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Also known as Votoplam (blinded)
Votoplam · 2 trials · 1 indication
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 | 1 | PHASE3 | Valbenazine |
| Hoffmann-La Roche | RHHBY | 3 | PHASE2 | Tominersen, RG6496, SPK-10001 |
| Alnylam Pharmaceuticals, Inc | ALNY | 1 | PHASE1 | ALN-HTT02 |
| uniQure N.V. | QURE | 2 | PHASE1 | AMT-130 |
| Sarepta Therapeutics, Inc. | SRPT | 1 | PHASE1 | SRP-1005 |
Votoplam is an investigational small molecule drug being developed by Novartis AG for the treatment of Huntington Disease, a neurological disorder. It is currently in Phase 3 clinical development and has not been approved by the FDA. Votoplam is also referred to in some study materials as Votoplam (blinded).
Votoplam is being studied as a treatment for Huntington Disease, a progressive neurological condition. It is an investigational therapy in Phase 3 development and is not approved for any indication. Its efficacy and safety in Huntington Disease are still being evaluated in controlled clinical trials.
Votoplam is developed by Novartis AG, which trades under the ticker NVS. Novartis is sponsoring the clinical development program for Votoplam in Huntington Disease, including a Phase 3 study and a long-term extension study.
Votoplam is in Phase 3 clinical development for Huntington Disease. It is investigational and has not received FDA approval. The Phase 3 study is currently recruiting, while a separate long-term extension study is active but no longer recruiting participants.
Votoplam is being evaluated in two clinical trials for Huntington Disease. The Phase 3 trial NCT07326709 is recruiting and plans to enroll 770 participants across 27 countries. The Phase 2 extension study NCT06254482 is active but not recruiting and has enrolled 144 participants. Both are randomized, double-blind, and controlled.
Yes, Votoplam (blinded) is an alternative name used for Votoplam. Both names refer to the same investigational small molecule developed by Novartis AG for Huntington Disease. The blinded designation reflects the double-blind design of the clinical trials, in which treatment assignment is masked.