The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.
Participants who completed the Treatment Period in the parent Phase 2a Study PTC518-CNS-002-HD (NCT05358717), fulfilled the enrollment criteria, and chose to enroll in this extension study will undergo baseline evaluations and be assessed for 54 additional months. All participants will receive active votoplam in this extension study. Participants who received votoplam in the parent Study PTC518-CNS-002-HD will continue at the same dose level they received in that study in a blinded fashion (5, 10 milligrams \[mg\]). Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a votoplam dose level according to the same dosing group in which they were previously randomized (5, 10 mg).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
144
Votoplam will be administered per dose and schedule specified in the arm.
Novartis Investigative Site
Clayton, Australia
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: Baseline up to Month 54
Blood Total Huntingtin Protein (tHTT) Levels
Time frame: Baseline up to Month 52
Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 48
Time frame: Baseline, Month 48
Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 48
Time frame: Baseline, Month 48
Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels
Time frame: Baseline up to Month 48
Blood mHTT Protein Levels
Time frame: Baseline up to Month 52
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Novartis Investigative Site
Westmead, Australia
Novartis Investigative Site
Innsbruck, Austria
Novartis Investigative Site
Ottawa, Canada
Novartis Investigative Site
Angers, France
Novartis Investigative Site
Marseille, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Berlin, Germany
Novartis Investigative Site
Bochum, Germany
Novartis Investigative Site
Münster, Germany
...and 14 more locations