Study
PIVOTHD: A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Experimental drug
PTC518: Splicing modifier
Route od administration
Oral QD
Dosage/choorts
5mg 10mg
Sponsor
PTC Therapeutics
Inclusion criteria
Key Inclusion Criteria:
- Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 42 to 50, inclusive
- A Unified Huntington's Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100
- A UHDRS Total Functional Capacity (TFC) score of 13
- A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD)
Numbers
N=162
Locations
Australia, Netherlands, UK, France, Germany, Canada
Study duration
12 Months
Expected results
Estimated Primary Completion Date: March 31, 2023
Estimated Study Completion Date: March 31, 2024
Learn more on clinicaltrials.gov
