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Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) | Clinical Trials | Clareo Health

ACTIVE_NOT_RECRUITINGPHASE2

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

NCT04239625•Alkeus Pharmaceuticals, Inc.

View on ClinicalTrials.gov

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

Age

8 - 70 years

Sex

ALL

Healthy Volunteers

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

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

Locations (8)

Alkeus Site

Phoenix, Arizona, United States

Alkeus Site

Los Angeles, California, United States

Alkeus Site

Aurora, Colorado, United States

Alkeus Site

Gainesville, Florida, United States

Alkeus Site

Indianapolis, Indiana, United States

Alkeus Site

Grand Rapids, Michigan, United States

Alkeus Site

Westbury, New York, United States

Alkeus Site

Silverdale, Washington, United States

Key Dates

Start Date

December 20, 2019

Primary Completion Date

December 1, 2026

Completion Date

December 1, 2026

Last Updated

April 27, 2025

Enrollment

200

ESTIMATED participants

Conditions

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

Interventions

ALK-001

DRUG

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: April 27, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt Disease

Oral Metformin for Treatment of ABCA4 Retinopathy

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Inherited Retinal Diseases: Natural History and Genotype-Phenotype Correlations

Restoration of Central Vision With PRIMA in Patients With Photoreceptor Degeneration