Table 1.
Clinical trials for Huntington’s disease (HD) currently active and/or recruiting participants registered at Clinicaltrials.gov (accessed on 2 July 2021).
| Intervention (Mechanism) | CT Identifier | Clinical Trial | Stage | Phase | Allocation | Masking | Population | Period |
|---|---|---|---|---|---|---|---|---|
| Dopaminergic Modulation | ||||||||
| Drug: Tetrabenazine | NCT02509793 | A Pilot Study Assessing Impulsivity in Patients with Huntington’s Disease on Xenazine (Tetrabenazine) | Recruiting | Phase IV | Single Group Assignment | Open Label |
20 | August 2018–July 2023 |
| Drug: Deutetrabenazine | NCT04301726 | Efficacy of Deutetrabenazine to Control Symptoms of Dysphagia Associated with HD | Not yet recruiting |
PhaseI | Randomized | Triple | 48 | September 2020–December 2022 |
| NCT04713982 | Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease | Recruiting | Phase II/III | N/A | Open Label | 30 | July 2021–February 2024 | |
| Drug: Valbenazine | NCT04102579 | Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated with Huntington Disease (KINECT-HD) |
Recruiting | Phase III | Randomized | Quadruple blind | 120 | November 2019–September 2021 |
| Drug: Risperidone | NCT04201834 | Study to assess the safety and benefit of risperidone for the treatment of chorea in Huntington’s disease | Recruiting | Phase II | N/A | Open Label | 12 | August 2020–August 2022 |
| Glutamatergic Modulation | ||||||||
| Drug: Dextromethorphan/ quinidine |
NCT03854019 | Evaluating the Efficacy of Dextromethorphan/Quinidine in Treating Irritability in Huntington’s Disease | Recruiting | Phase III | Randomized | Quadruple blind | 22 | April 2019–December 2021 |
| Synaptic Modulation | ||||||||
| Drug: Neflamapimod | NCT03980938 | Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease | Recruiting | PhaseII | Randomized | Quadruple blind | 16 | July 2019–July 2020 |
| BDNF Levels Modulation | ||||||||
| Drug: Pridopidine | NCT04556656 | Pridopidine’s Outcome on Function in Huntington Disease, PROOF- HD | Recruiting | Phase III | Randomized | Quadruple blind | 480 | October 2020–April 2023 |
| Mitochondrial Function and Biogenesis | ||||||||
| Drug: Fenofibrate | NCT03515213 | Safety and Efficacy of Fenofibrate as a Treatment for Huntington’s Disease | Active, not recruiting | Phase II | Randomized | Triple blind | 20 | April 2017–August 2021 |
| Drug: Triheptanoin oil | NCT02453061 | A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington’s Disease | Active, not recruiting | Phase II | Randomized | Quadruple blind | 100 | June 2015–December 2020 |
| Drug: Metformin | NCT04826692 | Study to Assess the Effect of Metformin, an Activator of AMPK, on Cognitive Measures of Progression in Huntington’s Disease Patients | Not yet recruiting | Phase III | Randomized | Double | 60 | September 2021–August 2024 |
| Aggregate Inhibition | ||||||||
| Drug: Nilotinib | NCT03764215 | Nilotinib in Huntington’s Disease | Recruiting | Phase I | Sequential Assignment | Open Label | 10 | November 2018–November 2020 |
| Stem Cell Therapies | ||||||||
| Biological: Cellavita | NCT02728115 | Safety Evaluation of Cellavita HD Administered Intravenously in Participants with Huntington’s Disease | Active, not recruiting | Phase I | Non-Randomized | Open Label | 6 | October 2017–December 2023 |
| NCT03252535 | Dose-response Evaluation of the Cellavita HD Product in Patients with Huntington’s Disease | Active, not recruiting | Phase II | Randomized | Triple blind | 35 | January 2018–April 2022 | |
| NCT04219241 | Clinical Extension Study for Assessing the Safety and Efficacy of the Intravenous Administration of Cellavita-HD in Huntington’s Disease Patients. | Active, not recruiting | Phase II/III | N/A | Open Label | 35 | January 2020–April 2022 | |
| Genetic Manipulations | ||||||||
| Drug: RO7234292 (RG 6042, IONIS-HTTRx) intrathecal injection | NCT03842969 | An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington’s Disease Patients Who Participated in Prior Roche and Genetech Sponsored Studies | Recruiting | Phase III | Randomized | Open Label | 950 | April 2019–June 2024 |
| NCT04000594 | 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 | Recruiting | Phase I | Non-Randomized | Open Label | 20 | September 2019–December 2021 | |
| Genetic: intra-striatal rAAV5-miHTT | NCT04120493 | Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults with Early Manifest Huntington Disease | Recruiting | Phase I/II | Randomized | Triple blind | 26 | September 2019–May 2026 |
| Genetic: Intraparenchymal rAAV1-(mi)RNA HTT | NCT04885114 | Safety and Tolerability Study With VY-HTT01, in Adults with Early Manifesting Huntington’s Disease | Not yet recruiting | Phase I | Randomized | Open Label | 22 | July 2021–December 2024 |
| Brain Stimulation | ||||||||
| Deep Brain Stimulation | NCT02535884 | Deep Brain Stimulation of the Globus Pallidus (GP) in Huntington’s Disease (HD) | Recruiting | N/A | Randomized | Quadruple blind | 50 | July 2014–December 2022 |
| NCT04244513 | Deep Brain Stimulation Treatment for Chorea in Huntington’s Disease | Recruiting | N/A | Randomized | Quadruple | 40 | February 2020–June 2022 | |
| Non-invasive Brain Stimulation | NCT04429230 | Efficacy of non-invasive brain stimulation via Transcranial pulsed current stimulation (tPCS) in patients of Huntington’s disease | Not yet recruiting | N/A | Randomized | Double | 15 | June 2021–December 2022 |
| Physical Activity | ||||||||
| Behavioral: Physical activity | NCT03344601 | Physical Activity and Exercise Outcomes in Huntington’s Disease (PACE-HD) | Active, not recruiting | N/A | Randomized | Open Label | 116 | February 2018–August 2020 |
| Behavioral: Adapted Physical Activity program | NCT04917133 | Adapted Physical Activity Effect on Abilities and Quality of Life of Huntington Patients and Relatives During Rehab Stay (HUNT’ACTIV) |
Not yet recruiting | N/A | Randomized | Open Label | 32 | June 2021–January 2023 |
| Dietary Supplement | ||||||||
| Dietary Supplement: Melatonin | NCT04421339 | Melatonin for Huntington’s Disease (HD) Gene Carriers with HD Related Sleep Disturbance—a Pilot Study | Recruiting | N/A | Randomized | Double | 20 | June 2020–July 2021 |
| Drug: combined oral thiamine with biotin | NCT04478734 | Trial of the Combined Use of Thiamine and Biotin in Patients with Huntington’s Disease (HUNTIAM) |
Not yet recruiting | Phase II | Randomized | Open Label | 24 | April 2021–August 2022 |
| Antibody Therapy | ||||||||
| Drug: ANX005 | NCT04514367 | An Open Label Study of ANX005 in Subjects With, or at Risk for, Manifest Huntington’s Disease | Recruiting | Phase II | N/A | Open Label | 24 | August 2020–June 2022 |
| Treatment Regimen | ||||||||
| Drugs: Deutetrabenazine, Risperidone, Zoloft and Idebenone (depending on demand and symptom) | NCT04071639 | Symptomatic Therapy for Patients with Huntington’s Disease | Recruiting | Phase I | Non-Randomized | Open Label | 60 | March 2020–December 2024 |