Study of ALK-001 on the Progression of Stargardt Disease

NCT07419334 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: ALK001-P10042026-525296-60-00
• Study Type: interventional
• Target: 230
• Locations: 1 country
• Sites: 3

Brief Summary

This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)

Conditions

Stargardt Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence

  Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence

  Month 6 to Month 24

Secondary Outcomes (1)

• Changes in Low Luminance Visual Acuity from baseline to Month 24

  baseline to Month 24

Study Arms (2)

Active

ACTIVE COMPARATOR

Drug: ALK-001

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

ALK-001 DRUG

Daily oral administration for 24 months

Also known as: Gildeuretinol acetate

Active

Placebo DRUG

Daily oral administration for 24 months

Placebo

Eligibility Criteria

• Age: 8 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female, 8 to 45 years of age (inclusive) on the day of screening.
• Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP.
• Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy
• Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation.
• Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.

You may not qualify if:

• Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.
• Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study.
• Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months.
• Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes.
• Anticipates participating in any other drug or device study within the duration of the study.

Sponsors & Collaborators

• Alkeus Pharmaceuticals, Inc.: lead

Study Sites (3)

Associated Retina Consultants

Phoenix, Arizona, 85020, United States

RECRUITING

Vitreo Retinal Associates

Gainesville, Florida, 32607, United States

NOT YET RECRUITING

Erie Retina Research

Erie, Pennsylvania, 16505, United States

RECRUITING

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

For trial questions: trials@alkeuspharma.com or 877-255-7476

CONTACT

877-255-7476; trials@alkeuspharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2026
• First Posted: February 19, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): October 1, 2029
• Study Completion (Estimated): December 1, 2030
• Last Updated: May 5, 2026

Record last verified: 2026-05

Locations

US (3)