A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Part of paid clinical trials in La Jolla, California.
- Sponsor
- Novartis Pharmaceuticals
- Study ID
- NCT07326709
- Phase
- PHASE3
- Status
- Recruiting
Conditions
Eligibility Criteria
- Sex
- ALL
- Age
- 21 Years - 70 Years
- Healthy Volunteers
- Not accepted
Interventions
- Votoplam (blinded) — DRUGVotoplam (blinded) active treatment
- Placebo — DRUGPlacebo
Study Details
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
Key Dates
- Start date
- Mar 24, 2026
- Status verified
- Jun 2026
- Primary completion
- Apr 2, 2030
- Completion
- Apr 30, 2030
Study Design
- Enrollment
- 770 participants (estimated)
- Allocation
- RANDOMIZED
- Intervention model
- PARALLEL
- Primary purpose
- TREATMENT
Arms
- Experimental: VotoplamVotoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
- Placebo Comparator: PlaceboPlacebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
Primary Outcome Measure
Change from Baseline in cUHDRS score [ Time Frame: Baseline, Month 36 ]
Central Contacts
- Novartis Pharmaceuticals1-888-669-6682
- Novartis Pharmaceuticals+41613241111
Locations (14)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of CA San Diego | La Jolla | California | 92037 | Jody Corey-Bloom (PRINCIPAL_INVESTIGATOR) |
| CenExcel Rocky Mtn Clin Research | Englewood | Colorado | 80113 | Meagen Salinas (PRINCIPAL_INVESTIGATOR) |
| Georgetown University | Washington D.C. | District of Columbia | 20007 | Robin Kuprewicz Karen Anderson (PRINCIPAL_INVESTIGATOR) |
| Parkinsons-Movement Disorder Ctr | Chicago | Illinois | 60611 | Danny Bega (PRINCIPAL_INVESTIGATOR) |
| University of Kansas Hospital | Fairway | Kansas | 66205 | Cameron Zoraghchi Richard Dubinsky (PRINCIPAL_INVESTIGATOR) |
| Albany Medical College | Albany | New York | 12208 | Era Kaur Hanspal (PRINCIPAL_INVESTIGATOR) |
| UBMD Neurology | Buffalo | New York | 14202 | Kelly Andrzejewski (PRINCIPAL_INVESTIGATOR) |
| Columbia University Irving Medical | New York | New York | 10032 | Karen Marder (PRINCIPAL_INVESTIGATOR) |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | Adam Margolius (PRINCIPAL_INVESTIGATOR) |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19107 | Gwenivere Toti Aaron Lasker (PRINCIPAL_INVESTIGATOR) |
| Veracity Neuroscience LLC | Memphis | Tennessee | 38157 | Mark Steven LeDoux (PRINCIPAL_INVESTIGATOR) |
| TX Movement Disorder Spec PLLC | Georgetown | Texas | 78628 | Wendy White Michael Soileau (PRINCIPAL_INVESTIGATOR) |
| U of TX Health Science Ct | Houston | Texas | 77030 | Erin Furr Stimming (PRINCIPAL_INVESTIGATOR) |
| University of Washington Medical Center | Seattle | Washington | 98195 | Suman Jayadev (PRINCIPAL_INVESTIGATOR) |
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