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NCT Number: NCT04239625

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Stargardt Disease Autosomal Recessive Stargardt Disease 1 (ABCA4-related) Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases Stargardt Macular Degeneration Stargardt Macular Dystrophy

Age range

8 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Alkeus Site, Phoenix, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALK-001

Drug

Oral administration of a pill for up to 24 months

Other names: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A

Primary outcomes

  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

Sponsors and collaborators

Lead sponsor

Alkeus Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2019
Primary completion
2026
Study completion
2026
First posted
Jan 27, 2020
Registry last updated
Apr 27, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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