Stargardt DiseasePhase 3NORTHSTARNCT07419334
Study of ALK-001 on the Progression of Stargardt Disease
A Phase 3 study testing ALK-001 in people with Stargardt Disease, run by Alkeus Pharmaceuticals, Inc. at 14 sites in United States.
Trial 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) Read more about this study
- Who is it for?
- Ages 8 to 45 years
- Conditions the study is for
- Stargardt Disease
- What is being tested?
- ALK-001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 230 participants
- Where
- 14 sitesUnited States
- Study dates
- 3 Jun 2026to Dec 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
14 sites in 1 country. Contact the study team or a site near you to ask about taking part.
For trial questions: trials@alkeuspharma.com or 877-255-7476 contact
14 sites in 1 country
United States14 sites, 14 recruiting
- Recruiting
University of Colorado SOM, Sue-Anschutz-Rodgers Eye Center
Aurora, Colorado, 80045
Diksha Pradhan, 303-724-9094, diksha.pradhan@cuanschutz.edu
Marc Mathias, MD
- Not yet recruiting
Tufts Medical Center - New England Eye Center
Boston, Massachusetts, 02116
Lainey Mullen, Lainey.Mullen@tuftsmedicine.org
Helen Rahman, Helen.Rahman@tuftsmedicine.org
- Recruiting
Ohio State Havener Eye Institute
Columbus, Ohio, 43212
Lindsey Pyers, 614-293-8116, lindsey.pyers@osumc.edu
Thomas Mendel, MD
- Recruiting
Retina Foundation of the Southwest
Dallas, Texas, 75231
Jessica Watson, 214-363-3911 ext. 136, jclark@retinafoundation.org
Karl Csaky, MD, PhD
- Recruiting
Erie Retina Research
Erie, Pennsylvania, 16505
Bethany Bielak-Scott, 814-200-9152, b.scott.research@pm.me
David Almeida, MD, MBA, PhD
- Recruiting
The Retina Group of Washington
Fairfax, Virginia, 22031
Katrina Arias, 703-698-9335, karias@rgw.com
Alexander Melamud, MD
- Recruiting
Star Retina
Fort Worth, Texas, 76132
Heather Gleason, 817-378-4777, hgleasonresearch@starretina.com
Courtney Crawford, MD, FACS
- Recruiting
Vitreo Retinal Associates
Gainesville, Florida, 32607
Hailey Ahrens, 352-300-8412, hailey@vra-pa.com
Christine Kay, MD
- Recruiting
Foundation for Vision Research
Grand Rapids, Michigan, 49546
Holly Vincent, 616-438-9395, holly@fvr.org
Liliya Sutherland, MD
- Recruiting
Wagner Kapoor Research Institute
Norfolk, Virginia, 23666
Sally Whitaker, 757-271-3346, research-staff@wagnerkapoor.com
Kapil Kapoor, MD
- Not yet recruiting
Mid Atlantic Retina
Philadelphia, Pennsylvania, 19073
Study Coordinator, research@midatlanticretina.com
Sunir Garg, MD
- Recruiting
Associated Retina Consultants
Phoenix, Arizona, 85020
Mallory Mintert, hello@doctrials.com
Benjamin Bakall, MD, PhD
- Recruiting
UPMC Vision Institute
Pittsburgh, Pennsylvania, 15219
Study Coordinator, clinicaltrialsoph@upmc.edu
Boris Rosin, MD
- Recruiting
Medical Center Ophthalmology Associates
San Antonio, Texas, 78240
Roxanne Casillas, 210-697-2020, rcasillas@mcoaeyecare.com
Michael Singer, MD
About this study
Official title: 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)
- Conditions
- Stargardt Disease
- Being tested
- ALK-001, Placebo
- Keywords
- Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALK-001Drug
Also called Gildeuretinol acetate
Daily oral administration for 24 months
PlaceboDrug
Daily oral administration for 24 months
| Study group | Role | What they receive |
|---|---|---|
| Active | Active comparator | Not described Receives: Drug: ALK-001 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence
Measured: Month 6 to Month 24
Secondary outcomes (1)
Changes in Low Luminance Visual Acuity from baseline to Month 24
Changes in Low Luminance Visual Acuity from baseline to Month 24
Measured: baseline to Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2026
- Study start3 Jun 2026
- Primary completionOct 2029 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Stargardt DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 230 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07419334
- Sponsor's protocol ID
- ALK001-P1004
- Other IDs
- 2026-525296-60-00
- Acronym
- NORTHSTAR
- Status last verified
- Sep 2026
- First submitted
- 10 Feb 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07419334
- Acronym / sponsor ID
- NORTHSTAR / ALK001-P1004
- Conditions
- Stargardt Disease
- Keywords
- Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 230 participants (estimated)
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Jun 2026 (actual)
- Primary completion
- Oct 2029 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 19 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Active comparator | Drug: ALK-001 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 8 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALK-001Also: Gildeuretinol acetateDaily oral administration for 24 months | Drug | Active |
| PlaceboDaily oral administration for 24 months | Drug | Placebo |
Primary outcome
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
1 secondary outcome
Changes in Low Luminance Visual Acuity from baseline to Month 24
Time frame: baseline to Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
For trial questions: trials@alkeuspharma.com or 877-255-7476 contact
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ALK-001 in people with Stargardt Disease, run by Alkeus Pharmaceuticals, Inc. at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact For trial questions: trials@alkeuspharma.com or 877-255-7476 (877-255-7476, trials@alkeuspharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence (time frame: Month 6 to Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.