A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

Part of paid clinical trials in Englewood, Colorado.

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) — DRUG
    Votoplam (blinded) active treatment
  • Placebo — DRUG
    Placebo

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: Votoplam
    Votoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)
  • Placebo Comparator: Placebo
    Placebo (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 Pharmaceuticals
    1-888-669-6682
  • Novartis Pharmaceuticals
    +41613241111

Locations (9)

FacilityCityStateZIPSite coordinators
CenExcel Rocky Mtn Clin ResearchEnglewoodColorado80113
Liza Heap
+1 303 357 5455
Meagen Salinas (PRINCIPAL_INVESTIGATOR)
Georgetown UniversityWashington D.C.District of Columbia20007
Robin Kuprewicz
Karen Anderson (PRINCIPAL_INVESTIGATOR)
Albany Medical CollegeAlbanyNew York12208
Alicia Leader
+1 518-262-5938
Era Kaur Hanspal (PRINCIPAL_INVESTIGATOR)
UBMD NeurologyBuffaloNew York14202
Sonia Joseph
+1 716 932 6080
Kelly Andrzejewski (PRINCIPAL_INVESTIGATOR)
Cleveland Clinic FoundationClevelandOhio44195
Margaret Andorf
+1 216 442 5232
Adam Margolius (PRINCIPAL_INVESTIGATOR)
University of PennsylvaniaPhiladelphiaPennsylvania19107
Gwenivere Toti
Aaron Lasker (PRINCIPAL_INVESTIGATOR)
TX Movement Disorder Spec PLLCGeorgetownTexas78628
Michael Soileau (PRINCIPAL_INVESTIGATOR)
U of TX Health Science CtHoustonTexas77030
Dane Weinig
+1 832 325 7080
Erin Furr Stimming (PRINCIPAL_INVESTIGATOR)
University of Washington Medical CenterSeattleWashington98195
Debra Del Castillo
+1 206 616 7117
Suman Jayadev (PRINCIPAL_INVESTIGATOR)

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