Disease landscape

Huntington's Disease clinical trials

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63 active trials 37 recruiting 7 Phase 3 51 sponsors 4 changes in 30 days

Data updated daily from ClinicalTrials.gov, last refresh 2026-10-02

What changed in the last 30 days

2026-09-03
notable
NCT07246941 A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of… · Hoffmann-La Roche · Phase 1
2026-09-23
minor
NCT07326709 A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants… · Novartis Pharmaceuticals · Phase 3
2026-09-06
minor
NCT06539169 FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · xCures

Active trials by phase

Early Phase 1
1
Phase 1
4
Phase 1/2
7
Phase 2
5
Phase 2/3
4
Phase 3
3
Not charted: 39 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials top 25 of 63

NCT IDTitleSponsorStatusUpdated
Phase 3
NCT07609108 Pridopidine Phase 3 Study in Huntington's Disease Prilenia Recruiting 2026-09-30
NCT07326709 A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants… Novartis Pharmaceuticals Recruiting 2026-09-22
NCT06312189 Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With… Neurocrine Biosciences Enrolling by Invitation 2026-06-18
Phase 2/3
NCT07378644 Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants… Skyhawk Therapeutics, Inc. Recruiting 2026-09-21
NCT04713982 Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease Vanderbilt University Medical Center Active, Not Recruiting 2026-08-05
NCT07537075 An Extension of SKY-0515 in Participants With Huntington's Disease Skyhawk Therapeutics, Inc. Enrolling by Invitation 2026-07-24
NCT06873334 Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With… Skyhawk Therapeutics, Inc. Active, Not Recruiting 2026-05-01
Phase 2
NCT06853743 The NAD-HD Study: A Study to Investigate Efficacy and Safety of Nicotinamide Riboside… Oslo University Hospital Recruiting 2025-03-19
NCT05509153 A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin… Western Sydney Local Health District Recruiting 2025-02-28
NCT04478734 Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease Fundación Pública Andaluza para la gestión de la Investigación en Sevilla Recruiting 2025-02-25
NCT05686551 GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen… Hoffmann-La Roche Active, Not Recruiting 2026-08-10
NCT06254482 An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in… Novartis Pharmaceuticals Active, Not Recruiting 2025-11-21
Phase 1/2
NCT05822908 A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD Vico Therapeutics B. V. Recruiting 2026-08-19
NCT06826612 A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease Hoffmann-La Roche Recruiting 2026-05-05
NCT07451613 Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease Leslie Thompson Recruiting 2026-04-13
NCT05541627 A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest… AskBio France, SAS, a subsidiary of AskBio Inc. Active, Not Recruiting 2026-05-14
NCT07339514 A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest… ExoRNA Bioscience Active, Not Recruiting 2026-01-14
NCT04120493 Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest… UniQure Biopharma B.V. Active, Not Recruiting 2025-10-21
NCT05243017 Safety and Efficacy of AMT-130 in European Adults With Early Manifest Huntington's Disease UniQure Biopharma B.V. Active, Not Recruiting 2025-03-10
Phase 1
NCT06585449 A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease Alnylam Pharmaceuticals Recruiting 2026-09-16
NCT07536061 A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's… Sarepta Therapeutics, Inc. Recruiting 2026-09-04
NCT07246941 A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of… Hoffmann-La Roche Active, Not Recruiting 2026-09-02
NCT07516899 Mass Balance Study of [14C] LPM3770164 in Healthy Participants Luye Pharma Group Ltd. Not Yet Recruiting 2026-04-08
Early Phase 1
NCT06634628 iMagemHTT-009- FIH Evaluation of Novel Mutant Huntingtin PET Radioligand… CHDI Foundation, Inc. Recruiting 2026-03-13
Not applicable
NCT07513844 Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in… First Affiliated Hospital of Fujian Medical University Recruiting 2026-04-29

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