search
Back to results

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

Primary Purpose

Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy

Status
Enrolling by invitation
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
ALK-001
Sponsored by
Alkeus Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Stargardt Disease

Eligibility Criteria

8 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Coordinating Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ALK-001

Arm Description

Outcomes

Primary Outcome Measures

Safety and tolerability of ALK-001 assessed by incidence and/or clinically-significant changes of a combination of ocular and non-ocular adverse events
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma

Secondary Outcome Measures

Full Information

First Posted
January 11, 2020
Last Updated
July 19, 2021
Sponsor
Alkeus Pharmaceuticals, Inc.
search

1. Study Identification

Unique Protocol Identification Number
NCT04239625
Brief Title
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Enrolling by invitation
Study Start Date
December 20, 2019 (Actual)
Primary Completion Date
December 2024 (Anticipated)
Study Completion Date
December 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alkeus Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
140 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ALK-001
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
ALK-001
Other Intervention Name(s)
C20-D3-Retinyl Acetate, C20 Deuterated vitamin A
Intervention Description
Oral administration of a pill for up to 24 months
Primary Outcome Measure Information:
Title
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
Title
Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma
Time Frame
Up to 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
8 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Leonide Saad, PhD
Organizational Affiliation
Alkeus Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Coordinating Center
City
Somerville
State/Province
Massachusetts
ZIP/Postal Code
02144
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Undecided
Citations:
PubMed Identifier
21156790
Citation
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.
Results Reference
background
PubMed Identifier
21075840
Citation
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.
Results Reference
background
PubMed Identifier
23914132
Citation
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.
Results Reference
background
PubMed Identifier
26106163
Citation
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.
Results Reference
background
PubMed Identifier
26427432
Citation
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.
Results Reference
background

Learn more about this trial

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

We'll reach out to this number within 24 hrs