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Completed
NCT05358717
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Conditions: Huntington Disease
Sex: All
Ages: 25 Years – N/A
Healthy volunteers: No
Phase: PHASE2
Enrollment: 159
Sponsor: Novartis Pharmaceuticals
Location: University of California, San Diego La Jolla California
Summary
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
Eligibility Criteria
Key Inclusion Criteria:
* Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 40 to 50, inclusive
Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C):
* 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)
Eligibility for HD-ISS Mild Stage 3 Group (Parts D, E, and F):
* A UHDRS Total Functional Capacity (TFC) score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS score of \
Source: ClinicalTrials.gov (NCT05358717). StuddyBuddy aggregates publicly available trial information.
