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

Launched by ALKEUS PHARMACEUTICALS, INC. · Jan 22, 2020

Keywords

ClinConnect Summary

The TEASE trial is studying a treatment called ALK-001 for people with Stargardt disease, a genetic condition that affects vision. This study aims to find out how safe ALK-001 is and how it might help slow down the progression of the disease over a period of 24 months. It is open to participants aged 8 and older who have been invited to join. To be eligible, individuals need to have a confirmed diagnosis of Stargardt disease and specific genetic mutations, along with a certain level of vision in at least one eye.

Participants in the study will take ALK-001 and will be monitored closely for any side effects or changes in their vision. Before joining, they must agree to follow the study rules and complete all required visits. It's important to know that women who can become pregnant will need to confirm they are using contraception. This trial is currently active but not recruiting new participants at this time. Overall, the TEASE trial hopes to provide valuable information about the long-term effects of ALK-001 for those living with Stargardt disease.

Gender

ALL

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