Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07326709 | A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease | PHASE3 | RECRUITING | 770 | — | — | Mar 24, 2026 | Apr 30, 2030 | Jun 8, 2026 | 20 | United States, Canada +4 |
| NCT06254482 | An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD) | PHASE2 | ACTIVE NOT_RECRUITING | 144 | — | — | Aug 25, 2023 | Jul 16, 2029 | Nov 21, 2025 | 24 | Australia, Austria +8 |
The Composite Unified Huntingtons Disease Ratings Scale (cUHDRS) is an improved composite measure to assess multi-domain clinical progression in HD. The cUHDRS is a combined weighted score of measures of motor function (TMS), cognition (SDMT and SWRT) and overall functional capacity (TFC): cUHDRS = \[(TFC-10.4)/1.9 - (TMS-29.7)/14.9 + (SDMT-28.4)/11.3 + (SWRT - 66.1)/20.1\] + 10. In conjunction with one another, these measures provide a comprehensive, sensitive and specific tool for monitoring disease progression, with lower scores indicating more severe disease. cUHDRS score range from -8 to 25.
| Arm | Type | Description |
|---|---|---|
| Votoplam | EXPERIMENTAL | Votoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo) |
| Placebo | PLACEBO_COMPARATOR | Placebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo) |
| Votoplam 5 mg | EXPERIMENTAL | Participants will receive Votoplam 5 mg tablets once daily orally for 48 months. |
| Votoplam 10 mg | EXPERIMENTAL | Participants will receive votoplam 10 mg tablets once daily orally for 48 months. |
| Name | Type | Description |
|---|---|---|
| Votoplam (blinded) | DRUG | Votoplam (blinded) active treatment |
| Placebo | DRUG | Placebo |
| Votoplam | DRUG | Votoplam will be administered per dose and schedule specified in the arm. |
Inclusion Criteria: * Signed informed consents must be obtained prior to participation in the study * Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature * Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) ...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novartis AG Sponsored ADR | NVS | 2 | PHASE3 | Votoplam |
| Neurocrine Biosciences, Inc. | NBIX | 2 | PHASE3 | Valbenazine |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | intra-striatal rAAV5-miHTT |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |