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

Alkeus Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: Stargardt Disease

Treatments: Drug: Placebo; Drug: ALK-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT07419334

2026-525296-60-00 (EU Trial (CTIS) Number)

ALK001-P1004

Details and patient eligibility

About

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)

Enrollment: 230 estimated patients

Sex: All

Ages: 8 to 45 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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230 participants in 2 patient groups, including a placebo group

Active

Active Comparator group

Treatment:

Drug: ALK-001

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo