- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04239625
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
January 25, 2024 updated by: Alkeus Pharmaceuticals, Inc.
A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease. This study is an extension of NCT02402660 and enrolls participants who are at least 8 years old. Enrollment is by invitation only.
Funding Source - FDA OOPD
Study Overview
Status
Enrolling by invitation
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Estimated)
140
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Massachusetts
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Somerville, Massachusetts, United States, 02144
- Coordinating Center
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
8 years to 70 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Simplified Inclusion Criteria:
- Clinical diagnosis of Stargardt disease (STGD1)
- Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
- Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye
- Healthy as judged by investigator
- Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
- Has been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate
- Female of childbearing potential has signed the attestation on contraception requirements
Simplified Exclusion Criteria:
- Is lactating or pregnant
- Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures
- Has abnormal laboratory result(s) at screening
- Has an ocular disorder that may confound ocular assessments
- Has a history of ocular intervention within 90 days of screening
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: ALK-001
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Oral administration of a pill for up to 24 months
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events
Time Frame: From baseline to 24 months
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From baseline to 24 months
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Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma
Time Frame: Up to 24 months
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Up to 24 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Leonide Saad, PhD, Alkeus Pharmaceuticals, Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- Ma L, Kaufman Y, Zhang J, Washington I. C20-D3-vitamin A slows lipofuscin accumulation and electrophysiological retinal degeneration in a mouse model of Stargardt disease. J Biol Chem. 2011 Mar 11;286(10):7966-7974. doi: 10.1074/jbc.M110.178657. Epub 2010 Dec 14.
- Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011 Mar 11;286(10):7958-7965. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12.
- Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D(3)-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013.
- Charbel Issa P, Barnard AR, Herrmann P, Washington I, MacLaren RE. Rescue of the Stargardt phenotype in Abca4 knockout mice through inhibition of vitamin A dimerization. Proc Natl Acad Sci U S A. 2015 Jul 7;112(27):8415-20. doi: 10.1073/pnas.1506960112. Epub 2015 Jun 23.
- Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
December 20, 2019
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
December 1, 2026
Study Registration Dates
First Submitted
January 11, 2020
First Submitted That Met QC Criteria
January 22, 2020
First Posted (Actual)
January 27, 2020
Study Record Updates
Last Update Posted (Estimated)
January 26, 2024
Last Update Submitted That Met QC Criteria
January 25, 2024
Last Verified
January 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Eye Diseases
- Retinal Degeneration
- Retinal Diseases
- Genetic Diseases, Inborn
- Eye Diseases, Hereditary
- Macular Degeneration
- Stargardt Disease
- Physiological Effects of Drugs
- Antineoplastic Agents
- Immunologic Factors
- Protective Agents
- Micronutrients
- Adjuvants, Immunologic
- Vitamins
- Anticarcinogenic Agents
- Vitamin A
- Retinol acetate
Other Study ID Numbers
- ALK001-P1002-EXT
- R01FD004098 (U.S. FDA Grant/Contract)
- R01FD006016 (Other Grant/Funding Number: FDA OOPD)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
UNDECIDED
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Stargardt Disease
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Alkeus Pharmaceuticals, Inc.RecruitingStargardt Disease | Stargardt Macular Degeneration | Stargardt Macular Dystrophy | Autosomal Recessive Stargardt Disease 1 (ABCA4-related)United States
-
Institute of Molecular and Clinical Ophthalmology...RecruitingStargardt Disease | Stargardt Disease 1 | Fundus Flavimaculatus | Macular Degeneration, Stargardt | Macular Dystrophy With Flecks, Type 1Switzerland
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West China HospitalActive, not recruiting
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Belite Bio, IncActive, not recruitingStargardt Disease 1United States, Australia, Belgium, China, France, Germany, Hong Kong, Netherlands, Switzerland, Taiwan, United Kingdom
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IVERIC bio, Inc.Active, not recruitingStargardt Disease 1Italy, United States, Israel, United Kingdom, Germany, Spain, Hungary, France
-
Stargazer Pharmaceuticals, Inc.Completed
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Queen's University, BelfastUniversity of SussexUnknownRetinitis Pigmentosa | Low Vision | Albinism | Stargardt Disease 1 | Stargardt Disease 3 | Stargardt Disease 4United Kingdom
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Ophthalmos Research and Education InstituteCompletedStargardt Disease 1 | Dry AMDFrance, Germany, Italy
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Pomeranian Medical University SzczecinUnknownRetinal Degeneration | Retinitis Pigmentosa | Age Related Macular Degeneration | Stargardt Disease 1Poland
-
Barcelona Macula FoundationRecruitingRetinitis Pigmentosa | Choroideremia | Stargardt Disease 1 | Best Disease | Pattern Dystrophy of MaculaSpain
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