Studies and Trials – Huntington’s Disease and other choreas

Ongoing

List of all Trials and Studies to which ERN-RND expert centers contribute
Type of studyAcronymTitleNCT IDOther study IDs
Clinical trialGENERATION HD2A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals With Prodromal and Early Manifest Huntington's DiseaseNCT05686551
Clinical trialA Randomized, Double-blind, Placebo-controlled Study, to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered Single Ascending Doses of ALN-HTT02 in Adult Patients with Huntington’s DiseaseEudra-CT number: 2024-515732-68-00
Clinical trialPIVOT-HDA phase 2A, randomized, placebo-controlled, dose-ranging study to evaluate the safety and efficacy of PTC518 in subjects with Huntington's diseaseNCT05358717
Clinical trialA Phase 2b, Double-Blind, Randomized Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington’s DiseaseEudra-CT number: 2023-504628-24-00; PTC number: PTC18-CNS-004-HD
Clinical trialA phase 1/2a, open-label trial to investigate the safety, tolerability, pharmaco-kinetics and pharmacodynamics of multiple ascending doses of intrathecally administered VO659 in participants with spinocerebellar ataxia types 1, 3 and Huntington’s diseaseNCT05822908
Clinical trialFALCON-HDStudy to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's DiseaseNCT07378644SKY-0515-004-WW; EudraCT number: 2025-522263-14
Observational studyENROLL-HDA Prospective Registry Study in a Global Huntington's Disease CohortNCT01574053
Observational studyHDClarity Multi-site cerebrospinal fluid collection initiative to facilitate therapeutic development for Huntington's diseaseNCT02855476
Observational studyEuropean natural history study of GNAO1-related disorders(not registered)
Observational studyNKX2-1 Registry (NKX2-1-related disorders registry)(not registered)
Observational studyValidation study of the GNAO1 Severity Score(not registered)

Completed / inactive

  • PROOF-HD – A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease (NCT04556656)
  • SELECT-HD – Study of WVE-003 in Patients With Huntington’s Disease (NCT05032196)
  • Vibrant-HD – Evaluate the Safety of Oral LMI070/​Branaplam in Early Manifest Huntington’s Disease (NCT05111249)

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