Study of ALK-001 on the Progression of Stargardt Disease (NORTHSTAR)

May 1, 2026 updated by: Alkeus Pharmaceuticals, Inc.

A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

230

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: For trial questions: trials@alkeuspharma.com or 877-255-7476
  • Phone Number: 877-255-7476
  • Email: trials@alkeuspharma.com

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85020
        • Recruiting
        • Associated Retina Consultants
        • Principal Investigator:
          • Benjamin Bakall, MD, PhD
        • Contact:
    • Florida
      • Gainesville, Florida, United States, 32607
        • Not yet recruiting
        • Vitreo Retinal Associates
        • Principal Investigator:
          • Christine Kay, MD
        • Contact:
    • Pennsylvania
      • Erie, Pennsylvania, United States, 16505
        • Recruiting
        • Erie Retina Research
        • Principal Investigator:
          • David Almeida, MD, MBA, PhD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria

  1. Male or female, 8 to 45 years of age (inclusive) on the day of screening.
  2. 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.
  3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy
  4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation.
  5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.

Exclusion Criteria

  1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.
  2. 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.
  3. 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.
  4. 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.
  5. Anticipates participating in any other drug or device study within the duration of the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Daily oral administration for 24 months
Active Comparator: Active
Drug: ALK-001
Daily oral administration for 24 months
Other Names:
  • Gildeuretinol acetate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence
Time Frame: Month 6 to Month 24
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence
Month 6 to Month 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in Low Luminance Visual Acuity from baseline to Month 24
Time Frame: baseline to Month 24
Changes in Low Luminance Visual Acuity from baseline to Month 24
baseline to Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alkeus Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

October 1, 2029

Study Completion (Estimated)

December 1, 2030

Study Registration Dates

First Submitted

February 10, 2026

First Submitted That Met QC Criteria

February 13, 2026

First Posted (Actual)

February 19, 2026

Study Record Updates

Last Update Posted (Actual)

May 5, 2026

Last Update Submitted That Met QC Criteria

May 1, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALK001-P1004
  • 2026-525296-60-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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