RecruitingPhase 1NCT07536061
Neurological
Huntington Disease
Also known as Huntington chorea, Huntington's disease, HD
Huntington disease is caused by an expanded CAG repeat in the HTT gene. The resulting mutant huntingtin protein is toxic to neurons, particularly in the striatum and cortex.
ORPHA:399 ↗Gene HTT (CAG repeat expansion)Prevalence 1-9 per 100,000 (Orphanet)Onset Adolescent, Adult, ChildhoodGenetic (autosomal dominant)
40
studies recruiting now
as of 7 Sept 2026
301
studies registered in total
as of 7 Sept 2026
29
countries with a recruiting site
as of 7 Sept 2026
17 Apr 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 2 / Phase 3NCT07378644
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
RecruitingPhase 3NCT07326709
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
RecruitingNCT06782230
ScATtEred Rare Disease Biobanks: a Model of Sample/Data Collection With susTainablE and Shared Criteria
RecruitingPhase 1 / Phase 2NCT05822908
A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 40 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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Registry: ENROLL-HD Global Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.
About Huntington Disease
Huntington disease is caused by an expanded CAG repeat in the HTT gene. The resulting mutant huntingtin protein is toxic to neurons, particularly in the striatum and cortex. It causes progressive motor dysfunction (chorea), cognitive decline, and psychiatric symptoms typically beginning in midlife. Children of a carrier have a 50% chance of inheriting the disease. HD has no disease-modifying treatment yet, making clinical trials critically important.
Common clinical features
ChoreaMental deteriorationMemory impairmentHyperreflexiaHypokinesiaGeneralized muscle weaknessClumsinessWeight loss
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
6 approved treatments and 47 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: Tetrabenazine (Nitoman)Approved: Lisdexamfetamine Dimesylate (Elvanse)Approved: Deutetrabenazine (Austedo)Approved: Atomoxetine Hydrochloride (Atomoxetine hydrochloride)Approved: Guanfacine Hydrochloride (Akfen)Approved: Methylphenidate Hydrochloride (Adhansia xr)
Phase 3Valbenazine
Phase 3Riluzole (Exservan)
Phase 3Metformin
Phase 3Creatine
Phase 3Dalzanemdor
Phase 3Pridopidine (Huntexil)
Phase 3Ubidecarenone
Phase 3Tominersen
+ 39 more in development
Before you apply
Things trial teams commonly ask about for Huntington Disease. Not eligibility rules; those are set by each study.
- CAG repeat length (typically 36+ repeats to be affected) is a required genetic confirmation for most trials
- Pre-manifest HD trials enroll gene-positive individuals before symptoms appear - high value if you test positive
- Total functional capacity (TFC) and UHDRS score are the standard disease stage measurements in trials
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).