PTC518-CNS-004-HD
Research type
Research Study
Full title
A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington’s Disease
IRAS ID
1007999
Contact name
Allison Fortenberry
Contact email
Sponsor organisation
PTC Therapeutics, Inc.
Eudract number
2022-001565-12
Research summary
PTC518 is being studied to determine if it can slow the progression of Huntington’s Disease (HD) and its associated symptoms by reducing the production of an abnormal version of a protein called huntingtin protein (sometimes called a mutant version or mHTT).
The PTC518-CNS-002-HD (PIVOT HD) study was developed to evaluate the safety and efficacy of PTC518 in participants with HD. The present study is designed to serve as an extension study to further evaluate the long-term effects of PTC518 in participants with HD.
The study drug PTC518 is an investigational drug being developed for the treatment of HD PTC Therapeutics, Inc., is developing PTC518 and is sponsoring this study.
Up to 250 participants will take part in this study across approximately 30 study sites in North America, Europe, United Kingdom and Oceania.
All participants will receive active PTC518 in this extension study. Participants who received PTC518 in the PIVOT HD study will continue at the same dose level they received in that study (5, 10, or 20 mg).
Participants who received placebo in the PIVOT HD study will be randomly assigned (by chance, like flipping a coin) to receive either 5, 10, or 20 mg PTC518. A computer program will place the participant in one of the dose groups in a process called “randomization.” The participant will have a 1 in 3 chance of being placed into each dose group.
The study will last approximately 4 years. The participation will last up to 30 months from the start of the extension study through the final safety follow-up visit. The study treatment period will last approximately 24 months.REC name
London - Brighton & Sussex Research Ethics Committee
REC reference
23/LO/0476
Date of REC Opinion
21 Sep 2023
REC opinion
Further Information Favourable Opinion