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RecruitingPhase 3

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

Novartis Pharmaceuticals · NCT07326709

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 21–70 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
770
US sites recruiting
40
Study start
March 24, 2026
Record last updated
September 22, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Votoplam (blinded); Placebo

Conditions: Huntington Disease

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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

Recruiting US locations

  • University of Alabama Birmingham

    Birmingham, AL 35233

    Site contact: Candace Cromer · +1 205 934 8314 · candacecromer@uabmc.edu

  • Univ of Arkansas for Med Sciences

    Little Rock, AR 72205

    Site contact: Anja Rassmann · +1 501 686 5838 · arassmann@uams.edu

  • University of CA San Diego

    La Jolla, CA 92037

    Site contact: Krisha Bagga · +1 858 249 0574 · kbagga@health.ucsd.edu

  • UC Davis Health System

    Sacramento, CA 95817

    Site contact: Isabella Knott · igknott@health.ucdavis.edu

  • UCSF

    San Francisco, CA 94115

    Site contact: Zach Lamson · +1 415 476 2900 · zach.lamson@ucsf.edu

  • Stanford Healthcare

    Stanford, CA 94305-5152

    Site contact: Jessica Yankulova · +1 650 723 6469 · jessky@stanford.edu

  • CenExcel Rocky Mtn Clin Research

    Englewood, CO 80113

    Site contact: Liza Heap · +1 303 762 6674 · e.heap@cenexel.com

  • U Conn Health

    Farmington, CT 06030

    Site contact: Sumith Abraham Varghese · +1 860 679 3265 · abrahamvarghese@uchc.edu

  • Georgetown University

    Washington D.C., DC 20007

    Site contact: Robin Kuprewicz · (202) 687-0100 · rk1028@georgetown.edu

  • UM Department Of Neurology

    Miami, FL 33136

    Site contact: Wilfredo Rodriguez- Somoza · 3052436732 · wxr205@med.miami.edu

  • University Of South Florida

    Tampa, FL 33612

    Site contact: Ruth Cajuste · +1 813 974 6378 · rsc@usf.edu

  • Grady Memorial Hospital and Clinics

    Atlanta, GA 30303

    Site contact: Patricia Noren · +1 404 616 7636 · pnoren@gmh.edu

  • Parkinsons-Movement Disorder Ctr

    Chicago, IL 60611

    Site contact: Zsa Zsa Brown · +1 312 503 5706 · zsazsa.brown@northwestern.edu

  • Rush University Medical Center

    Chicago, IL 60612

    Site contact: Tyler Svymbersky · +1 312 942 8011 · Tyler_Svymbersky@rush.edu

  • Indiana University Health

    Indianapolis, IN 46202

    Site contact: Jill Buck · jilmbuck@iu.edu

  • University of Iowa Health Care

    Iowa City, IA 52242-1091

    Site contact: Harika Anedal Kekinagath · +1 319 335 7555 · harika-anedalkekinagath@uiowa.edu

  • University of Kansas Hospital

    Fairway, KS 66205

    Site contact: Cameron Zoraghchi · czoraghchi2@kumc.edu

  • Hereditary Neurological Disease Centre

    Wichita, KS 67226

    Site contact: Gregory Suter · gregory@hndcentre.com

  • Johns Hopkins University

    Baltimore, MD 21287

    Site contact: Etta Bernard · +1 412 533 2264 · egoldm17@jhmi.edu

  • Beth Israel Deaconess Med Ctr

    Boston, MA 02215

    Site contact: Caroline Wolff · cwolff@bidmc.harvard.edu

  • Henry Ford Health System

    Detroit, MI 48202

    Site contact: Blagica Veselinova · bveseli1@hfhs.org

  • Trinity Health Michigan

    Grand Rapids, MI 49503

    Site contact: Melissa Brown Culbertson · melissa.brown-culbertson@trinity-health.org

  • Albany Medical College

    Albany, NY 12208

    Site contact: Alicia Leader · +1 518-262-5938 · downeya@amc.edu

  • UBMD Neurology

    Buffalo, NY 14202

    Site contact: Sonia Joseph · +1 716 932 6080 · joseph53@buffalo.edu

  • Columbia University Irving Medical

    New York, NY 10032

    Site contact: Sandra Rizer · +1 212 305 1718 · sz2667@cumc.columbia.edu

  • Universi of Rochester School of Med

    Rochester, NY 14609

    Site contact: Hannah Mesmer · +1 585 275 6412 · hannah_mesmer@urmc.rochester.edu

  • University Of NC At Chapel Hill

    Chapel Hill, NC 27599-9500

    Site contact: Alexandra Seemungal · +1 984 974 5790 · Alexandra_Seemungal@med.unc.edu

  • Duke University Health System

    Durham, NC 27710

    Site contact: Lisa Gauger · lisa.gauger@duke.edu

  • Cleveland Clinic Foundation

    Cleveland, OH 44195

    Site contact: Margaret Andorf · +1 216 442 5232 · andorfm@ccf.org

  • Oregon Health Sciences University

    Portland, OR 97239

    Site contact: Emily Leonard · +1 503 494 5742 · leonarde@ohsu.edu

  • University of Pennsylvania

    Philadelphia, PA 19107

    Site contact: Gwenivere Toti · gwenivere.toti@pennmedicine.upenn.edu

  • University Of Pittsburgh

    Pittsburgh, PA 15213

    Site contact: Patty Conlon · +1 412 648 0776 · patricia.conlon@pitt.edu

  • Veracity Neuroscience LLC

    Memphis, TN 38157

    Site contact: Faye Ward · +1 901 237 3561 · faye@veracityneuroscience.com

  • Vanderbilt University Medical CenterX

    Nashville, TN 37221

    Site contact: Elisa Martinez Cancino · +1 615 878 7403 · Elisa.cancino@vumc.org

  • TX Movement Disorder Spec PLLC

    Georgetown, TX 78628

    Site contact: Wendy White · wendy@txmds.net

  • U of TX Health Science Ct

    Houston, TX 77030

    Site contact: Dane Weinig · +1 832 325 7080 · Dane.R.Weinig@uth.tmc.edu

  • The University of Vermont MC

    Burlington, VT 05401

    Site contact: Nasim Nehzat · +1 802 847 4256 · nasim.nehzat@uvmhealth.org

  • VCU Health Systems MCV

    Richmond, VA 23298

    Site contact: Brooke Foley · +1 804 828 9000 · brooke.foley@vcuhealth.org

  • University of Washington Medical Center

    Seattle, WA 98195

    Site contact: Debra Del Castillo · +1 206 616 7117 · debradel@uw.edu

  • Uni Of Wisconsin Hosp And Clinic

    Madison, WI 53705-3611

    Site contact: Amy Nagel · +1 608 263 4505 · anagel@neurology.wisc.edu

Study contacts (from the official record)

  • Novartis Pharmaceuticals · 1-888-669-6682 · novartis.email@novartis.com
  • Novartis Pharmaceuticals · +41613241111
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A Study to Investigate the Efficacy, Safety and Tolerability of… (NCT07326709) | Bored Studies