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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-12-20

Study Completion Date

2026-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

NCT02402660

Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy +1 more

ENROLLING_BY_INVITATION PHASE2

Phase 1 Safety Study of ALK-001 in Healthy Volunteers

NCT02230228

Stargardt Disease Age-related Macular Degeneration Other Retinal Dystrophies

COMPLETED PHASE1

Safety and Efficacy of Cyclosporine Ophthalmic Emulsion in Patients With Primary Pterygium

NCT01109056

Pterygium

COMPLETED PHASE2

An Open-Label Study of the Effect of an Ophthalmic Solution on Asteroid Hyalosis

NCT00825942

Asteroid Hyalosis

UNKNOWN PHASE2

Topical Cyclosporine-A for Management of Epiphora

NCT04637633

Epiphora

COMPLETED PHASE2/PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

ALK-001

Group Type EXPERIMENTAL

ALK-001

Intervention Type DRUG

Oral administration of a pill for up to 24 months

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

ALK-001

Oral administration of a pill for up to 24 months

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

C20-D3-Retinyl Acetate C20 Deuterated vitamin A

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

8 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No