A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

Conditions

• Huntington Disease

Eligibility Criteria

Sex

ALL

Age

21 Years - 70 Years

Healthy Volunteers

Not accepted

Interventions

• Votoplam (blinded) — DRUG
  Votoplam (blinded) active treatment
• Placebo — DRUG
  Placebo

Study Details

The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

Key Dates

First listed

Jan 8, 2026

Start date

Mar 24, 2026

Status verified

Sep 2026

Primary completion

Apr 2, 2030

Completion

Apr 30, 2030

Study Design

Enrollment

770 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Votoplam
  Votoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
• Placebo Comparator: Placebo
  Placebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)

Primary Outcome Measure

Change from Baseline in cUHDRS score [ Time Frame: Baseline, Month 36 ]

Central Contacts

• Novartis Pharmaceuticals
  1-888-669-6682
  [email protected]
• Novartis Pharmaceuticals
  +41613241111

Locations (40)

Find similar trials in Birmingham, AL

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