NCT05358717

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 159 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 25 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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. Participants who complete this study and agree to participate in a long-term extension (LTE) study, will be enrolled in a separate LTE study PTC518-CNS-004-HD (NCT06254482).

Conditions

• Huntington Disease

Interventions

Type	Name	Description
DRUG	PTC518	PTC518 will be administered per dose and schedule specified in the arm.
DRUG	Placebo	Placebo matching to PTC518 will be administered per schedule specified in the arm.

Timeline

• Start date: 2022-06-03
• Primary completion: 2025-02-07
• Completion: 2025-07-31
• First posted: 2022-05-03
• Last updated: 2026-01-13
• Results posted: 2026-01-13

Locations

28 sites across 11 countries: United States, Australia, Austria, Canada, France, Germany, Italy, Netherlands, New Zealand, Spain, United Kingdom

Regulatory

• FDA-regulated drug study