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

Who is this study for? Patients with Stargardt Disease
What treatments are being studied? ALK-001 Oral Capsule
Status: Enrolling_by_invitation
Location: See all (14) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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

Eligibility
Participation Requirements
Sex: All
Minimum Age: 8
Maximum Age: 70
Healthy Volunteers: f
View:

• 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

Locations
United States
Arizona
Alkeus Site
Phoenix
California
Alkeus Site
Los Angeles
Colorado
Alkeus Site
Aurora
Florida
Alkeus Site
Gainesville
Alkeus Site
Miami
Indiana
Alkeus Site
Indianapolis
Maryland
Alkeus Site
Baltimore
Michigan
Alkeus Site
Grand Rapids
New York
Alkeus Site
New York
Alkeus Site
Westbury
Texas
Alkeus Site
Houston
Utah
Alkeus Site
Salt Lake City
Washington
Alkeus Site
Silverdale
Wisconsin
Alkeus Site
Milwaukee
Time Frame
Start Date: 2015-08
Completion Date: 2026-11-30
Participants
Target number of participants: 160
Treatments
Experimental: ALK-001
Daily, oral administration of one capsule. See details below.
Placebo_comparator: Placebo
Daily, oral administration of one capsule. See details below.
Sponsors
Leads: Alkeus Pharmaceuticals, Inc.

This content was sourced from clinicaltrials.gov

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