Last Update: Aug 11, 2026 A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease ClinicalTrials.gov Identifier: [NCT07326709](https://clinicaltrials.gov/ct2/show/NCT07326709) Novartis Reference Number:CHTT227A12301 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study Description 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. This study will have a variable double-blind treatment duration of up to 36 months. As part of the study design, not every participant will complete 36 months of treatment. The study consists of 3 periods: \* Screening Period: A period of up to 42-days to assess participants eligibility \* Double-blind Treatment Period: This period will have variable individual treatment duration, up to 36 months. The double-blind treatment period concludes when ≥50% patients complete Month 36. The maximum treatment duration for an individual participant is 36 months. \* Safety Follow-up Period: A period consisting of one safety follow-up visit, conducted on site or by phone call, for all participants not continuing treatment in the separate open-label extension study or discontinuing early. The visit/phone call will take place 30 days after End of Study (EOS) Condition Huntington Disease Phase Phase3 Overall Status Recruiting Number of Participants770 Start Date Mar 24, 2026 Completion Date Apr 30, 2030 Gender All Age(s) 21 Years - 70 Years (Adult, Older Adult) ## Interventions Drug ### Placebo Placebo Drug ### Votoplam (blinded) Votoplam (blinded) active treatment ## Eligibility Criteria 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) 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 Exclusion Criteria: \* History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD \* Serologic evidence for active viral hepatitis as indicated by: \* positive anti-HBc IgM \* positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA \* positive HCV ab test confirmed by positive HCV RNA \* Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result \* History or current diagnosis of ECG or cardiac abnormalities indicating significant risk of safety for participants such as: \* Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker \* History of familial long QT syndrome or known family history of Torsade de Pointes \* Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral salpingectomy at least six weeks before taking study treatment. In the case of oophorectomy alone, the reproductive status of the woman needs to have been confirmed by follow-up hormone level assessment. o WOCBP are excluded unless they are using highly effective methods of contraception (failure rate \\< 1% per year) while taking study treatment and for 8 months after stopping study treatment. \* Pregnant or nursing (breastfeeding) women Other protocol defined inclusion/exclusion criteria may apply Argentina #### Novartis Investigative Site Recruiting CABA,Buenos Aires,C1056abi,Argentina #### Novartis Investigative Site Recruiting CABA,Buenos Aires,1425,Argentina Australia #### Novartis Investigative Site Recruiting Caulfield South,Victoria,3162,Australia Belgium #### Novartis Investigative Site Recruiting Leuven,Vlaams Brabant,3000,Belgium Canada #### CUSM Montreal Neurological Institute Recruiting Montreal,Quebec,H3a 2b4,Canada ###### Anne-Louise Lafontaine ###### Yara Nasr Email: #### North York General Hospital Recruiting North York,Ontario,M2k1e1,Canada ###### Ragini Srinivasan, MD ###### Clare Gibbons Phone: [+416 756 6788]() Email: #### Centre de recherche du CHUM Recruiting Montreal,Quebec,H2w 1t8,Canada ###### Martine Comeau Phone: [+1514 890 8000]() Email: ###### Alby Richard China #### Novartis Investigative Site Recruiting Changsha,Hunan,410011,China #### Novartis Investigative Site Recruiting Wuhan,Hubei,430030,China #### Novartis Investigative Site Recruiting Hangzhou,Zhejiang,310002,China #### West China Hospital of Sichuan Uni Recruiting Chengdu,Sichuan,610041,China ###### Huifang Shang France #### Novartis Investigative Site Recruiting Montpellier,34090,France Israel #### Tel Aviv Sourasky Med Ctr Ichilov Recruiting Tel Aviv,6423906,Israel ###### Tanya Gurevich ###### Tanya Gurevich Email: Japan #### Novartis Investigative Site Recruiting Bunkyo Ku,Tokyo,113-8655,Japan South Korea #### Seoul National University Hospital Recruiting Seoul,Seoul,03080,South Korea ###### Seonmin Park Phone: [82 2 2072 2193]() Email: #### Novartis Investigative Site Recruiting Daejeon,Korea,35015,South Korea #### SMG SNU Boramae Medical Center Recruiting Dongjak Gu,Seoul,07061,South Korea ###### Yun Won Choi Phone: [02-870-3696](tel:02-870-3696) Email: <0428crc@naver.com> #### Inje University Busan Paik Hospital Recruiting Busan,47392,South Korea ###### Min A Son Phone: [+82-51-890-5781](tel:+82-51-890-5781) Email: Spain #### Novartis Investigative Site Recruiting Seville,41009,Spain Switzerland #### Novartis Investigative Site Recruiting Muri bei Bern,3073,Switzerland #### Novartis Investigative Site Recruiting Basel,4031,Switzerland Taiwan #### Novartis Investigative Site Recruiting Taipei,11217,Taiwan #### Novartis Investigative Site Recruiting Changhua,50006,Taiwan United Kingdom #### Novartis Investigative Site Recruiting Exeter,Devon,Ex2 5dw,United Kingdom #### Novartis Investigative Site Recruiting Oxford,Ox3 9du,United Kingdom #### Novartis Investigative Site Recruiting Glasgow,Eh9 1lf,United Kingdom #### University College Hospital Recruiting London,Nw1 2bu,United Kingdom ###### Sarah Tabrizi ###### Kate Fayer Phone: [02031087483](tel:02031087483) Email: #### Novartis Investigative Site Recruiting Cambridge,Cb2 0qq,United Kingdom #### Southampton General Hospital Recruiting Southampton,So16 6yd,United Kingdom ###### Giorgia Cortorillo Phone: [02381206132](tel:02381206132) Email: ###### Christopher Kipps #### Novartis Investigative Site Recruiting Cardiff,Cf14 4xw,United Kingdom #### Novartis Investigative Site Recruiting Aberdeen,Grampian Region,Ab25 2zn,United Kingdom United States #### Uni Of Wisconsin Hosp And Clinic Recruiting Madison,Wisconsin,53705-3611,United States ###### Kathleen Shannon ###### Amy Nagel Phone: [+1 608 263 4505]() Email: #### The University of Vermont MC Recruiting Burlington,Vermont,05401,United States ###### James Thomas Boyd ###### Nasim Nehzat Phone: [+1 802 847 4256]() Email: #### University Of Pittsburgh Recruiting Pittsburgh,Pennsylvania,15213,United States ###### Valerie Suski ###### Patty Conlon Phone: [+1 412 648 0776]() Email: #### Duke University Health System Recruiting Durham,North Carolina,27710,United States ###### Lisa Gauger Email: ###### Burton Scott #### University Of NC At Chapel Hill Recruiting Chapel Hill,North Carolina,27599-9500,United States ###### Claudia Testa ###### Alexandra Seemungal Email: [Alexandra\_Seemungal@med.unc.edu](mailto:Alexandra_Seemungal@med.unc.edu) #### Henry Ford Health System Recruiting Detroit,Michigan,48202,United States ###### Bisena Bulica ###### Blagica Veselinova Email: #### Stanford Healthcare Recruiting Stanford,California,94305-5152,United States ###### Hengameh Zahed Kargaran ###### Jessica Yankulova Phone: [+1 650 723 6469]() Email: #### U Conn Health Recruiting Farmington,Connecticut,06030,United States ###### Sumith Abraham Varghese Phone: [+1 860 679 3265]() Email: ###### Shakaib Khan #### Indiana University Health Recruiting Indianapolis,Indiana,46202,United States ###### Jill Buck Email: ###### Christopher James #### UC Davis Health System Recruiting Sacramento,California,95817,United States ###### Alexandra Duffy ###### Isabella Knott Email: #### University of Washington Medical Center Recruiting Seattle,Washington,98195,United States ###### Debra Del Castillo Phone: [+1 206 616 7117]() Email: ###### Suman Jayadev #### Parkinsons-Movement Disorder Ctr Recruiting Chicago,Illinois,60611,United States ###### Zsa Zsa Brown Phone: [+1 312 503 5706]() Email: ###### Danny Bega #### Cleveland Clinic Foundation Recruiting Cleveland,Ohio,44195,United States ###### Margaret Andorf Phone: [+1 216 442 5232]() Email: ###### Adam Margolius #### TX Movement Disorder Spec PLLC Recruiting Georgetown,Texas,78628,United States ###### Wendy White Email: ###### Michael Soileau #### University of Pennsylvania Recruiting Philadelphia,Pennsylvania,19107,United States ###### Gwenivere Toti Email: ###### Aaron Lasker #### University of Kansas Hospital Recruiting Fairway,Kansas,66205,United States ###### Cameron Zoraghchi Email: ###### Richard Dubinsky #### U of TX Health Science Ct Recruiting Houston,Texas,77030,United States ###### Erin Furr Stimming ###### Dane Weinig Phone: [+1 832 325 7080]() Email: #### Albany Medical College Recruiting Albany,New York,12208,United States ###### Era Kaur Hanspal ###### Alicia Leader Phone: [+1 518-262-5938]() Email: #### UBMD Neurology Recruiting Buffalo,New York,14202,United States ###### Sonia Joseph Phone: [+1 716 932 6080]() Email: ###### Kelly Andrzejewski #### CenExcel Rocky Mtn Clin Research Recruiting Englewood,Colorado,80113,United States ###### Meagen Salinas ###### Liza Heap Phone: [+1 303 762 6674]() Email: #### Georgetown University Recruiting Washington D.C.,District of Columbia,20007,United States ###### Robin Kuprewicz Phone: [(202) 687-0100]() Email: ###### Karen Anderson #### University Of South Florida Recruiting Tampa,Florida,33612,United States ###### Theresa Zesiewicz ###### Ruth Cajuste Phone: [+1 813 974 6378]() Email: #### Vanderbilt University Medical CenterX Recruiting Nashville,Tennessee,37221,United States ###### Amy Brown ###### Elisa Martinez Cancino Phone: [+1 615 878 7403]() Email: #### Universi of Rochester School of Med Recruiting Rochester,New York,14609,United States ###### Hannah Mesmer Phone: [+1 585 275 6412]() Email: [hannah\_mesmer@urmc.rochester.edu](mailto:hannah_mesmer@urmc.rochester.edu) ###### Frederick Marshall #### Trinity Health Michigan Recruiting Grand Rapids,Michigan,49503,United States ###### Melissa Brown Culbertson Email: ###### Danette C Taylor #### University of Iowa Health Care Recruiting Iowa City,Iowa,52242-1091,United States ###### Harika Anedal Kekinagath Phone: [+1 319 335 7555]() Email: ###### Annie Killoran #### University of CA San Diego Recruiting La Jolla,California,92037,United States ###### Krisha Bagga Phone: [+1 858 249 0574]() Email: ###### Jody Corey-Bloom #### Veracity Neuroscience LLC Recruiting Memphis,Tennessee,38157,United States ###### Mark Steven LeDoux ###### Faye Ward Phone: [+1 901 237 3561]() Email: #### Columbia University Irving Medical Recruiting New York,New York,10032,United States ###### Sandra Rizer Phone: [+1 212 305 1718]() Email: ###### Karen Marder ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: