4
|
39.20 ng/mL
Standard Deviation 14.136
|
42.24 ng/mL
Standard Deviation 8.426
|
47.91 ng/mL
Standard Deviation 6.648
|
42.81 ng/mL
Standard Deviation 10.851
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 4 Change from Baseline
|
1.26 ng/mL
Standard Deviation 5.650
|
6.58 ng/mL
Standard Deviation 9.341
|
6.74 ng/mL
Standard Deviation 9.747
|
-1.55 ng/mL
Standard Deviation 11.176
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Week 2, Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Day 1 Percent Change from Baseline
|
3.95 % change from Baseline
Standard Deviation 22.015
|
1.41 % change from Baseline
Standard Deviation 15.449
|
-12.63 % change from Baseline
Standard Deviation 25.020
|
-25.05 % change from Baseline
Standard Deviation 20.228
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 2 Percent Change from Baseline
|
2.48 % change from Baseline
Standard Deviation 16.756
|
-0.07 % change from Baseline
Standard Deviation 22.408
|
-7.81 % change from Baseline
Standard Deviation 23.664
|
-33.87 % change from Baseline
Standard Deviation 12.548
|
|
Serum Pharmacodynamics Biomarkers (Bone Turnover) -Osteocalcin
Week 4 Percent Change from Baseline
|
2.72 % change from Baseline
Standard Deviation 14.063
|
20.91 % change from Baseline
Standard Deviation 28.644
|
18.74 % change from Baseline
Standard Deviation 25.095
|
-2.43 % change from Baseline
Standard Deviation 25.932
|
SECONDARY outcome
Timeframe: Baseline Day 1 Week 2 Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Baseline
|
555.8 ng/mL
Standard Deviation 184.72
|
480.7 ng/mL
Standard Deviation 118.20
|
508.2 ng/mL
Standard Deviation 94.36
|
511.5 ng/mL
Standard Deviation 106.50
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Day 1
|
474.0 ng/mL
Standard Deviation 116.45
|
443.7 ng/mL
Standard Deviation 112.38
|
417.1 ng/mL
Standard Deviation 87.35
|
475.2 ng/mL
Standard Deviation 147.10
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Day 1 Change from Baseline
|
-81.8 ng/mL
Standard Deviation 124.24
|
-34.5 ng/mL
Standard Deviation 101.97
|
-91.1 ng/mL
Standard Deviation 64.64
|
-36.5 ng/mL
Standard Deviation 144.04
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 2
|
443.8 ng/mL
Standard Deviation 93.86
|
407.8 ng/mL
Standard Deviation 96.54
|
346.6 ng/mL
Standard Deviation 68.59
|
303.7 ng/mL
Standard Deviation 56.38
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 2 Change from Baseline
|
-112.0 ng/mL
Standard Deviation 125.08
|
-70.6 ng/mL
Standard Deviation 123.69
|
-161.6 ng/mL
Standard Deviation 73.52
|
-207.8 ng/mL
Standard Deviation 78.16
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 4
|
573.8 ng/mL
Standard Deviation 251.02
|
496.7 ng/mL
Standard Deviation 117.57
|
492.0 ng/mL
Standard Deviation 81.92
|
566.3 ng/mL
Standard Deviation 149.32
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 4 Change from Baseline
|
18.1 ng/mL
Standard Deviation 153.10
|
21.3 ng/mL
Standard Deviation 122.87
|
-16.2 ng/mL
Standard Deviation 79.64
|
54.8 ng/mL
Standard Deviation 118.09
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Week 2, Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Day 1 Percent Change from Baseline
|
-12.2 % change from Baseline
Standard Deviation 17.07
|
-5.4 % change from Baseline
Standard Deviation 24.58
|
-17.4 % change from Baseline
Standard Deviation 12.59
|
-5.7 % change from Baseline
Standard Deviation 30.76
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 2 Percent Change from Baseline
|
-17.5 % change from Baseline
Standard Deviation 14.65
|
-11.2 % change from Baseline
Standard Deviation 29.19
|
-30.9 % change from Baseline
Standard Deviation 11.80
|
-39.9 % change from Baseline
Standard Deviation 9.22
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)- Procollagen 1 N-Terminal Propeptide
Week 4 Percent Change from Baseline
|
2.8 % change from Baseline
Standard Deviation 25.57
|
7.8 % change from Baseline
Standard Deviation 30.86
|
-1.4 % change from Baseline
Standard Deviation 17.47
|
11.8 % change from Baseline
Standard Deviation 28.24
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Baseline
|
871.0 pg/mL
Standard Deviation 160.85
|
935.8 pg/mL
Standard Deviation 286.50
|
936.8 pg/mL
Standard Deviation 256.25
|
889.3 pg/mL
Standard Deviation 186.68
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Day 1
|
974.8 pg/mL
Standard Deviation 252.77
|
940.8 pg/mL
Standard Deviation 227.60
|
838.3 pg/mL
Standard Deviation 233.30
|
786.8 pg/mL
Standard Deviation 331.68
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Day 1 Change from Baseline
|
72.4 pg/mL
Standard Deviation 241.87
|
-12.9 pg/mL
Standard Deviation 233.44
|
-98.5 pg/mL
Standard Deviation 237.69
|
-115.7 pg/mL
Standard Deviation 421.04
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 2
|
963.7 pg/mL
Standard Deviation 157.68
|
903.3 pg/mL
Standard Deviation 251.01
|
710.4 pg/mL
Standard Deviation 180.03
|
625.7 pg/mL
Standard Deviation 203.19
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 2 Change from Baseline
|
85.0 pg/mL
Standard Deviation 185.90
|
-19.9 pg/mL
Standard Deviation 266.99
|
-226.4 pg/mL
Standard Deviation 185.99
|
-263.7 pg/mL
Standard Deviation 229.69
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 4
|
915.9 pg/mL
Standard Deviation 263.13
|
983.5 pg/mL
Standard Deviation 298.47
|
949.8 pg/mL
Standard Deviation 303.76
|
989.2 pg/mL
Standard Deviation 216.29
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 4 Change from Baseline
|
17.4 pg/mL
Standard Deviation 267.45
|
34.8 pg/mL
Standard Deviation 271.50
|
12.9 pg/mL
Standard Deviation 181.45
|
99.8 pg/mL
Standard Deviation 211.90
|
SECONDARY outcome
Timeframe: Baseline, Day 1, Week 4Population: Safety Population: All subjects who receive at least one dose of vamorolone study medication will be included in the Safety Population.
Pharmacodynamic biomarkers were measured to investigate the effects of single and multiple oral doses of vamorolone on serum PD biomarkers in ambulant boys ages 4-\< 7 years with DMD.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 2 Percent Change from Baseline
|
11.9 % change from Baseline
Standard Deviation 20.52
|
2.2 % change from Baseline
Standard Deviation 26.44
|
-22.5 % change from Baseline
Standard Deviation 15.27
|
-27.7 % change from Baseline
Standard Deviation 26.50
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Day 1 Percent Change from Baseline
|
9.9 % change from Baseline
Standard Deviation 25.38
|
2.7 % change from Baseline
Standard Deviation 23.76
|
-8.0 % change from Baseline
Standard Deviation 23.93
|
-6.7 % change from Baseline
Standard Deviation 53.72
|
|
Serum Pharmacodynamic Biomarkers (Bone Turnover)-Type I Collagen C-Telopeptides
Week 4 Percent Change from Baseline
|
3.6 % change from Baseline
Standard Deviation 28.96
|
6.8 % change from Baseline
Standard Deviation 24.06
|
2.7 % change from Baseline
Standard Deviation 19.03
|
14.5 % change from Baseline
Standard Deviation 32.98
|
SECONDARY outcome
Timeframe: Day 1, Week 2Population: All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. \[Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. tmax= time when plasma concentration is at maximum.
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) Assessments (Tmax)
Day 1
|
3.6 hour
Standard Deviation 1.2
|
4.6 hour
Standard Deviation 2.1
|
2.5 hour
Standard Deviation 1.3
|
2.7 hour
Standard Deviation 1.3
|
|
Pharmacokinetic (PK) Assessments (Tmax)
Week 2
|
3.8 hour
Standard Deviation 1.80
|
3.8 hour
Standard Deviation 2.2
|
2.8 hour
Standard Deviation 1
|
2.3 hour
Standard Deviation 0.86
|
SECONDARY outcome
Timeframe: Day 1, Week 2Population: All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. \[Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. AUC inf= Area under the concentration vs. time curve to time infinity.
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) Assessments (AUC Inf)
Day 1
|
118 [(hr)(ng)/mL]
Standard Deviation 48
|
379 [(hr)(ng)/mL]
Standard Deviation 117
|
761 [(hr)(ng)/mL]
Standard Deviation 352
|
3279 [(hr)(ng)/mL]
Standard Deviation 1693
|
|
Pharmacokinetic (PK) Assessments (AUC Inf)
Week 2
|
164 [(hr)(ng)/mL]
Standard Deviation 61
|
544 [(hr)(ng)/mL]
Standard Deviation 155
|
1138 [(hr)(ng)/mL]
Standard Deviation 467
|
3606 [(hr)(ng)/mL]
Standard Deviation 897
|
SECONDARY outcome
Timeframe: Day 1, Week 2Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) Assessments CL (ml/hr/kg)
Day 1
|
2459 ml/hr/kg
Standard Deviation 897
|
2285 ml/hr/kg
Standard Deviation 1103
|
2697 ml/hr/kg
Standard Deviation 1285
|
2320 ml/hr/kg
Standard Deviation 1375
|
|
Pharmacokinetic (PK) Assessments CL (ml/hr/kg)
Week 2
|
1828 ml/hr/kg
Standard Deviation 919
|
1509 ml/hr/kg
Standard Deviation 482
|
2047 ml/hr/kg
Standard Deviation 771
|
1777 ml/hr/kg
Standard Deviation 476
|
SECONDARY outcome
Timeframe: Day 1, Week 2Population: All subjects who receive at least one dose of vamorolone study medication and have sufficient quantifiable plasma concentration data for PK analysis will be included in the PK population. \[Note that the PK population will be determined by the study pharmacokineticist upon review of the concentration information for each subject in each cohort.
Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay. t1/2= elimination half life.
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) Assessments t(1/2)
Day 1
|
2.1 hour
Standard Deviation 0.85
|
1.8 hour
Standard Deviation 0.43
|
1.9 hour
Standard Deviation 0.79
|
1.9 hour
Standard Deviation 0.95
|
|
Pharmacokinetic (PK) Assessments t(1/2)
Week 2
|
1.9 hour
Standard Deviation 0.96
|
2.1 hour
Standard Deviation 0.8
|
1.9 hour
Standard Deviation 1.02
|
1.4 hour
Standard Deviation 0.35
|
SECONDARY outcome
Timeframe: Day 1, Week 2Plasma concentrations of vamorolone were measured using a specific and validated liquid chromatography tandem mass spectrometry (LC-MS) assay
Outcome measures
| Measure |
Dose Level Group 1
n=11 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 Participants
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 Participants
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 Participants
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Pharmacokinetic (PK) Assessments (Cmax)
Day 1
|
22.9 ng/mL
Standard Deviation 13.4
|
75.9 ng/mL
Standard Deviation 25.9
|
199 ng/mL
Standard Deviation 111
|
855.6 ng/mL
Standard Deviation 471
|
|
Pharmacokinetic (PK) Assessments (Cmax)
Week 2
|
32.2 ng/mL
Standard Deviation 15.2
|
124.7 ng/mL
Standard Deviation 42.5
|
252.2 ng/mL
Standard Deviation 96
|
970 ng/mL
Standard Deviation 270
|
SECONDARY outcome
Timeframe: Week 2 (Day 14)A portion of each blood sample of the Week 2 (Day 14) pharmacokinetic assessment time points for the subjects receiving vamorolone 2 mg/kg/day was used for analysis of vamorolone metabolites.
Outcome measures
| Measure |
Dose Level Group 1
n=12 Participants
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Metabolites in Safety Testing (MIST) Assessment
M1
|
34.42 % of total drug related exposure
Standard Deviation 2.79
|
—
|
—
|
—
|
|
Metabolites in Safety Testing (MIST) Assessment
M2
|
1.16 % of total drug related exposure
Standard Deviation 0.06
|
—
|
—
|
—
|
|
Metabolites in Safety Testing (MIST) Assessment
M3
|
1.21 % of total drug related exposure
Standard Deviation 0.07
|
—
|
—
|
—
|
|
Metabolites in Safety Testing (MIST) Assessment
M4
|
37.84 % of total drug related exposure
Standard Deviation 3.78
|
—
|
—
|
—
|
|
Metabolites in Safety Testing (MIST) Assessment
M5
|
2.73 % of total drug related exposure
Standard Deviation 0.17
|
—
|
—
|
—
|
|
Metabolites in Safety Testing (MIST) Assessment
Vamorolone
|
22.64 % of total drug related exposure
Standard Deviation 0.69
|
—
|
—
|
—
|
Adverse Events
Dose Level Group 1
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Dose Level Group 2
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Dose Level Group 3
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Dose Level Group 4
Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Dose Level Group 1
n=12 participants at risk
Participants enrolled in Dose Level Group 1 will receive vamorolone 0.25 mg/kg/day.
Vamorolone 0.25 mg/kg/day: Oral administration of 0.25 mg/kg/day daily for 14 days.
|
Dose Level Group 2
n=12 participants at risk
Participants enrolled in Dose Level Group 2 will receive vamorolone 0.75 mg/kg/day.
Vamorolone 0.75 mg/kg/day: Oral administration of 0.75 mg/kg/day daily for 14 days.
|
Dose Level Group 3
n=12 participants at risk
Participants enrolled in Dose Level Group 3 will receive vamorolone 2.0 mg/kg/day.
Vamorolone 2.0 mg/kg/day: Oral administration of 2.0 mg/kg/day daily for 14 days.
|
Dose Level Group 4
n=12 participants at risk
Participants enrolled in Dose Level Group 4 will receive vamorolone 6.0 mg/kg/day.
Vamorolone 6.0 mg/kg/day: Oral administration of 6 mg/kg/day daily for 14 days.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
16.7%
2/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Faeces Discoloured
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Lip Swelling
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Gastrointestinal disorders
Vomiting
|
16.7%
2/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
General disorders
Pyrexia
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Infections and infestations
Enterobiasis
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Infections and infestations
Lice Infestation
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Infections and infestations
Viral Upper Respiratory Tract Infection
|
25.0%
3/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
16.7%
2/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Musculoskeletal and connective tissue disorders
Muscle Spasms
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
25.0%
3/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Nervous system disorders
Headache
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Nervous system disorders
Lethargy
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Psychiatric disorders
Agitation
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Psychiatric disorders
Emotional Disorder
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Psychiatric disorders
Insomnia
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Psychiatric disorders
Irritability
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Psychiatric disorders
Restlessness
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Renal and urinary disorders
Chromaturia
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
16.7%
2/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Skin and subcutaneous tissue disorders
Rash Macular
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
|
Vascular disorders
Flushing
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
0.00%
0/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
8.3%
1/12 • Adverse events will be recorded from the date of informed consent and through the time of the subject's last study visit. Serious adverse events will be recorded from the date of informed consent and for up to 30 days after final drug administration.
The incidence of TEAEs will be summarized by dose level, SOC and preferred term; dose level, SOC, and intensity (CTCAE grade; CTCAE version 4.03); dose level, SOC, and outcome; and dose level, SOC and relationship to study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place