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
Apply to this trial

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

First listed
Jan 8, 2026
Start date
Mar 24, 2026
Status verified
Jul 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
    [email protected]
  • Novartis Pharmaceuticals
    +41613241111

Locations (25)

FacilityCityStateZIPSite coordinators
University of CA San DiegoLa JollaCalifornia92037
Krisha Bagga
[email protected]
+1 858 249 0574
Jody Corey-Bloom (PRINCIPAL_INVESTIGATOR)
Stanford HealthcareStanfordCalifornia94305 5152
Jessica Yankulova
[email protected]
+1 650 723 6469
Hengameh Zahed Kargaran (PRINCIPAL_INVESTIGATOR)
CenExcel Rocky Mtn Clin ResearchEnglewoodColorado80113
Liza Heap
[email protected]
+1 303 762 6674
Meagen Salinas (PRINCIPAL_INVESTIGATOR)
Georgetown UniversityWashington D.C.District of Columbia20007
Robin Kuprewicz
[email protected]
(202) 687-0100
Karen Anderson (PRINCIPAL_INVESTIGATOR)
University Of South FloridaTampaFlorida33612
Ruth Cajuste
[email protected]
+1 813 974 6378
Theresa Zesiewicz (PRINCIPAL_INVESTIGATOR)
Parkinsons-Movement Disorder CtrChicagoIllinois60611
Zsa Zsa Brown
[email protected]
+1 312 503 5706
Danny Bega (PRINCIPAL_INVESTIGATOR)
Indiana University HealthIndianapolisIndiana46202
Christopher James (PRINCIPAL_INVESTIGATOR)
University of Iowa Health CareIowa CityIowa52242-1091
Harika Anedal Kekinagath
[email protected]
+1 319 335 7555
Annie Killoran (PRINCIPAL_INVESTIGATOR)
University of Kansas HospitalFairwayKansas66205
Cameron Zoraghchi
[email protected]
Richard Dubinsky (PRINCIPAL_INVESTIGATOR)
Henry Ford Health SystemDetroitMichigan48202
Blagica Veselinova
[email protected]
Bisena Bulica (PRINCIPAL_INVESTIGATOR)
Trinity Health MichiganGrand RapidsMichigan49503
Melissa Brown Culbertson
[email protected]
Danette C Taylor (PRINCIPAL_INVESTIGATOR)
Albany Medical CollegeAlbanyNew York12208
Alicia Leader
[email protected]
+1 518-262-5938
Era Kaur Hanspal (PRINCIPAL_INVESTIGATOR)
UBMD NeurologyBuffaloNew York14202
Sonia Joseph
[email protected]
+1 716 932 6080
Kelly Andrzejewski (PRINCIPAL_INVESTIGATOR)
Columbia University Irving MedicalNew YorkNew York10032
Sandra Rizer
[email protected]
+1 212 305 1718
Karen Marder (PRINCIPAL_INVESTIGATOR)
Universi of Rochester School of MedRochesterNew York14609
Hannah Mesmer
[email protected]
+1 585 244 2529
Frederick Marshall (PRINCIPAL_INVESTIGATOR)
University Of NC At Chapel HillChapel HillNorth Carolina27599 9500
Alexandra Seemungal
[email protected]
Claudia Testa (PRINCIPAL_INVESTIGATOR)
Cleveland Clinic FoundationClevelandOhio44195
Margaret Andorf
[email protected]
+1 216 442 5232
Adam Margolius (PRINCIPAL_INVESTIGATOR)
University of PennsylvaniaPhiladelphiaPennsylvania19107
Gwenivere Toti
[email protected]
Aaron Lasker (PRINCIPAL_INVESTIGATOR)
Veracity Neuroscience LLCMemphisTennessee38157
Faye Ward
[email protected]
+1 901 237 3561
Mark Steven LeDoux (PRINCIPAL_INVESTIGATOR)
Vanderbilt University Medical CenterXNashvilleTennessee37221
Hadassah Pegues
[email protected]
+1 615 878 7403
Amy Brown (PRINCIPAL_INVESTIGATOR)
TX Movement Disorder Spec PLLCGeorgetownTexas78628
Michael Soileau (PRINCIPAL_INVESTIGATOR)
U of TX Health Science CtHoustonTexas77030
Dane Weinig
[email protected]
+1 832 325 7080
Erin Furr Stimming (PRINCIPAL_INVESTIGATOR)
The University of Vermont MCBurlingtonVermont05401
Nasim Nehzat
[email protected]
+1 802 847 4256
James Thomas Boyd (PRINCIPAL_INVESTIGATOR)
University of Washington Medical CenterSeattleWashington98195
Debra Del Castillo
[email protected]
+1 206 616 7117
Suman Jayadev (PRINCIPAL_INVESTIGATOR)
Uni Of Wisconsin Hosp And ClinicMadisonWisconsin53705-3611
Amy Nagel
[email protected]
+1 608 263 4505
Kathleen Shannon (PRINCIPAL_INVESTIGATOR)

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By research site
University of CA San Diego· La Jolla, CAStanford Healthcare· Stanford, CACenExcel Rocky Mtn Clin Research· Englewood, COGeorgetown University· Washington D.C., DCUniversity Of South Florida· Tampa, FLParkinsons-Movement Disorder Ctr· Chicago, IL

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