Study of ALK-001 on the Progression of Stargardt Disease | NCT07419334

Studies
Study of ALK-001 on the Progression of Stargardt Disease

NCT07419334

Sponsor

Alkeus Pharmaceuticals, Inc.

Status

Recruiting

Last Update Posted

Jul 2, 2026Actual

Enrollment

230Estimated

Phase

Phase 3

Conditions

Stargardt Disease

Interventions

ALK-001
Placebo

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
2026-525296-60-00	EU Trial (CTIS) Number

Status Module

Record Verification Date

Jul 2026

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2026Estimated

Primary Completion Date

Oct 2029Estimated

Completion Date

Dec 2030Estimated

First Submitted Date

Feb 10, 2026

First Submission Date that Met QC Criteria

Feb 13, 2026

First Posted Date

Feb 19, 2026Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 1, 2026

Last Update Posted Date

Jul 2, 2026Actual

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

230Estimated

Label	Type	Description	Intervention Names
Active	Active Comparator		Drug: ALK-001
Placebo	Placebo Comparator		Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
ALK-001	Drug	Daily oral administration for 24 months	Active	Gildeuretinol acetate

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

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

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria

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.

Exclusion Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

8 Years

Maximum Age

45 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D015785	Eye Diseases, Hereditary
D005128	Eye Diseases
D008268	Macular Degeneration
D012162	Retinal Degeneration