A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Huntington Disease Clinical Trial

Official title: A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease

Status Recruiting

Clinical Trial Summary

The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.

Clinical Trial Description

Participants will first be randomized to Part A or Part B or Parts D or E in a 1:1 randomization ratio, depending on their Huntington's disease Integrated Staging System (HD-ISS) staging criteria and then to active treatment (PTC518 5 mg in Parts A and D and 10 mg in Parts B and E) or matching placebo within each part in a 2:1 ratio of active treatment to placebo. A Drug Safety Monitoring Board (DSMB) Charter will undertake an unblinded review of safety data from the 5 and 10 mg dosing groups and provide a recommendation on when Parts C and F (with a 20 mg active treatment arm) can be initiated. At that time, participants will be randomized to any study Part that is currently open for enrollment, and then to either active treatment or placebo (in a 2:1 ratio) within that Part. ;

Related Conditions & MeSH terms

• Huntington Disease

• NCT number: NCT05358717
• Study type: Interventional
• Source: PTC Therapeutics
• Contact: Patient Advocacy
• Phone: 1-866-562-4620
• Email: medinfo@ptcbio.com
• Status: Recruiting
• Phase: Phase 2
• Start date: April 25, 2022
• Completion date: July 31, 2024