46 completed, 11 terminated, 8 active not recruiting, 7 recruiting. 76 Phase 2, 18 Phase 1, 7 Phase 3.
Follow Huntington Disease: new trials by email Literature Review of Huntington Disease trials Research Report on Huntington Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Extension of SKY-0515 in Participants With Huntington's Disease SKY-0515 - Huntington's Disease (HD) | Phase 2 / Phase 3 | Enrolling by invitation | Skyhawk Therapeutics, Inc. | 500 | 2026-04-10 | 2026-07-24 |
| Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease hNSC-01 - Huntington Disease | Phase 1 / Phase 2 | Recruiting | Leslie Thompson | 21 | 2026-04-06 | 2026-04-13 |
| Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease | Phase 2 / Phase 3 | Recruiting | Skyhawk Therapeutics, Inc. | 400 | 2026-01-06 | 2026-10-29 |
| Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease SKY-0515, SKY-0515 Placebo - Huntington Disease, Huntingtons Disease | Phase 2 / Phase 3 | Active not recruiting | Skyhawk Therapeutics, Inc. | 120 | 2025-05-27 | 2026-05-01 |
| The NAD-HD Study: A Study to Investigate Efficacy and Safety of Nicotinamide Riboside Compared With Placebo in Huntington's Disease Nicotinamide Riboside (NR), Placebo - Huntington Disease | Phase 2 | Recruiting | Oslo University Hospital | 120 | 2025-03-18 | 2025-03-19 |
| A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease SPK-10001, Placebo Surgery Control - Huntington Disease | Phase 1 / Phase 2 | Recruiting | Hoffmann-La Roche | 53 | 2025-02-21 | 2026-05-05 |
| A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease. ER2001 intravenous injection, Placebo - Huntington Disease | Phase 1 / Phase 2 | Active not recruiting | ExoRNA Bioscience | 27 | 2025-02-11 | 2026-01-14 |
| A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers NAC, Placebo - Huntington Disease | Phase 2 | Recruiting | Western Sydney Local Health District | 160 | 2024-06-01 | 2025-02-28 |
| Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of MBF-015 in Huntington's Disease Patients MBF-015 16 mg oral capsules - Huntington Disease | Phase 2 | Completed | Medibiofarma S.L. | 10 | 2024-02-01 | 2025-03-03 |
| An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD) Votoplam - Huntington Disease | Phase 2 | Active not recruiting | Novartis Pharmaceuticals | 144 | 2023-08-25 | 2025-11-21 |
| Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease Moderate doses of Thiamine y Biotin, High doses of Thiamine y Biotin - Huntington Disease | Phase 2 | Recruiting | Fundación Pública Andaluza para la gestión de la Investigación en Sevilla | 24 | 2023-04-12 | 2025-02-25 |
| A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD VO659 - Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 3 | Phase 1 / Phase 2 | Recruiting | Vico Therapeutics B. V. | 68 | 2023-02-14 | 2026-08-19 |
| GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease Tominersen, Placebo - Huntington Disease | Phase 2 | Active not recruiting | Hoffmann-La Roche | 301 | 2023-02-03 | 2026-08-10 |
| A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease AB-1001 Gene Therapy - Huntington Disease | Phase 1 / Phase 2 | Active not recruiting | AskBio France, SAS, a subsidiary of AskBio Inc. | 5 | 2022-10-12 | 2026-05-14 |
| Efficacy and Safety on SOM3355 in Huntington's Disease Chorea Placebo capsules, SOM3355 200 mg capsules, SOM3355 300 mg capsules - Huntington Chorea | Phase 2 | Completed | SOM Innovation Biotech SA | 139 | 2022-08-02 | 2025-08-28 |
| A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) PTC518, Placebo - Huntington Disease | Phase 2 | Completed | Novartis Pharmaceuticals | 159 | 2022-06-03 | 2026-06-16 |
| 28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease SAGE-718, Placebo - Huntington Disease | Phase 2 | Completed | Supernus Pharmaceuticals, Inc. | 69 | 2022-05-26 | 2025-09-15 |
| A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD) SAGE-718, Placebo - Huntington's Disease | Phase 2 | Completed | Supernus Pharmaceuticals, Inc. | 189 | 2022-01-26 | 2025-09-15 |
| A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease Branaplam, Placebo - Early Manifest Huntington Disease | Phase 2 | Terminated | Novartis Pharmaceuticals | 26 | 2021-12-08 | 2025-05-16 |
| Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease Deutetrabenazine - Huntington Disease | Phase 2 / Phase 3 | Active not recruiting | Vanderbilt University Medical Center | 30 | 2021-11-19 | 2026-08-05 |
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Chorea (11) · Neurodegenerative Diseases (3) · Rare Diseases (3) · Attention Deficit Disorder with Hyperactivity (2) · Behavioral Symptoms (2) · Movement Disorders (2) · Behavior (1) · Brain Diseases (1) · Central Nervous System Diseases (1) · Cognition Disorders (1) · Dyskinesias (1) · Genetic Diseases, Inborn (1) · Lethargy (1) · Machado-Joseph Disease (1)
Basal Ganglia Diseases (76) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (76) · Dementia (76) · Heredodegenerative Disorders, Nervous System (76) · Mental Disorders (76) · Brain Diseases (75) · Central Nervous System Diseases (75) · Cognition Disorders (75) · Dyskinesias (75) · Genetic Diseases, Inborn (75) · Nervous System Diseases (75) · Neurocognitive Disorders (75) · Movement Disorders (74) · Neurodegenerative Diseases (73) · Chorea (65)
Creatine (5) · pridopidine (4) · 2-((4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)phenoxy)methyl)quinoline (2) · Cell- and Tissue-Based Therapy (2) · Minocycline (2) · latrepirdine (2) · tominersen (2) · triheptanoin (2) · 4-phenylbutyric acid (1) · 6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide (1) · Acetylcysteine (1) · Amantadine (1) · Atomoxetine Hydrochloride (1) · BID protein, human (1) · Bupropion (1)
Wave Life Sciences USA, Inc. (5) · Massachusetts General Hospital (4) · Novartis Pharmaceuticals (4) · Hoffmann-La Roche (3) · Pfizer (3) · Prilenia (3) · Skyhawk Therapeutics, Inc. (3) · Azidus Brasil (2) · Charite University, Berlin, Germany (2) · Institut National de la Santé Et de la Recherche Médicale, France (2) · Medivation, Inc. (2) · National Institute of Neurological Disorders and Stroke (NINDS) (2) · SOM Innovation Biotech SA (2) · Supernus Pharmaceuticals, Inc. (2) · Teva Branded Pharmaceutical Products R&D, Inc. (2)
United States (39) · Germany (24) · Canada (23) · United Kingdom (22) · France (19) · Australia (15) · Poland (12) · Spain (12) · Denmark (8) · Italy (8) · Netherlands (8) · Austria (5) · New Zealand (5) · Brazil (3) · Russia (3)
Diana Rosas, MD, MS (3) · Josef Priller, MD (2) · Karl Kieburtz, MD, MPH (2) · Leigh J Beglinger, Ph.D. (2) · Steven M Hersch, MD, PhD (2) · Amy E Brown, MD (1) · Andrew Feigin, MD (1) · Benjamin Hoehn, MD (1) · Blair R. Leavitt, MD,CM,FRCPC (1) · Carolyn Orr (1)
PMID 36463457, PMID 38489195, PMID 29480210, PMID 32250312, PMID 31381524, PMID 36811812, PMID 11130527, PMID 18334537, PMID 20142523, PMID 24375941, PMID 25062857, PMID 25063332, PMID 25467848, PMID 25568297, PMID 25689146, PMID 26444022, PMID 26714284, PMID 27159993, PMID 27296315, PMID 28826192, PMID 30103342, PMID 30563778, PMID 30572891, PMID 30776019, PMID 31059641, PMID 32666599, PMID 33974419, PMID 35567614, PMID 35626701, PMID 35766355, PMID 35941373, PMID 36494329, PMID 38055260, PMID 38058356, PMID 38280392, PMID 38615323, PMID 39027419, PMID 39804569, PMID 40235521, PMID 40770775, PMID 41491108, PMID 42857615
2014 (4), 2009 (2), 2015 (2), 2023 (2), 2018 (1), K23NS055733 (1), P01NS058793 (1), U44NS090616 (1)