A Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington’s Disease

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-504628-24-00

Acronym

PTC518-CNS-004-HD

Enrollment

172

Registered

2023-09-04

Start date

2023-10-31

Completion date

Unknown

Last updated

2025-10-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Huntington's Disease

Brief summary

Safety profile as characterized by TEAEs, laboratory abnormalities, vital signs, physical examination, C-SSRS, Evaluate blood tHTT protein levels over time

Detailed description

Change in caudate volume as assessed by vMRI over time, Change in cUHDRS scores over time, Evaluate CSF mHTT protein levels over time, Evaluate blood mHTT protein levels over time

Interventions

DRUGPlacebo to 10 mg tablet

DRUGPTC518

DRUGPlacebo to 5 mg tablet

DRUGPlacebo to 20 mg tablet

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Safety profile as characterized by TEAEs, laboratory abnormalities, vital signs, physical examination, C-SSRS, Evaluate blood tHTT protein levels over time	—

Secondary

Measure	Time frame
Change in caudate volume as assessed by vMRI over time, Change in cUHDRS scores over time, Evaluate CSF mHTT protein levels over time, Evaluate blood mHTT protein levels over time	—

Countries

Austria, France, Germany, Italy, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026