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Vitamin A Derivative

ALK-001 for Stargardt Disease

Phase 2

Waitlist Available

Research Sponsored by Alkeus Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor

Clinical diagnosis of Stargardt disease (STGD1)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 24 months

Awards & highlights

Study Summary

This trial is looking at the long-term safety and effects of a retinal drug for people with Stargardt disease, a rare genetic disorder that causes vision loss. The study is an extension of a previous trial, enrolling participants who are at least 8 years old.

Who is the study for?

This trial is for individuals at least 8 years old with a clinical diagnosis of Stargardt disease, who have two specific gene mutations (unless the sponsor says otherwise), and vision better than approximately 20/160 in one eye. Participants must be healthy overall, able to follow the study plan for 24 months, not pregnant or breastfeeding, and without recent ocular interventions or conditions that could affect study results.Check my eligibility

What is being tested?

The TEASE trial tests ALK-001's long-term safety and its impact on slowing down Stargardt disease progression. It's an extension of a previous study (NCT02402660) where participants are invited to continue treatment. The drug's behavior in the body over time will also be studied.See study design

What are the potential side effects?

While specific side effects aren't listed here, potential risks may include reactions related to vitamin A derivatives such as skin changes or vision disturbances since ALK-001 is a form of vitamin A. Regular monitoring will help identify any adverse effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have two mutations causing my ABCA4 disease, unless the sponsor has made an exception.

Select...

I have been diagnosed with Stargardt disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma

Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events

Trial Design

1Treatment groups

Experimental Treatment

Group I: ALK-001Experimental Treatment1 Intervention

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Alkeus Pharmaceuticals, Inc.Lead Sponsor

3 Previous Clinical Trials

480 Total Patients Enrolled

Leonide Saad, PhDStudy DirectorAlkeus Pharmaceuticals, Inc.

3 Previous Clinical Trials

480 Total Patients Enrolled

Media Library

ALK-001 (Vitamin A Derivative) Clinical Trial Eligibility Overview. Trial Name: NCT04239625 — Phase 2

Fundus Flavimaculatus Research Study Groups: ALK-001

Fundus Flavimaculatus Clinical Trial 2023: ALK-001 Highlights & Side Effects. Trial Name: NCT04239625 — Phase 2

ALK-001 (Vitamin A Derivative) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04239625 — Phase 2

Fundus Flavimaculatus Patient Testimony for trial: Trial Name: NCT04239625 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who would best benefit from participating in this trial?

"Patients aged 8 to 70 with Stargardt disease are encouraged to participate in this medical trial. Eligibility requirements include the presence of at least two ABCA4 mutations, visual acuity greater than 20/160 in one eye, and a commitment to adhere to study guidelines for 24 months."

Answered by AI

Does this medical trial represent a pioneering approach to treatment?

"Ever since 2015, ALK-001 has been trialled. Initially sponsored by Alkeus Pharmaceuticals Inc., the first study concluded in 2015 and was comprised of 140 patients. After successful Phase 1 results, 8 subsequent clinical trials were initiated across 10 cities in 2 nations."

Answered by AI

Are there still vacancies for participants in this clinical experiment?

"The clinicaltrial.gov website reveals that this medical study has finished recruiting participants, with the post dating back to December 20th 2019 and last updated on July 19th 2021. Fortunately, 168 other trials are presently enrolling candidates at present."

Answered by AI

Are there any age restrictions for enrollment in this experiment?

"The criteria for partaking in this trial necessitates that participants are between the ages of 8 to 70. There is a separate cohort with 15 trials open exclusively to people below 18, and another one accessible by patients over 65 which consists of 160 individuals."

Answered by AI

Has the drug ALK-001 been granted authorization by the FDA?

"Although there is some evidence pointing to ALK-001's safety, its efficacy has not been validated making it receive a rating of 2 on the scale."

Answered by AI

What medical issues is ALK-001 most often used to treat?

"ALK-001 is traditionally used to manage Vitamin d deficiency, but can also be beneficial in treating Vitamin A deficiencies and aiding with nutritional supplementation."

Answered by AI

What is the current number of individuals participating in this research?

"Unfortunately, this clinical trial is no longer in need of participants. The study launched on December 20th 2019 and ended its recruitment phase as recently as July 19th 2021. For those seeking research opportunities, there are currently 160 trials for Stargardt's Disease and 8 studies involving ALK-001 that are actively enrolling patients."

Answered by AI

Could you provide a synopsis of the previous research conducted with ALK-001?

"Presently, 8 clinical trials are exploring ALK-001's efficacy. Two of these studies have reached Phase 3, and can be found at 13 medical centres across America; the majority being in Somerville, Massachusetts."

Answered by AI