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

Novartis

Status and phase: Active, not recruiting; Phase 2

Conditions: Huntington Disease

Treatments: Drug: Votoplam

Study type: Interventional

Funder types: Industry

Identifiers

NCT06254482

PTC518-CNS-004-HD

2023-504628-24-00 (Other Identifier)

CHTT227A12201 (Other Identifier)

Details and patient eligibility

About

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

Full description

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).

Enrollment: 144 patients

Sex: All

Ages: 25+ years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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144 participants in 2 patient groups

Votoplam 5 mg

Experimental group

Description:

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

Treatment:

Drug: Votoplam

Votoplam 10 mg

Experimental group

Description:

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

Treatment:

Drug: Votoplam