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

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

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

160 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-08-31

Study Completion Date

2026-11-30

Brief Summary

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The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 8 and 70 years old.

Funding Source - FDA OOPD

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Detailed Description

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This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related). Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. Stargardt disease is caused by a defective ABCA4 gene, which affects the processing of vitamin A in the eye and leads to the formation of toxic vitamin A aggregates (called "vitamin A dimers") in the eye. Vitamin A dimers are thought to contribute to vision loss in Stargardt disease. ALK-001, the investigational drug, is a chemically-modified vitamin A designed as a replacement of vitamin A to prevent the formation of toxic vitamin A dimers in the eye. Trial participants will receive either ALK-001 or placebo, and follow-up visits will take place periodically for up to 24 months. There is currently no treatment for Stargardt disease.

Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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ALK-001

Daily, oral administration of one capsule. See details below.

Group Type EXPERIMENTAL

ALK-001

Intervention Type DRUG

Daily, oral administration for 24 months

Placebo

Daily, oral administration of one capsule. See details below.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Daily, oral administration for 24 months

Interventions

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ALK-001

Daily, oral administration for 24 months

Intervention Type DRUG

Placebo

Daily, oral administration for 24 months

Intervention Type DRUG

Other Intervention Names

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C20-D3-Retinyl Acetate C20 Deuterated vitamin A

Eligibility Criteria

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Inclusion Criteria

* Male or female between 8 and 70 years old (inclusive), with any visual acuity
* Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)
* Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required.
* At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study
* Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging
* 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 signed and dated the informed consent forms (or assent where appropriate) to participate
* Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements

Exclusion Criteria

* Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
* Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization
* Has concurrent medical condition or history, which in the opinion of the investigator, is likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or study procedures
* Has clinically significant abnormal laboratory result(s) at screening
* Has active or historical acute or chronic liver disorder
* Has active or historical ocular disorder in the primary study eye that, in the opinion of the investigator, may confound assessment of the retina morphologically or functionally (this could include for example cataract surgery within the past 6 months, choroidal neovascularization (CNV), glaucoma, recurring uveitis, diabetic retinopathy, other retinal disease, etc.)
* Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit
* Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater

Minimum Eligible Age

8 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No