Trial Outcomes & Findings for Phase 2/3 Study of ALK-001 in Geographic Atrophy (NCT NCT03845582)
NCT ID: NCT03845582
Last Updated: 2025-11-03
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
200 participants
Primary outcome timeframe
Baseline to 24 months
Results posted on
2025-11-03
Participant Flow
Target enrollment was 200 participants but ultimately only 198 were enrolled.
Participant milestones
| Measure |
ALK-001
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Overall Study
STARTED
|
135
|
63
|
|
Overall Study
COMPLETED
|
94
|
43
|
|
Overall Study
NOT COMPLETED
|
41
|
20
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2/3 Study of ALK-001 in Geographic Atrophy
Baseline characteristics by cohort
| Measure |
ALK-001
n=135 Participants
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 Participants
Capsule
Placebo oral capsule: Daily administration for 24 months
|
Total
n=198 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
>=65 years
|
130 Participants
n=3 Participants
|
60 Participants
n=15 Participants
|
190 Participants
n=18 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=3 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=18 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=3 Participants
|
3 Participants
n=15 Participants
|
8 Participants
n=18 Participants
|
|
Sex: Female, Male
Female
|
91 Participants
n=3 Participants
|
45 Participants
n=15 Participants
|
136 Participants
n=18 Participants
|
|
Sex: Female, Male
Male
|
44 Participants
n=3 Participants
|
18 Participants
n=15 Participants
|
62 Participants
n=18 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=3 Participants
|
4 Participants
n=15 Participants
|
6 Participants
n=18 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
133 Participants
n=3 Participants
|
59 Participants
n=15 Participants
|
192 Participants
n=18 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=3 Participants
|
0 Participants
n=15 Participants
|
0 Participants
n=18 Participants
|
|
Region of Enrollment
United States
|
135 participants
n=3 Participants
|
63 participants
n=15 Participants
|
198 participants
n=18 Participants
|
PRIMARY outcome
Timeframe: Baseline to 24 monthsOutcome measures
| Measure |
ALK-001
n=135 Participants
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 Participants
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)
|
1.62 mm2/year
Standard Error 0.081
|
1.87 mm2/year
Standard Error 0.116
|
SECONDARY outcome
Timeframe: Baseline to 24 monthsOutcome measures
| Measure |
ALK-001
n=135 Participants
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 Participants
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Number of Participants With Adverse Events
|
97 Participants
|
52 Participants
|
SECONDARY outcome
Timeframe: Baseline to 24 monthsChanges in LLVA and BCVA
Outcome measures
| Measure |
ALK-001
n=135 Participants
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 Participants
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Changes in Visual Acuity
LLVA
|
-3.896 letters
Standard Error 1.316
|
-8.298 letters
Standard Error 1.835
|
|
Changes in Visual Acuity
BCVA
|
-6.862 letters
Standard Error 1.251
|
-10.161 letters
Standard Error 1.771
|
Adverse Events
ALK-001
Serious events: 26 serious events
Other events: 59 other events
Deaths: 5 deaths
Placebo
Serious events: 14 serious events
Other events: 34 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
ALK-001
n=135 participants at risk
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 participants at risk
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Invasive ductal breast carcinoma
|
0.74%
1/135 • Two years
|
1.6%
1/63 • Two years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Large intestine benign neoplasm
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myeloid leukaemia
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Peritoneal carcinoma metastatic
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant peritoneal neoplasm
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
General disorders
Death
|
2.2%
3/135 • Two years
|
1.6%
1/63 • Two years
|
|
Injury, poisoning and procedural complications
Fall
|
1.5%
2/135 • Two years
|
1.6%
1/63 • Two years
|
|
Injury, poisoning and procedural complications
Jaw fracture
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Injury, poisoning and procedural complications
Concussion
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.00%
0/135 • Two years
|
3.2%
2/63 • Two years
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary fibrosis
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Product Issues
Device malfunction
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Vascular disorders
Aortic stenosis
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Hepatobiliary disorders
Biliary dilatation
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Infections and infestations
Gastrointestinal infection
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Infections and infestations
Rhinovirus infection
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Infections and infestations
Sepsis
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Infections and infestations
Urinary tract infection
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Infections and infestations
Corona virus infection
|
0.00%
0/135 • Two years
|
4.8%
3/63 • Two years
|
|
Infections and infestations
Infection
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Cardiac disorders
Atrial Fibrillation
|
1.5%
2/135 • Two years
|
0.00%
0/63 • Two years
|
|
Cardiac disorders
Acute coronary syndrome
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Cardiac disorders
Acute myocardial infarction
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Cardiac disorders
Cardiac failure
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Cardiac disorders
Cardiac failure congestive
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Cardiac disorders
Coronary artery disease
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Nervous system disorders
Cerebrovascular accident
|
3.7%
5/135 • Two years
|
1.6%
1/63 • Two years
|
|
Nervous system disorders
Dysarthria
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Nervous system disorders
Presyncope
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Nervous system disorders
Unresponsive to stimuli
|
0.74%
1/135 • Two years
|
0.00%
0/63 • Two years
|
|
Nervous system disorders
Syncope
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Cardiac disorders
Supraventricular extrasystoles
|
0.00%
0/135 • Two years
|
1.6%
1/63 • Two years
|
|
Infections and infestations
Pneumonia
|
2.2%
3/135 • Two years
|
0.00%
0/63 • Two years
|
Other adverse events
| Measure |
ALK-001
n=135 participants at risk
Capsule
ALK-001 oral capsule: Daily administration for 24 months
|
Placebo
n=63 participants at risk
Capsule
Placebo oral capsule: Daily administration for 24 months
|
|---|---|---|
|
Investigations
Blood triglycerides increased
|
11.1%
15/135 • Two years
|
7.9%
5/63 • Two years
|
|
Investigations
Blood alkaline phosphatase increased
|
7.4%
10/135 • Two years
|
7.9%
5/63 • Two years
|
|
Investigations
Glomerular filtration rate decreased
|
4.4%
6/135 • Two years
|
6.3%
4/63 • Two years
|
|
Infections and infestations
Corona virus infection
|
5.2%
7/135 • Two years
|
9.5%
6/63 • Two years
|
|
Infections and infestations
Urinary tract infection
|
3.7%
5/135 • Two years
|
7.9%
5/63 • Two years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.2%
7/135 • Two years
|
4.8%
3/63 • Two years
|
|
Injury, poisoning and procedural complications
Fall
|
6.7%
9/135 • Two years
|
9.5%
6/63 • Two years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place