A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington’s Disease

A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington’s Disease

Publication date: Jan 07, 2026

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.

Concepts Keywords
Blind HD
Childbearing HTT227
Salpingectomy HUNTINGTON DISEASE
Viral INVEST-HD
Weeks mHTT
NfL
UHDRS
VOTOPLAM

Semantics

Type Source Name
disease MESH Huntington’s Disease
disease MESH disease progression
disease MESH PTC
drug DRUGBANK Adenine
drug DRUGBANK Guanine
drug DRUGBANK Tilmicosin
disease MESH CAP
drug DRUGBANK Hepatitis B Vaccine (Recombinant)
disease MESH cardiac arrhythmias
disease MESH ventricular tachycardia
disease MESH AV block
disease MESH long QT syndrome
disease MESH Torsade de
pathway KEGG Huntington disease

Original Article

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