A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Status: Recruiting
Location: See all (81) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 21
Maximum Age: 70
Healthy Volunteers: f
View:
• 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) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening.
• Meets all of the following criteria:
‣ UHDRS IS score ≥90
⁃ UHDRS TFC score = 13
⁃ UHDRS TMS score = 7-25, inclusive
⁃ CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) / 6.49
Locations
United States
Alabama
University of Alabama Birmingham
RECRUITING
Birmingham
Arkansas
Univ of Arkansas for Med Sciences
RECRUITING
Little Rock
California
University of CA San Diego
RECRUITING
La Jolla
UC Davis Health System
RECRUITING
Sacramento
Stanford Healthcare
RECRUITING
Stanford
Colorado
CenExcel Rocky Mtn Clin Research
RECRUITING
Englewood
Connecticut
U Conn Health
RECRUITING
Farmington
Washington, D.c.
Georgetown University
RECRUITING
Washington D.c.
Florida
University Of South Florida
RECRUITING
Tampa
Georgia
Grady Memorial Hospital and Clinics
RECRUITING
Atlanta
Iowa
University of Iowa Health Care
RECRUITING
Iowa City
Illinois
Parkinsons-Movement Disorder Ctr
RECRUITING
Chicago
Indiana
Indiana University Health
RECRUITING
Indianapolis
Kansas
University of Kansas Hospital
RECRUITING
Fairway
Massachusetts
Beth Israel Deaconess Med Ctr
RECRUITING
Boston
Michigan
Henry Ford Health System
RECRUITING
Detroit
Trinity Health Michigan
RECRUITING
Grand Rapids
North Carolina
University Of NC At Chapel Hill
RECRUITING
Chapel Hill
Duke University Health System
RECRUITING
Durham
New York
Albany Medical College
RECRUITING
Albany
UBMD Neurology
RECRUITING
Buffalo
Columbia University Irving Medical
RECRUITING
New York
Universi of Rochester School of Med
RECRUITING
Rochester
Ohio
Cleveland Clinic Foundation
RECRUITING
Cleveland
Pennsylvania
University of Pennsylvania
RECRUITING
Philadelphia
University Of Pittsburgh
RECRUITING
Pittsburgh
Tennessee
Veracity Neuroscience LLC
RECRUITING
Memphis
Vanderbilt University Medical CenterX
RECRUITING
Nashville
Texas
TX Movement Disorder Spec PLLC
RECRUITING
Georgetown
U of TX Health Science Ct
RECRUITING
Houston
Virginia
VCU Health Systems MCV
RECRUITING
Richmond
Vermont
The University of Vermont MC
RECRUITING
Burlington
Washington
University of Washington Medical Center
RECRUITING
Seattle
Wisconsin
Uni Of Wisconsin Hosp And Clinic
RECRUITING
Madison
Other Locations
Argentina
Novartis Investigative Site
RECRUITING
Caba
Novartis Investigative Site
RECRUITING
Caba
Novartis Investigative Site
RECRUITING
Ciudad De
Australia
Novartis Investigative Site
RECRUITING
Caulfield South
Novartis Investigative Site
RECRUITING
Westmead
Belgium
Novartis Investigative Site
RECRUITING
Leuven
Bulgaria
Novartis Investigative Site
RECRUITING
Sofia
Canada
Centre de recherche du CHUM
RECRUITING
Montreal
CUSM Montreal Neurological Institute
RECRUITING
Montreal
North York General Hospital
RECRUITING
North York
China
The Second Xiangya Hospital of Central South University
RECRUITING
Changsha
West China Hospital of Sichuan Uni
RECRUITING
Chengdu
Novartis Investigative Site
RECRUITING
Guangzhou
Novartis Investigative Site
RECRUITING
Guangzhou
The Second Affiliated Hosp Of Zhejiang Univ School Of Medicine
RECRUITING
Hangzhou
Tongji Hos Affiliated Tongji Medical College of Huazhong
RECRUITING
Wuhan
France
Novartis Investigative Site
RECRUITING
Marseille
Novartis Investigative Site
RECRUITING
Montpellier
Germany
Novartis Investigative Site
RECRUITING
Bochum
Hungary
Novartis Investigative Site
RECRUITING
Budapest
Israel
Tel Aviv Sourasky Med Ctr Ichilov
RECRUITING
Tel Aviv
Japan
Novartis Investigative Site
RECRUITING
Bunkyo Ku
Netherlands
Novartis Investigative Site
RECRUITING
Leiden
Republic of Korea
Inje Univesrity Busan Paik Hospital
RECRUITING
Busan
Novartis Investigative Site
RECRUITING
Daejeon
SMG SNU Boramae Medical Center
RECRUITING
Dongjak Gu
Seoul National University Hospital
RECRUITING
Seoul
Romania
Novartis Investigative Site
RECRUITING
Bucharest
Novartis Investigative Site
RECRUITING
Târgu Mureş
Saudi Arabia
Novartis Investigative Site
RECRUITING
Riyadh
Slovakia
Novartis Investigative Site
RECRUITING
Košice
Spain
Novartis Investigative Site
RECRUITING
Barcelona
Novartis Investigative Site
RECRUITING
Barcelona
Novartis Investigative Site
RECRUITING
Seville
Switzerland
Novartis Investigative Site
RECRUITING
Basel
Novartis Investigative Site
RECRUITING
Muri Bei Bern
Taiwan
Novartis Investigative Site
RECRUITING
Changhua
Novartis Investigative Site
RECRUITING
Taipei
United Kingdom
Novartis Investigative Site
RECRUITING
Aberdeen
Novartis Investigative Site
RECRUITING
Cambridge
Novartis Investigative Site
RECRUITING
Cardiff
Novartis Investigative Site
RECRUITING
Exeter
Novartis Investigative Site
RECRUITING
Glasgow
Novartis Investigative Site
RECRUITING
London
University College Hospital
RECRUITING
London
Novartis Investigative Site
RECRUITING
Oxford
Southampton General Hospital
RECRUITING
Southampton
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
1-888-669-6682
Backup
Novartis Pharmaceuticals
+41613241111
Time Frame
Start Date: 2026-03-24
Estimated Completion Date: 2030-04-30
Participants
Target number of participants: 770
Treatments
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)
Related Therapeutic Areas
Sponsors
Leads: Novartis Pharmaceuticals