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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. ;


Study Design


Related Conditions & MeSH terms


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

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