Trial Outcomes & Findings for Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I (NCT NCT02702115)
NCT ID: NCT02702115
Last Updated: 2023-01-26
Results Overview
Number of Participants with Treatment-Emergent Adverse Events
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
Up to 36 months after the SB-318 infusion
Results posted on
2023-01-26
Participant Flow
The original protocol anticipated 9-12 subjects in 3 cohorts at ascending doses. However, enrollment ended after 3 subjects were enrolled in 2 cohorts. (No subjects were enrolled in cohort 3). After interim data analysis, the sponsor decided to stop enrollment in this study at 3 subjects enrolled, all adults, in 2 cohorts. (No subjects were enrolled in cohort 3) The sponsor continues to monitor the subjects in a 10-year,follow-up study ST-IVPRP-LT01 (NCT04628871).
Participant milestones
| Measure |
Cohort 1: SB-318: Starting Dose 1.00E+13vg/kg
A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose 5.00E+13vg/kg
A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose 1.20E+14vg/kg
A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Overall Study
STARTED
|
1
|
2
|
0
|
|
Overall Study
COMPLETED
|
1
|
2
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
Baseline characteristics by cohort
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
3 Participants
n=31 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=39 Participants
|
1 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
2 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
2 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
1 Participants
n=31 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
0 Participants
n=41 Participants
|
0 Participants
n=35 Participants
|
0 Participants
n=31 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=39 Participants
|
2 participants
n=41 Participants
|
—
|
3 participants
n=31 Participants
|
|
Baseline Iduronidase (IDUA) Activity
|
1.200 nmol/hr/mg
STANDARD_DEVIATION NA • n=39 Participants
|
9.955 nmol/hr/mg
STANDARD_DEVIATION 2.7789 • n=41 Participants
|
—
|
7.037 nmol/hr/mg
STANDARD_DEVIATION 5.4232 • n=31 Participants
|
|
Baseline Urine Glycosaminoglycans (GAG Levels)
Dermatan Sulfate, Urine
|
129.3500 g/mol creatinine
STANDARD_DEVIATION NA • n=39 Participants
|
15.6680 g/mol creatinine
STANDARD_DEVIATION 6.37527 • n=41 Participants
|
—
|
53.5620 g/mol creatinine
STANDARD_DEVIATION 65.78896 • n=31 Participants
|
|
Baseline Urine Glycosaminoglycans (GAG Levels)
Heparan Sulfate Urine
|
109.4200 g/mol creatinine
STANDARD_DEVIATION NA • n=39 Participants
|
12.3345 g/mol creatinine
STANDARD_DEVIATION 3.72575 • n=41 Participants
|
—
|
44.6963 g/mol creatinine
STANDARD_DEVIATION 56.11422 • n=31 Participants
|
|
Baseline Urine Glycosaminoglycans (GAG Levels)
Total GAG
|
11.360 g/mol creatinine
STANDARD_DEVIATION NA • n=39 Participants
|
3.620 g/mol creatinine
STANDARD_DEVIATION 0.8485 • n=41 Participants
|
—
|
6.200 g/mol creatinine
STANDARD_DEVIATION 4.5088 • n=31 Participants
|
PRIMARY outcome
Timeframe: Up to 36 months after the SB-318 infusionPopulation: No subjects were enrolled in Cohort 3
Number of Participants with Treatment-Emergent Adverse Events
Outcome measures
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events
|
1 Participants
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline and Month 36 after the SB-318 infusionPopulation: No subjects were enrolled in Cohort 3
Change from baseline clinical laboratory in measurement of IDUA activity measured in leukocytes.
Outcome measures
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Effect of SB-318 on IDUA Activity
|
-1.200 nmol/hr/mg
Standard Deviation NA
Only one subject was enrolled in Cohort 1
|
-2.515 nmol/hr/mg
Standard Deviation 5.9185
|
—
|
SECONDARY outcome
Timeframe: Baseline and 24 months after the SB-318 infusionPopulation: No subjects were enrolled in Cohort 3
Change from baseline in total GAG, Dermatan Sulfate GAG, and Heparan Sulfate GAG measured in urine at Month 24
Outcome measures
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Effect of SB-318 on Urine Glycosaminoglycans (GAG) Levels
Dermatan Sulfate
|
0.510 g/mol creatinine
Standard Deviation NA
Only one subject was enrolled in Cohort 1
|
-0.090 g/mol creatinine
Standard Deviation 0.3960
|
—
|
|
Effect of SB-318 on Urine Glycosaminoglycans (GAG) Levels
Heparan Sulfate
|
30.2000 g/mol creatinine
Standard Deviation NA
Only one subject was enrolled in Cohort 1
|
3.6005 g/mol creatinine
Standard Deviation 8.22436
|
—
|
|
Effect of SB-318 on Urine Glycosaminoglycans (GAG) Levels
Total GAG
|
0.940 g/mol creatinine
Standard Deviation NA
Only one subject was enrolled in Cohort 1
|
0.240 g/mol creatinine
Standard Deviation 0.0424
|
—
|
SECONDARY outcome
Timeframe: Up to 24 months after the SB-318 infusionPopulation: No subjects were enrolled in Cohort 3
Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24.
Outcome measures
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 Participants
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
AAV2/6 Clearance in Plasma, Saliva, Urine, Stool, and Semen
|
1 Participants
|
2 Participants
|
0 Participants
|
Adverse Events
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 participants at risk
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 participants at risk
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Infections and infestations
Adenovirus infection
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Cardiac disorders
Atrial flutter
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
Other adverse events
| Measure |
Cohort 1: SB-318: Starting Dose_1.00E+13 vg/kg
n=1 participants at risk
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 2: SB-318 at Next Ascending Dose_5.00E+13 vg/kg
n=2 participants at risk
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
Cohort 3: SB-318 at Next Ascending Dose_1.20E+14 vg/kg
SB-318: A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.
|
|---|---|---|---|
|
Cardiac disorders
Aortic valve stenosis
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Cardiac disorders
Atrial fibrillation
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Cardiac disorders
Cardiac failure
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
100.0%
2/2 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Cardiac disorders
Left atrial enlargement
|
100.0%
1/1 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
0.00%
0/2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
General disorders
Oedema peripheral
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Infections and infestations
Bronchitis
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Injury, poisoning and procedural complications
Excoriation
|
100.0%
1/1 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
0.00%
0/2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Investigations
Blood cortisol decreased
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
100.0%
2/2 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
100.0%
1/1 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Nervous system disorders
Dizziness
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Respiratory, thoracic and mediastinal disorders
Tracheal stenosis
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Skin and subcutaneous tissue disorders
Acne
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
100.0%
2/2 • Number of events 3 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Infections and infestations
Upper Respiratory Infection
|
100.0%
1/1 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
100.0%
2/2 • Number of events 4 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Nervous system disorders
Headache
|
100.0%
1/1 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 2 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Renal and urinary disorders
Renal Cyst
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
50.0%
1/2 • Number of events 1 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
—
0/0 • Adverse event data was collected from the subject's date of screening until their end of study visit at 36 months.
No subjects were enrolled in Cohort 3
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place