Recruiting

Phase 3

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

Sponsor:

Novartis Pharmaceuticals

Code:

NCT07326709

Conditions

Huntington Disease

Eligibility Criteria

Sex: All

Age: 21 - 70

Healthy Volunteers: Not accepted

Interventions

Votoplam (blinded)

Placebo

Study Details

Brief 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.

Conditions

Huntington Disease

Study ID

NCT07326709

Start date

Mar 24, 2026

Status verified date

Sep, 2026

Completion date

Apr 30, 2030

Anticipated

Primary completion date

Apr 2, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 21 - 70

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

770 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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)

Interventions

Votoplam (blinded)

Votoplam (blinded) active treatment

Placebo

Primary outcome measure

Change from Baseline in cUHDRS score [ Time Frame: Baseline, Month 36 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682 novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

University of Alabama Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Candace Cromer

+1 205 934 8314 candacecromer@uabmc.edu

Principal Investigator:

Marissa Dean

Univ of Arkansas for Med Sciences

Recruiting

Little Rock, Arkansas, United States, 72205

Contacts

Anja Rassmann

+1 501 686 5838 arassmann@uams.edu

Principal Investigator:

Rohit Dhall

University of CA San Diego

Recruiting

La Jolla, California, United States, 92037

Contacts

Krisha Bagga

+1 858 249 0574 kbagga@health.ucsd.edu

Principal Investigator:

Jody Corey-Bloom

UC Davis Health System

Recruiting

Sacramento, California, United States, 95817

Contacts

Isabella Knott

igknott@health.ucdavis.edu

Principal Investigator:

Alexandra Duffy

UCSF

Recruiting

San Francisco, California, United States, 94115

Contacts

Zach Lamson

+1 415 476 2900 zach.lamson@ucsf.edu

Principal Investigator:

Cameron Dietiker

Stanford Healthcare

Recruiting

Stanford, California, United States, 94305-5152

Contacts

Jessica Yankulova

+1 650 723 6469 jessky@stanford.edu

Principal Investigator:

Hengameh Zahed Kargaran

CenExcel Rocky Mtn Clin Research

Recruiting

Englewood, Colorado, United States, 80113

Contacts

Liza Heap

+1 303 762 6674 e.heap@cenexel.com

Principal Investigator:

Meagen Salinas

U Conn Health

Recruiting

Farmington, Connecticut, United States, 06030

Contacts

Sumith Abraham Varghese

+1 860 679 3265 abrahamvarghese@uchc.edu

Principal Investigator:

Shakaib Khan

Georgetown University

Recruiting

Washington D.C., District of Columbia, United States, 20007

Contacts

Robin Kuprewicz

(202) 687-0100 rk1028@georgetown.edu

Principal Investigator:

Karen Anderson

UM Department Of Neurology

Recruiting

Miami, Florida, United States, 33136

Contacts

Wilfredo Rodriguez- Somoza

3052436732 wxr205@med.miami.edu

Principal Investigator:

Henry Moore

University Of South Florida

Recruiting

Tampa, Florida, United States, 33612

Contacts

Ruth Cajuste

+1 813 974 6378 rsc@usf.edu

Principal Investigator:

Theresa Zesiewicz

Grady Memorial Hospital and Clinics

Recruiting

Atlanta, Georgia, United States, 30303

Contacts

Patricia Noren

+1 404 616 7636 pnoren@gmh.edu

Principal Investigator:

Chantale Octavia Branson

Parkinsons-Movement Disorder Ctr

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Zsa Zsa Brown

+1 312 503 5706 zsazsa.brown@northwestern.edu

Principal Investigator:

Danny Bega

Rush University Medical Center

Recruiting

Chicago, Illinois, United States, 60612

Contacts

Tyler Svymbersky

+1 312 942 8011 Tyler_Svymbersky@rush.edu

Principal Investigator:

Deborah Hall

Indiana University Health

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Jill Buck

jilmbuck@iu.edu

Principal Investigator:

Christopher James

University of Iowa Health Care

Recruiting

Iowa City, Iowa, United States, 52242-1091

Contacts

Harika Anedal Kekinagath

+1 319 335 7555 harika-anedalkekinagath@uiowa.edu

Principal Investigator:

Annie Killoran

University of Kansas Hospital

Recruiting

Fairway, Kansas, United States, 66205

Contacts

Cameron Zoraghchi

czoraghchi2@kumc.edu

Principal Investigator:

Richard Dubinsky

Hereditary Neurological Disease Centre

Recruiting

Wichita, Kansas, United States, 67226

Contacts

Gregory Suter

gregory@hndcentre.com

Principal Investigator:

William Mallonee

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287

Contacts

Etta Bernard

+1 412 533 2264 egoldm17@jhmi.edu

Principal Investigator:

Jee Bang

Beth Israel Deaconess Med Ctr

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Caroline Wolff

cwolff@bidmc.harvard.edu

Principal Investigator:

Samuel Arthur Frank

Henry Ford Health System

Recruiting

Detroit, Michigan, United States, 48202

Contacts

Blagica Veselinova

bveseli1@hfhs.org

Principal Investigator:

Bisena Bulica

Trinity Health Michigan

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

Melissa Brown Culbertson

melissa.brown-culbertson@trinity-health.org

Principal Investigator:

Danette C Taylor

Albany Medical College

Recruiting

Albany, New York, United States, 12208

Contacts

Alicia Leader

+1 518-262-5938 downeya@amc.edu

Principal Investigator:

Era Kaur Hanspal

UBMD Neurology

Recruiting

Buffalo, New York, United States, 14202

Contacts

Sonia Joseph

+1 716 932 6080 joseph53@buffalo.edu

Principal Investigator:

Kelly Andrzejewski

Columbia University Irving Medical

Recruiting

New York, New York, United States, 10032

Contacts

Sandra Rizer

+1 212 305 1718 sz2667@cumc.columbia.edu

Principal Investigator:

Karen Marder

Universi of Rochester School of Med

Recruiting

Rochester, New York, United States, 14609

Contacts

Hannah Mesmer

+1 585 275 6412 hannah_mesmer@urmc.rochester.edu

Principal Investigator:

Frederick Marshall

University Of NC At Chapel Hill

Recruiting

Chapel Hill, North Carolina, United States, 27599-9500

Contacts

Alexandra Seemungal

+1 984 974 5790 Alexandra_Seemungal@med.unc.edu

Principal Investigator:

Claudia Testa

Duke University Health System

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Lisa Gauger

lisa.gauger@duke.edu

Principal Investigator:

Burton Scott

Cleveland Clinic Foundation

Recruiting

Cleveland, Ohio, United States, 44195

Contacts

Margaret Andorf

+1 216 442 5232 andorfm@ccf.org

Principal Investigator:

Adam Margolius

Oregon Health Sciences University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Emily Leonard

+1 503 494 5742 leonarde@ohsu.edu

Principal Investigator:

Lauren Talman

University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19107

Contacts

Gwenivere Toti

gwenivere.toti@pennmedicine.upenn.edu

Principal Investigator:

Aaron Lasker

University Of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Contacts

Patty Conlon

+1 412 648 0776 patricia.conlon@pitt.edu

Principal Investigator:

Valerie Suski

Veracity Neuroscience LLC

Recruiting

Memphis, Tennessee, United States, 38157

Contacts

Faye Ward

+1 901 237 3561 faye@veracityneuroscience.com

Principal Investigator:

Mark Steven LeDoux

Vanderbilt University Medical CenterX

Recruiting

Nashville, Tennessee, United States, 37221

Contacts

Elisa Martinez Cancino

+1 615 878 7403 Elisa.cancino@vumc.org

Principal Investigator:

Amy Brown

TX Movement Disorder Spec PLLC

Recruiting

Georgetown, Texas, United States, 78628

Contacts

Wendy White

wendy@txmds.net

Principal Investigator:

Michael Soileau

U of TX Health Science Ct

Recruiting

Houston, Texas, United States, 77030

Contacts

Dane Weinig

+1 832 325 7080 Dane.R.Weinig@uth.tmc.edu

Principal Investigator:

Erin Furr Stimming

The University of Vermont MC

Recruiting

Burlington, Vermont, United States, 05401

Contacts

Nasim Nehzat

+1 802 847 4256 nasim.nehzat@uvmhealth.org

Principal Investigator:

James Thomas Boyd

VCU Health Systems MCV

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Brooke Foley

+1 804 828 9000 brooke.foley@vcuhealth.org

Principal Investigator:

Stephanie Bissonnette

University of Washington Medical Center

Recruiting

Seattle, Washington, United States, 98195

Contacts

Debra Del Castillo

+1 206 616 7117 debradel@uw.edu

Principal Investigator:

Suman Jayadev

Uni Of Wisconsin Hosp And Clinic

Recruiting

Madison, Wisconsin, United States, 53705-3611

Contacts

Amy Nagel

+1 608 263 4505 anagel@neurology.wisc.edu

Principal Investigator:

Kathleen Shannon

North York General Hospital

Recruiting

North York, Ontario, Canada, M2K1E1

Contacts

Clare Gibbons

+416 756 6788 clare.gibbons@nygh.on.ca

Principal Investigator:

Ragini Srinivasan, MD

Centre de recherche du CHUM

Recruiting

Montreal, Quebec, Canada, H2W 1T8

Contacts

Martine Comeau

+1514 890 8000 martine.comeau.chum@ssss.gouv.qc.ca

Principal Investigator:

Alby Richard

CUSM Montreal Neurological Institute

Recruiting

Montreal, Quebec, Canada, H3A 2B4

Contacts

Yara Nasr

yara.nasr@mcgill.ca

Principal Investigator:

Anne-Louise Lafontaine

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Sep 22, 2026

Last verified

Sep, 2026

Keywords

HD
INVEST-HD
HTT227
VOTOPLAM
HUNTINGTON DISEASE
UHDRS
mHTT
NfL

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-05. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-09-22. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.