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

Who is this study for? Children, adolescents, and adults with Stargardt macular degeneration
What treatments are being studied? ALK-001
Status: Enrolling by invitation
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2

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

Participation Requirements
Sex: All
Minimum Age: 8
Maximum Age: 70
Healthy Volunteers: No

• 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

United States
Coordinating Center
Time Frame
Start Date: December 20, 2019
Estimated Completion Date: December 2024
Target number of participants: 140
Experimental: ALK-001
Leads: Alkeus Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov

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