Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

Phase 2

Active, not recruiting

• Conditions: Stargardt Macular Dystrophy; Autosomal Recessive Stargardt Disease 1 (ABCA4-related); Stargardt Disease; Stargardt Macular Degeneration
• Interventions: Drug: ALK-001

• Registration Number: NCT04239625
• Lead Sponsor: Alkeus Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2019-12-20
• Study First Submitted: 2020-01-11
• Study First Submitted QC: 2020-01-22
• Study First Posted: 2020-01-27
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-24
• Last Update Posted: 2025-04-27
• Primary Completion: 2026-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 200

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ALK-001	ALK-001	-

Primary Outcome Measures

Name	Time	Method
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma	Up to 24 months
Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events	From baseline to 24 months

Trial Locations

Locations (1)

Alkeus Site; 🇺🇸 Silverdale, Washington, United States