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Trial record 17 of 36 for:    stargardt

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. Identifier: NCT04239625
Recruitment Status : Enrolling by invitation
First Posted : January 27, 2020
Last Update Posted : January 27, 2020
Information provided by (Responsible Party):
Alkeus Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE January 11, 2020
First Posted Date  ICMJE January 27, 2020
Last Update Posted Date January 27, 2020
Actual Study Start Date  ICMJE December 20, 2019
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 22, 2020)
  • 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 ]
  • Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma [ Time Frame: Up to 24 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
Official Title  ICMJE 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
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

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Stargardt Disease
  • Stargardt Macular Degeneration
  • Stargardt Macular Dystrophy
  • Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Intervention  ICMJE Drug: ALK-001
Oral administration of a pill for up to 24 months
Other Names:
  • C20-D3-Retinyl Acetate
  • C20 Deuterated vitamin A
Study Arms  ICMJE Experimental: ALK-001
Intervention: Drug: ALK-001
Publications *

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: January 22, 2020)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT04239625
Other Study ID Numbers  ICMJE ALK001-P1002-EXT
R01FD004098 ( U.S. FDA Grant/Contract )
R01FD006016 ( Other Grant/Funding Number: FDA OOPD )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party Alkeus Pharmaceuticals, Inc.
Study Sponsor  ICMJE Alkeus Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Leonide Saad, PhD Alkeus Pharmaceuticals, Inc.
PRS Account Alkeus Pharmaceuticals, Inc.
Verification Date January 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP