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

NCT06254482 · tracked via the Priya Life Science UK tracker

Sponsor

Novartis Pharmaceuticals

Phase

Phase 2

Started

2023-08-25

Last updated

2025-11-21

Condition(s) studied

Huntington Disease

Investigational drug(s) / intervention(s)

Votoplam

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

Study summary

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

Eligibility

Sex

ALL

Min age

25 Years

Max age

—

Healthy volunteers

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.

Primary outcome measure(s)

Number of Participants With Treatment-emergent Adverse Events (TEAEs) — Baseline up to Month 54
Blood Total Huntingtin Protein (tHTT) Levels — Baseline up to Month 52

Trial sites (24)

Facility	City	Region
Novartis Investigative Site	Clayton	Australia
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
Novartis Investigative Site	Ulm	Germany
Novartis Investigative Site	Bologna	Italy
Novartis Investigative Site	Milan	Italy
Novartis Investigative Site	San Giovanni Rotondo	Italy
Novartis Investigative Site	Leiden	Netherlands
Novartis Investigative Site	Christchurch	New Zealand
Novartis Investigative Site	Barakaldo	Spain
Novartis Investigative Site	Barcelona	Spain
Novartis Investigative Site	Burgos	Spain
Novartis Investigative Site	Madrid	Spain
Novartis Investigative Site	Birmingham	United Kingdom
Novartis Investigative Site	Cardiff	United Kingdom
Novartis Investigative Site	London	United Kingdom
Novartis Investigative Site	Manchester	United Kingdom

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Other trials for the same condition

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GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease Active, not recruiting

Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease Active, not recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06254482 on ClinicalTrials.gov ↗ ← All trials in the UK