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Vitamin A Derivative

ALK-001 for Stargardt Disease (TEASE Trial)

Phase 2
Recruiting
Research Sponsored by Alkeus Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female between 8 and 70 years old (inclusive), with any visual acuity
Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 24 months
Awards & highlights

TEASE Trial Summary

This trial is exploring the long term safety and effectiveness of a treatment for Stargardt disease, a genetic disorder that causes vision loss. The treatment, ALK-001 (C20-D3-retinyl acetate), is being given to patients 8 to 70 years old to see if it slows the progression of the disease. The trial is funded by the FDA's Office of Orphan Products Development.

Who is the study for?
This trial is for individuals aged 8-70 with Stargardt disease, a genetic eye condition. Participants must have clear vision media and no allergies to eye dilation drops, be healthy overall, able to follow the study plan for two years, and females of childbearing age must agree to use contraception. Those with certain medical conditions or recent eye treatments are excluded.Check my eligibility
What is being tested?
The trial tests ALK-001 (C20-D3-retinyl acetate) against a placebo to assess its safety over the long term and its impact on slowing down the progression of Stargardt disease. Patients will either receive ALK-001 or a placebo in this randomized study.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any adverse reactions due to ALK-001 as compared to those receiving a placebo. Side effects could range from minor issues at the site of administration to more systemic responses.

TEASE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I am between 8 and 70 years old and my ability to see is not limited.
Select...
I have been diagnosed with Stargardt disease.
Select...
My eyes can be clearly imaged and I'm not allergic to dilation drops.

TEASE Trial Timeline

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
Safety and tolerability of 24 months of daily dosing of ALK-001 assessed by Incidence and/or clinically significant changes of a combination of ocular and non-ocular adverse events
Secondary outcome measures
Effects of ALK-001 on the progression of Stargardt disease
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma

TEASE Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: ALK-001Experimental Treatment1 Intervention
Daily, oral administration of one capsule. See details below.
Group II: PlaceboPlacebo Group1 Intervention
Daily, oral administration of one capsule. See details below.

Find a Location

Who is running the clinical trial?

Alkeus Pharmaceuticals, Inc.Lead Sponsor
3 Previous Clinical Trials
480 Total Patients Enrolled
Hendrik Scholl, MDStudy DirectorUniversity of Basel
1 Previous Clinical Trials
259 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: NCT02402660 — Phase 2
Fundus Flavimaculatus Research Study Groups: ALK-001, Placebo
Fundus Flavimaculatus Clinical Trial 2023: ALK-001 Highlights & Side Effects. Trial Name: NCT02402660 — Phase 2
ALK-001 (Vitamin A Derivative) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02402660 — Phase 2
Fundus Flavimaculatus Patient Testimony for trial: Trial Name: NCT02402660 — Phase 2

Frequently Asked Questions

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

What risks should be considered when using ALK-001?

"Despite there being no evidence of ALK-001's efficacy, the drug has been assigned a safety score of 2 owing to existing clinical data."

Answered by AI

What afflictions can ALK-001 be employed to ameliorate?

"ALK-001 is typically used to address vitamin d deficiencies, but it may also be prescribed for diseases related to vitamin A deficiency and nutritional supplementation."

Answered by AI

What is the current number of participants in this clinical trial?

"This trial necessitates 140 properly qualified candidates, who can be sourced from multiple locations including the University of California Los Angeles - Jules Stein Eye Institute in LA and Columbia University Medical Center - Harkness Eye Institute located in NYC."

Answered by AI

How many sites are actively administering this research?

"This research project is being conducted at 4 different medical sites, which include Los Angeles, New york and Gainesville. For the convenience of those interested in enrolling, it's crucial to select a clinic close by thus reducing travel time."

Answered by AI

Is this experimental medical investigation unique and original?

"Alkeus Pharmaceuticals, Inc. first began researching ALK-001 in 2015 and conducted a clinical trial with 140 participants that year; it was subsequently granted Phase 2 drug approval. Currently, 8 distinct studies on ALK-001 are ongoing across 10 cities spread over two countries."

Answered by AI

Are there any analogous experiments involving ALK-001?

"As of now, 8 clinical trials for ALK-001 are active with 2 being in Phase 3. Primarily based in Somerville, MA, 13 different sites across the country have studies involving this drug."

Answered by AI

Is the target demographic of this research investigation inclusive of individuals over 20 years old?

"This research initiative seeks participants aged 8 and above, but below the age of 70."

Answered by AI

Do I possess the necessary qualifications to partake in this clinical study?

"Individuals with stargardt disease who are between 8 and 70 years old have the opportunity to apply for this trial. A total of 140 applicants will be accepted."

Answered by AI

Is this research project available for participants at the moment?

"Affirmative, clinicaltrials.gov attests that this medical trial is recruiting volunteers for participation. Initially posted on August 1st 2015 and most recently updated on February 15th 2022, the experiment intends to enlist 140 individuals from 4 separate sites."

Answered by AI

Who else is applying?

What state do they live in?
Ontario
Tennessee
Other
California
How old are they?
65+
18 - 65
< 18
What site did they apply to?
Vitreoretinal Associates
Other
University of California Los Angeles - Jules Stein Eye Institute
Columbia University Medical Center - Harkness Eye Institute
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
3+
0

Why did patients apply to this trial?

My doctor recommends that I enroll in a clinical trial to help with my failing eyesight.
PatientReceived 2+ prior treatments
I have sent all my images to your trial 2 years ago and want to try again. I never had follow through. I also did testing for The Dragon Trial Biolife at Stanford but was not selected. I want to stop progression, my eyes changed drastically this past year. I will be attending college this fall and want to make sure I advocate for myself and my eye health and opportunities.
PatientReceived 1 prior treatment

What questions have other patients asked about this trial?

How often would I have to go to Los Angles? When is the trial complete? What phase is the trial in?
PatientReceived 1 prior treatment

How responsive is this trial?

Most responsive sites:
  1. University of California Los Angeles - Jules Stein Eye Institute: < 48 hours
Average response time
  • < 2 Days
Typically responds via
Email
Recent research and studies
~9 spots leftby Dec 2024