NCT04239625Active, not recruitingPhase 2
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
Source: ClinicalTrials.gov · Last updated 21 Sept 2026
Brief summary (registry, in English)
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only. Funding Source - FDA OOPD
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 200 (estimated)
- First submitted
- 11 Jan 2020
- Start date
- 20 Dec 2019
- Primary completion date
- Dec 2028
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.
- Diseases
- Macular degeneration
- NCT number
- NCT04239625
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 200
- From 20 Dec 2019 to the primary completion date, Dec 2028
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Criteria the registry did not label
- Simplified Inclusion Criteria:
- Clinical diagnosis of Stargardt disease (STGD1)
- Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
- Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
- Healthy as judged by investigator
- Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
- Has been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
- Female of childbearing potential has signed the attestation on contraception requirements
- Simplified Exclusion Criteria:
- Is lactating or pregnant
- Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
- Has abnormal laboratory result(s) at screening
- Has an ocular disorder that may confound ocular assessments
- Has a history of ocular intervention within 90 days of screening
Age in the registry: 8 years to 70 years · Sex: All · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - Coordinator of this site? Confirm its status | Aurora | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Gainesville | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Grand Rapids | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Indianapolis | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Silverdale | United States | Unknown status |
| - Coordinator of this site? Confirm its status | Westbury | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.