Trial Outcomes & Findings for Safety and Efficacy of Emixustat in Stargardt Disease (NCT NCT03772665)
NCT ID: NCT03772665
Last Updated: 2024-05-30
Results Overview
Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
194 participants
Primary outcome timeframe
24 months
Results posted on
2024-05-30
Participant Flow
Participant milestones
| Measure |
Emixustat
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
|---|---|---|
|
Overall Study
STARTED
|
128
|
66
|
|
Overall Study
COMPLETED
|
127
|
66
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Emixustat
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
|---|---|---|
|
Overall Study
Protocol Violation
|
1
|
0
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Emixustat
n=128 Participants
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
n=66 Participants
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
Total
n=194 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=128 Participants
|
2 Participants
n=66 Participants
|
7 Participants
n=194 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
122 Participants
n=128 Participants
|
64 Participants
n=66 Participants
|
186 Participants
n=194 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=128 Participants
|
0 Participants
n=66 Participants
|
1 Participants
n=194 Participants
|
|
Age, Continuous
|
43.9 years
STANDARD_DEVIATION 15.03 • n=128 Participants
|
43.0 years
STANDARD_DEVIATION 14.07 • n=66 Participants
|
43.6 years
STANDARD_DEVIATION 14.75 • n=194 Participants
|
|
Sex: Female, Male
Female
|
59 Participants
n=128 Participants
|
29 Participants
n=66 Participants
|
88 Participants
n=194 Participants
|
|
Sex: Female, Male
Male
|
69 Participants
n=128 Participants
|
37 Participants
n=66 Participants
|
106 Participants
n=194 Participants
|
|
Race and Ethnicity Not Collected
|
—
|
—
|
0 Participants
Race and Ethnicity were not collected from any participant.
|
|
Region of Enrollment
Canada
|
3 participants
n=128 Participants
|
2 participants
n=66 Participants
|
5 participants
n=194 Participants
|
|
Region of Enrollment
Netherlands
|
3 participants
n=128 Participants
|
2 participants
n=66 Participants
|
5 participants
n=194 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=128 Participants
|
19 participants
n=66 Participants
|
53 participants
n=194 Participants
|
|
Region of Enrollment
Brazil
|
20 participants
n=128 Participants
|
9 participants
n=66 Participants
|
29 participants
n=194 Participants
|
|
Region of Enrollment
Denmark
|
6 participants
n=128 Participants
|
4 participants
n=66 Participants
|
10 participants
n=194 Participants
|
|
Region of Enrollment
Italy
|
24 participants
n=128 Participants
|
9 participants
n=66 Participants
|
33 participants
n=194 Participants
|
|
Region of Enrollment
South Africa
|
11 participants
n=128 Participants
|
7 participants
n=66 Participants
|
18 participants
n=194 Participants
|
|
Region of Enrollment
United Kingdom
|
6 participants
n=128 Participants
|
3 participants
n=66 Participants
|
9 participants
n=194 Participants
|
|
Region of Enrollment
France
|
8 participants
n=128 Participants
|
4 participants
n=66 Participants
|
12 participants
n=194 Participants
|
|
Region of Enrollment
Germany
|
8 participants
n=128 Participants
|
5 participants
n=66 Participants
|
13 participants
n=194 Participants
|
|
Region of Enrollment
Spain
|
5 participants
n=128 Participants
|
2 participants
n=66 Participants
|
7 participants
n=194 Participants
|
PRIMARY outcome
Timeframe: 24 monthsPopulation: ITT
Mean rate of change in total area of macular atrophy, as measured by fundus autofluorescence (FAF)
Outcome measures
| Measure |
Emixustat
n=128 Participants
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
n=66 Participants
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
|---|---|---|
|
Mean Rate of Change in Total Area of Macular Atrophy, as Measured by Fundus Autofluorescence (FAF)
|
1.280 Rate of change from BL (mm^2/yr)
Standard Error 0.0783
|
1.309 Rate of change from BL (mm^2/yr)
Standard Error 0.1002
|
Adverse Events
Emixustat
Serious events: 9 serious events
Other events: 1 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Emixustat
n=127 participants at risk
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
n=66 participants at risk
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
|---|---|---|
|
Cardiac disorders
agina pectoris
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
1.5%
1/66 • Number of events 1 • 24 months
MedDRA version 25.0
|
|
Cardiac disorders
atrial fibrillation
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
neoplasm
|
1.6%
2/127 • Number of events 2 • 24 months
MedDRA version 25.0
|
1.5%
1/66 • Number of events 1 • 24 months
MedDRA version 25.0
|
|
Infections and infestations
Infections and infestations
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
1.5%
1/66 • Number of events 1 • 24 months
MedDRA version 25.0
|
|
Eye disorders
Visual disorder
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
|
Injury, poisoning and procedural complications
Injury, posioning and procedural
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
|
Nervous system disorders
Nervous system disorder
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
|
Vascular disorders
Vascular disorder
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
Other adverse events
| Measure |
Emixustat
n=127 participants at risk
10 mg
Emixustat: Once daily oral tablet taken for 24 months
|
Placebo
n=66 participants at risk
Includes identical tablets with only inactive ingredients (0 mg).
Placebo: Once daily oral tablet taken for 24 months
|
|---|---|---|
|
Eye disorders
Visual acuity reduced
|
0.79%
1/127 • Number of events 1 • 24 months
MedDRA version 25.0
|
0.00%
0/66 • 24 months
MedDRA version 25.0
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place