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Clinical Trials in the USA / NCT04239625
Active, not recruiting Phase 2

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

NCT04239625 · tracked via the Priya Life Science USA tracker
Sponsor
Alkeus Pharmaceuticals, Inc
Phase
Phase 2
Started
2019-12-20
Last updated
2026-09-21

Condition(s) studied

Stargardt DiseaseStargardt Macular DegenerationStargardt Macular DystrophyAutosomal Recessive Stargardt Disease 1 (ABCA4-related)

Investigational drug(s) / intervention(s)

ALK-001 →

ALK-001: Oral administration of a pill for up to 24 months

Study 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

Eligibility

Sex
ALL
Min age
8 Years
Max age
70 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • 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
  • Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma — Up to 24 months

Trial sites (8)

FacilityCityRegionStatus
Alkeus Site Phoenix Arizona
Alkeus Site Los Angeles California
Alkeus Site Aurora Colorado
Alkeus Site Gainesville Florida
Alkeus Site Indianapolis Indiana
Alkeus Site Grand Rapids Michigan
Alkeus Site Westbury New York
Alkeus Site Silverdale Washington

More Alkeus Pharmaceuticals, Inc. trials in the USA

Study of ALK-001 on the Progression of Stargardt Disease Recruiting
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease Active, not recruiting

Other trials for the same condition

Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) Recruiting
Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease Recruiting
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions Recruiting
Study of ALK-001 on the Progression of Stargardt Disease Recruiting
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt Disease Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04239625 on ClinicalTrials.gov ↗ ← All trials in the USA