Trial Outcomes & Findings for Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease (NCT NCT03033108)
NCT ID: NCT03033108
Last Updated: 2021-05-19
Results Overview
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
23 participants
Primary outcome timeframe
Baseline and 1 month
Results posted on
2021-05-19
Participant Flow
Subjects were recruited at 6 sites in the United States from January to September 2017
Participant milestones
| Measure |
Emixustat Dose 1
lowest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 2
middle dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 3
highest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
|---|---|---|---|
|
Overall Study
STARTED
|
7
|
9
|
7
|
|
Overall Study
COMPLETED
|
7
|
8
|
7
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
Baseline characteristics by cohort
| Measure |
Emixustat Dose 1
n=7 Participants
lowest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 2
n=9 Participants
middle dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 3
n=7 Participants
highest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
53.6 years
n=99 Participants
|
54.3 years
n=107 Participants
|
46.3 years
n=206 Participants
|
51.6 years
n=7 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
8 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
15 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
22 Participants
n=7 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=7 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=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 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=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
4 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
18 Participants
n=7 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=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Region of Enrollment
United States
|
7 participants
n=99 Participants
|
9 participants
n=107 Participants
|
7 participants
n=206 Participants
|
23 participants
n=7 Participants
|
|
Number of ABCA4 gene mutations
One mutation
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
5 Participants
n=7 Participants
|
|
Number of ABCA4 gene mutations
Two mutations
|
6 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
18 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Baseline and 1 monthPopulation: Subjects with evaluable ERGs at both Baseline and Month 1
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
Outcome measures
| Measure |
Emixustat Dose 1
n=6 Participants
lowest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 2
n=7 Participants
middle dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 3
n=6 Participants
highest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
|---|---|---|---|
|
Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
|
-12.23 percent suppression
Interval -41.0 to 54.7
|
68.00 percent suppression
Interval 1.0 to 91.5
|
96.69 percent suppression
Interval 64.9 to 100.0
|
SECONDARY outcome
Timeframe: 1 monthAssessment of safety profile
Outcome measures
| Measure |
Emixustat Dose 1
n=7 Participants
lowest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 2
n=9 Participants
middle dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 3
n=7 Participants
highest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
|---|---|---|---|
|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Subjects with 1 or more adverse events
|
6 Participants
|
8 Participants
|
6 Participants
|
|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Subjects with 1 or more mild adverse events
|
5 Participants
|
5 Participants
|
4 Participants
|
|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Subjects with 1 or more moderate adverse events
|
1 Participants
|
2 Participants
|
2 Participants
|
|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Subjects with 1 or more severe adverse events
|
0 Participants
|
1 Participants
|
1 Participants
|
|
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Subjects with 1 or more serious adverse events
|
0 Participants
|
0 Participants
|
0 Participants
|
Adverse Events
Emixustat Dose 1
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Emixustat Dose 2
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Emixustat Dose 3
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 |
Emixustat Dose 1
n=7 participants at risk
lowest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 2
n=9 participants at risk
middle dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
Emixustat Dose 3
n=7 participants at risk
highest dose of once-daily oral emixustat
Emixustat: Once daily, tablet for oral administration
|
|---|---|---|---|
|
Eye disorders
Delayed Dark Adaptation
|
14.3%
1/7 • 1 month
|
66.7%
6/9 • 1 month
|
57.1%
4/7 • 1 month
|
|
Eye disorders
Erythropsia
|
14.3%
1/7 • 1 month
|
33.3%
3/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Eye disorders
Vision blurred
|
28.6%
2/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Eye disorders
Photophobia
|
14.3%
1/7 • 1 month
|
22.2%
2/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Eye disorders
Visual impairment
|
14.3%
1/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Eye disorders
Blindness day
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Eye disorders
Chromatopsia
|
0.00%
0/7 • 1 month
|
0.00%
0/9 • 1 month
|
28.6%
2/7 • 1 month
|
|
Eye disorders
Night blindness
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Eye disorders
Xanthopsia
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Nervous system disorders
Headache
|
0.00%
0/7 • 1 month
|
22.2%
2/9 • 1 month
|
28.6%
2/7 • 1 month
|
|
Eye disorders
Asthenopia
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Eye disorders
Eye Pain
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Eye disorders
Eye Pruritis
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Eye disorders
Lenticular opacities
|
0.00%
0/7 • 1 month
|
0.00%
0/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrheoea
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Infections and infestations
Upper respiratory tract infections
|
0.00%
0/7 • 1 month
|
11.1%
1/9 • 1 month
|
0.00%
0/7 • 1 month
|
|
Eye disorders
Visual acuity reduced
|
0.00%
0/7 • 1 month
|
0.00%
0/9 • 1 month
|
14.3%
1/7 • 1 month
|
|
Nervous system disorders
Visual field defect
|
14.3%
1/7 • 1 month
|
0.00%
0/9 • 1 month
|
0.00%
0/7 • 1 month
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place