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A Phase 2a, randomized, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of PTC518 in subjects with Huntington's disease

Trial Profile

A Phase 2a, randomized, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of PTC518 in subjects with Huntington's disease

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 13 May 2025

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At a glance

  • Drugs Votoplam (Primary)
  • Indications Huntington's disease
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms PIVOT-HD
  • Sponsors PTC Therapeutics

Most Recent Events

  • 06 May 2025 According to a PTC Therapeutics media release, company plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval
  • 05 May 2025 Primary endpoint has been met. (Change From Baseline in Blood Total Huntingtin Protein (tHTT) at Month 3)
  • 05 May 2025 Results presented in the PTC Therapeutics media release.

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