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

NCT ID: NCT06254482

Last Updated: 2025-11-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

144 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-25

Study Completion Date

2029-07-16

Brief Summary

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

Detailed Description

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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).

Conditions

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Huntington Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Votoplam 5 mg

Participants will receive Votoplam 5 mg tablets once daily orally for 48 months.

Group Type EXPERIMENTAL

Votoplam

Intervention Type DRUG

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

Votoplam 10 mg

Participants will receive votoplam 10 mg tablets once daily orally for 48 months.

Group Type EXPERIMENTAL

Votoplam

Intervention Type DRUG

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

Interventions

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Votoplam

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

Intervention Type DRUG

Other Intervention Names

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HTT227 / PTC518

Eligibility Criteria

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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.
Minimum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Novartis Investigative Site

Clayton, , Australia

Site Status

Novartis Investigative Site

Westmead, , Australia

Site Status

Novartis Investigative Site

Innsbruck, , Austria

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Novartis Investigative Site

Ottawa, , Canada

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Novartis Investigative Site

Angers, , France

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Novartis Investigative Site

Marseille, , France

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Novartis Investigative Site

Paris, , France

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Novartis Investigative Site

Berlin, , Germany

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Novartis Investigative Site

Bochum, , Germany

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Novartis Investigative Site

Münster, , Germany

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Novartis Investigative Site

Ulm, , Germany

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Novartis Investigative Site

Bologna, , Italy

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Novartis Investigative Site

Milan, , Italy

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Novartis Investigative Site

San Giovanni Rotondo, , Italy

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Novartis Investigative Site

Leiden, , Netherlands

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Novartis Investigative Site

Christchurch, , New Zealand

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Novartis Investigative Site

Barakaldo, , Spain

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Novartis Investigative Site

Barcelona, , Spain

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Burgos, , Spain

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Madrid, , Spain

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Birmingham, , United Kingdom

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Novartis Investigative Site

Cardiff, , United Kingdom

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Novartis Investigative Site

London, , United Kingdom

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Novartis Investigative Site

Manchester, , United Kingdom

Site Status

Countries

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Australia Austria Canada France Germany Italy Netherlands New Zealand Spain United Kingdom

Other Identifiers

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2023-504628-24-00

Identifier Type: OTHER

Identifier Source: secondary_id

CHTT227A12201

Identifier Type: OTHER

Identifier Source: secondary_id

PTC518-CNS-004-HD

Identifier Type: -

Identifier Source: org_study_id

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