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Active, Not Recruiting

NCT Number: NCT06254482

An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Disease Attributes Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases

Age range

25 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Clayton, Australia

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.

Exclusion criteria

  • Participants who have not previously completed the Treatment Period in Study PTC518-CNS-002-HD.

Note: Other inclusion and exclusion criteria may apply.

Treatment and study plan

Votoplam

Drug

Votoplam will be administered per dose and schedule specified in the arm.

Other names: HTT227 / PTC518

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: Baseline up to Month 54

  2. Blood Total Huntingtin Protein (tHTT) Levels

    Time frame: Baseline up to Month 52

Secondary outcomes

  1. Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 48

    Time frame: Baseline, Month 48

  2. Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 48

    Time frame: Baseline, Month 48

  3. Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) Levels

    Time frame: Baseline up to Month 48

  4. Blood mHTT Protein Levels

    Time frame: Baseline up to Month 52

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2029
Study completion
2029
First posted
Feb 12, 2024
Registry last updated
Nov 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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