Stargardt Disease
Conditions
Keywords
Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Brief summary
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)
Interventions
DRUGALK-001
Daily oral administration for 24 months
DRUGPlacebo
Daily oral administration for 24 months
Sponsors
Alkeus Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
8 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
1. Male or female, 8 to 45 years of age (inclusive) on the day of screening. 2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP. 3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy 4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation. 5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.
Exclusion criteria
1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization. 2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study. 3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months. 4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes. 5. Anticipates participating in any other drug or device study within the duration of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence | Month 6 to Month 24 | Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Changes in Low Luminance Visual Acuity from baseline to Month 24 | baseline to Month 24 | Changes in Low Luminance Visual Acuity from baseline to Month 24 |
Countries
United States
Contacts
CONTACTFor trial questions: trials@alkeuspharma.com or 877-255-7476
Outcome results
None listed