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Home › Conditions › Huntington's Disease

Huntington's Disease

Part of Dementia

9 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for Huntington's Disease

19 trials tracked for Huntington's Disease: 3 in phase 3 or 4 and 2 with published results. The most-cited published study has 171 citations.

19Trials tracked
3Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 4 1 Phase 3 2 Phase 2 9 Phase 1 3 Other / NA 4
  1. Phase 3 A Study of Treatment With Pridopidine (ACR16) in Participants With Huntington's Disease Completed · 171 cited
  2. Phase 3 A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease Completed · 37 cited
  3. Phase 4 Study of Memantine to Treat Huntington's Disease Completed
  4. Phase 2 A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD) Completed
  5. Phase 2 Tolerability, Safety, and Activity of SRX246 in Irritable Subjects With Huntington's Disease Completed
  6. Phase 2 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease Completed
Show 13 more trials
  1. Phase 2 Study in PRE-manifest Huntington's Disease of Coenzyme Q10 (UbiquinonE) Leading to Preventive Trials (PREQUEL) Completed
  2. Phase 2 Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease Completed
  3. Phase 2 A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139) Completed
  4. Phase 2 Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease Completed
  5. Phase 2 A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod Completed
  6. Phase 2 A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease Completed
  7. Phase 1 Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease Completed
  8. Phase 1 A Study To Evaluate The Abuse Potential Of Single Oral Doses Of Dimebon (Latrepirdine) In Healthy Recreational Polydrug Users Completed
  9. Phase 1 A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease Completed
  10. N/A Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease Completed
  11. N/A Impact of Xenazine(Tetrabenazine)on Gait and Functional Activity in Individuals With Huntington's Disease Completed
  12. N/A Electronic-health Application To Measure Outcomes REmotely Clinical Trial Completed
  13. N/A The Effect of Video Game Exercise on Dynamic Balance and Gait in Individuals With Huntington's Disease Completed

Showing the 19 most-cited and recently-updated of 19 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Huntington's Disease: what the trials found

Clinical investigations into RO7234292 (RG6042) demonstrated significant changes in brain structure, specifically showing a mean percentage change in the Ventricular Volume Boundary Shift Integral of 46.09% and Caudate Volume Boundary Shift Integral of 8.64% over 15 months 7. Safety data for this intervention also noted that 100% and 95.7% of participants experienced adverse events across study arms 7, with no reported suicidal ideation or behavior in specific cohorts 14.

PF-02545920 demonstrated a statistically significant reduction in the Total Maximum Chorea (TMC) score of the UHDRS after 26 weeks (p=0.0030), alongside a significant improvement in Clinical Global Impression of Improvement (CGI-I) scores at both 13 and 26 weeks (p=0.0181) 11. However, this same intervention did not produce statistically significant changes in the Total Motor Score (TMS) at day 28 (p=0.68) or in fMRI tasks related to monetary incentive delay 11.

Other interventions evaluated for Huntington's Disease included Memantine 1, ACR16, which showed no significant change in Modified Motor Scores at week 26 (p=0.456) 2, and PF-02545920, which showed a statistically significant difference in the Grip Strength Incentive Motivation Task (p=0.04) 10.

Recent results — preliminary, needs further review

  • SAGE-718: No statistically significant changes were observed in SDMT (p=0.1675), UHDRS Independence Scale (p=0.4872), or Trail Making Test Part B (p=0.4089) 4.

For the clinician treating this condition

  • RO7234292 (RG6042) is associated with measurable changes in ventricular and caudate volumes over a 15-month period 7.
  • PF-02545920 has shown efficacy in reducing Total Maximum Chorea scores and improving Clinical Global Impression of Improvement, though it did not significantly impact total motor scores at the 28-day mark 11.
  • Clinical trials for ACR16 and SAGE-718 did not reach statistical significance for primary motor or cognitive endpoints in the reported periods [2, 4].

AI synthesis of 7 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Prognostic covariate adjustment reduces required sample size by 15-28% in Huntington's Disease trials
Neurology Phase III
Prognostic covariate adjustment reduces required sample size by 15-28% in Huntington's Disease trials New Modeling Improves Data Accuracy for Huntington's Disease Trials
This modeling study evaluated the impact of prognostic covariate adjustment on statistical power in Huntington's Disease trials. The analysi…
New modeling techniques could improve the accuracy of data in Huntington's disease clinical trials, helping researchers identify meaningful …
medRxiv Oct 22, 2026
HMGB1 acts as a progression-associated inflammatory amplifier in Alzheimer's, Parkinson's, and ALS models
Neurology Sys. Review
HMGB1 acts as a progression-associated inflammatory amplifier in Alzheimer's, Parkinson's, and ALS models New research explores a protein linked to several brain diseases
This narrative review explores the role of HMGB1 in various neurodegenerative diseases including Alzheimer's, Parkinson's, and ALS. The auth…
A specific protein called HMGB1 may act as a driver of inflammation in conditions like Alzheimer's and Parkinson's disease.
Frontiers Sep 30, 2026
Mesenchymal stromal cells and MSC-derived extracellular vesicles may reprogram neuroimmune responses in neurodegenerative diseases
Hematology Sys. Review
Mesenchymal stromal cells and MSC-derived extracellular vesicles may reprogram neuroimmune responses in neurodegenerative diseases Stem cells and vesicles show potential for neurodegenerative diseases
This critical review examines the mechanisms and translational barriers of mesenchymal stromal cells (MSCs) and MSC-derived extracellular ve…
New research explores how stem cell products might protect the brain and repair blood-brain barriers in diseases like Parkinson's and Alzhei…
Frontiers Sep 29, 2026
Ginseng components show potential neuroprotective effects in preclinical models of multiple neurodegenerative diseases
Neurology Meta-analysis
Ginseng components show potential neuroprotective effects in preclinical models of multiple neurodegenerative diseases Ginsenosides Show Potential Neuroprotective Effects for Several Brain Diseases
This systematic review synthesizes evidence regarding the role of ginseng and its active components, such as ginsenosides, in models of Alzh…
Research into ginseng components suggests potential neuroprotective benefits for conditions like Alzheimer's and Parkinson's, though results…
Frontiers Aug 20, 2026
Diabetes mellitus acts as a context-dependent modifier of risk and phenotype in neurological disorders
Neurology Sys. Review
Diabetes mellitus acts as a context-dependent modifier of risk and phenotype in neurological disorders Diabetes May Influence Risk and Progress of Neurological Disorders
This narrative review synthesizes evidence regarding the role of diabetes mellitus (DM) as a disease modifier across various neurological co…
New research suggests that diabetes can change how certain neurological conditions progress, potentially opening doors for new treatment opt…
Frontiers Jun 26, 2026
Narrative review of Huntington's disease highlights limited clinical validation and safety concerns
Neurology Sys. Review
Narrative review of Huntington's disease highlights limited clinical validation and safety concerns Huntington’s Disease May Start Outside the Brain, New Research Suggests
This narrative review addresses Huntington's disease but does not report a specific population, sample size, or intervention details. The au…
Huntington’s disease may involve the gut and immune system, opening new paths for treatment and drug delivery.
Frontiers May 7, 2026
FDA Approves Ingrezza Sprinkle (valbenazine) for Tardive Dyskinesia and Chorea Associated with Huntington’s Disease
Neurology FDA Approval
FDA Approves Ingrezza Sprinkle (valbenazine) for Tardive Dyskinesia and Chorea Associated with Huntington’s Disease FDA approves a new sprinkle version of Ingrezza for two movement disorders
The FDA has approved Ingrezza Sprinkle, a VMAT2 inhibitor, for the treatment of adults with tardive dyskinesia and chorea associated with Hu…
FDA approves sprinkle form of Ingrezza to treat involuntary movements in adults with tardive dyskinesia or Huntington's disease chorea.
FDA Apr 10, 2026
Luteolin shows neuroprotective potential in preclinical models for Alzheimer's, Parkinson's, Huntington's, and multiple sclerosis
Neurology Cohort
Luteolin shows neuroprotective potential in preclinical models for Alzheimer's, Parkinson's, Huntington's, and multiple sclerosis Luteolin shows potential benefits in early studies for neurodegenerative diseases
This review of preclinical studies and preliminary clinical observations suggests luteolin may reduce neuroinflammation and improve function…
Luteolin may help slow brain decline in Alzheimer's and Parkinson's by calming inflammation and boosting antioxidant defenses in early studi…
Frontiers Apr 9, 2026
Ketamine shows cognitive effects in animal models but cognitive worsening in human Huntington's disease study
Neurology Meta-analysis
Ketamine shows cognitive effects in animal models but cognitive worsening in human Huntington's disease study Can ketamine help brain injuries? Animal studies say maybe, but human evidence is thin
A systematic review and meta-analysis of 22 studies (21 animal, 1 human) found ketamine showed positive cognitive effects in 93.2% of animal…
Animal studies suggest ketamine might help brain injuries, but the only human trial found it worsened thinking in patients with Huntington's…
Frontiers Apr 2, 2026

Questions about Huntington's Disease

What limitations exist regarding clinical validation for Huntington's Disease treatments?

Clinical validation for Huntington's disease treatments is limited by a lack of biomarkers to measure early disease, reliance on motor symptoms that appear late, and mixed results from drug trials that often fail to slow overall progression.

Full answer →