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Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04239625
Recruitment Status : Enrolling by invitation
First Posted : January 27, 2020
Last Update Posted : July 21, 2021
Sponsor:
Information provided by (Responsible Party):
Alkeus Pharmaceuticals, Inc.

Study Description
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Brief Summary:

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


Condition or disease Intervention/treatment Phase
Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy Autosomal Recessive Stargardt Disease 1 (ABCA4-related) Drug: ALK-001 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : December 20, 2019
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: ALK-001 Drug: ALK-001
Oral administration of a pill for up to 24 months
Other Names:
  • C20-D3-Retinyl Acetate
  • C20 Deuterated vitamin A


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]
  2. Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma [ Time Frame: Up to 24 months ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04239625


Locations
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United States, Massachusetts
Coordinating Center
Somerville, Massachusetts, United States, 02144
Sponsors and Collaborators
Alkeus Pharmaceuticals, Inc.
Investigators
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Study Director: Leonide Saad, PhD Alkeus Pharmaceuticals, Inc.
More Information
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Publications:

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Responsible Party: Alkeus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04239625    
Other Study ID Numbers: ALK001-P1002-EXT
R01FD004098 ( U.S. FDA Grant/Contract )
R01FD006016 ( Other Grant/Funding Number: FDA OOPD )
First Posted: January 27, 2020    Key Record Dates
Last Update Posted: July 21, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Macular Degeneration
Stargardt Disease
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Vitamin A
Retinol acetate
 Vitamins
Micronutrients
Physiological Effects of Drugs
Protective Agents
Anticarcinogenic Agents
Antineoplastic Agents
Adjuvants, Immunologic
Immunologic Factors