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Taking people in United StatesPhase 3

Tests votoplam in people with early symptomatic Huntington's disease

Compares votoplam with placebo in adults with early Huntington's disease.

Key facts

Who runs it
Novartis Pharmaceuticals
Where
Albany, Atlanta, Baltimore, Birmingham, Boston, Buffalo, Burlington, Chapel Hill, Chicago, Cleveland, Detroit, Durham, Englewood, Fairway, Farmington, Georgetown, Grand Rapids, Houston, Indianapolis, Iowa City, La Jolla, Little Rock, Madison, Memphis, Miami, Nashville, New York, Philadelphia, Pittsburgh, Portland, Richmond, Rochester, Sacramento, San Francisco, Seattle, Stanford, Tampa, Washington D.C., and Wichita, United StatesSee the 40 sitesAlso in 32 other countries, 128 sites
Trial code
Registry title
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Status
Recruiting
Phase
Phase 3
People taking part
770 people, planned
Started
24 Mar 2026
Expected to end
30 Apr 2030
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • Votoplam (blinded)
  • Placebo
Record updated
22 September 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with early symptomatic Huntington's disease, confirmed by a gene test, who can walk on their own.

Age
21 to 70 years old
Sex
Men and women
Disease stage
Early symptomatic Huntington's disease, who can walk (ambulatory). Scores on the UHDRS scales (IS ≥90, TFC = 13, TMS 7-25) and CAP100 ≥ 70 are required.
Earlier treatment
Cannot have had gene therapy, cell transplantation or any other experimental brain surgery for Huntington's disease.
Biomarker or test result
Huntington's disease confirmed by a gene test, with a CAG repeat length of 40 or above, known before screening.

Main reasons for not taking part

  • Active viral hepatitis
  • Immunodeficiency disease, including a positive HIV test
  • Heart rhythm or ECG problems that carry a significant safety risk
  • Women who could become pregnant and are not using highly effective contraception
  • Pregnant or breastfeeding women

The registry criteria, word for word

Show all 9 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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
  • 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
  • Pregnant or nursing (breastfeeding) women; Other protocol defined inclusion/exclusion criteria may apply

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 21 and 70 years old?

Where in United States

Also in 32 other countries, 128 sites

  • Albany Medical CollegeAlbany, United StatesContact: Alicia LeaderTaking people
  • Grady Memorial Hospital and ClinicsAtlanta, United StatesContact: Patricia NorenTaking people
  • Johns Hopkins UniversityBaltimore, United StatesContact: Etta BernardTaking people
  • University of Alabama BirminghamBirmingham, United StatesContact: Candace CromerTaking people
  • Beth Israel Deaconess Med CtrBoston, United StatesContact: Caroline WolffTaking people
  • UBMD NeurologyBuffalo, United StatesContact: Sonia JosephTaking people
  • The University of Vermont MCBurlington, United StatesContact: Nasim NehzatTaking people
  • University Of NC At Chapel HillChapel Hill, United StatesContact: Alexandra SeemungalTaking people
  • Parkinsons-Movement Disorder CtrChicago, United StatesContact: Zsa Zsa BrownTaking people
  • Rush University Medical CenterChicago, United StatesContact: Tyler SvymberskyTaking people
  • Cleveland Clinic FoundationCleveland, United StatesContact: Margaret AndorfTaking people
  • Henry Ford Health SystemDetroit, United StatesContact: Blagica VeselinovaTaking people
  • Duke University Health SystemDurham, United StatesContact: Lisa GaugerTaking people
  • CenExcel Rocky Mtn Clin ResearchEnglewood, United StatesContact: Liza HeapTaking people
  • University of Kansas HospitalFairway, United StatesContact: Cameron ZoraghchiTaking people
  • U Conn HealthFarmington, United StatesContact: Sumith Abraham VargheseTaking people
  • TX Movement Disorder Spec PLLCGeorgetown, United StatesContact: Wendy WhiteTaking people
  • Trinity Health MichiganGrand Rapids, United StatesContact: Melissa Brown CulbertsonTaking people
  • U of TX Health Science CtHouston, United StatesContact: Dane WeinigTaking people
  • Indiana University HealthIndianapolis, United StatesContact: Jill BuckTaking people
  • University of Iowa Health CareIowa City, United StatesContact: Harika Anedal KekinagathTaking people
  • University of CA San DiegoLa Jolla, United StatesContact: Krisha BaggaTaking people
  • Univ of Arkansas for Med SciencesLittle Rock, United StatesContact: Anja RassmannTaking people
  • Uni Of Wisconsin Hosp And ClinicMadison, United StatesContact: Amy NagelTaking people
  • Veracity Neuroscience LLCMemphis, United StatesContact: Faye WardTaking people
  • UM Department Of NeurologyMiami, United StatesContact: Wilfredo Rodriguez- SomozaTaking people
  • Vanderbilt University Medical CenterXNashville, United StatesContact: Elisa Martinez CancinoTaking people
  • Columbia University Irving MedicalNew York, United StatesContact: Sandra RizerTaking people
  • University of PennsylvaniaPhiladelphia, United StatesContact: Gwenivere TotiTaking people
  • University of PittsburghPittsburgh, United StatesContact: Patty ConlonTaking people
  • Oregon Health Sciences UniversityPortland, United StatesContact: Emily LeonardTaking people
  • VCU Health Systems MCVRichmond, United StatesContact: Brooke FoleyTaking people
  • Universi of Rochester School of MedRochester, United StatesContact: Hannah MesmerTaking people
  • UC Davis Health SystemSacramento, United StatesContact: Isabella KnottTaking people
  • UCSFSan Francisco, United StatesContact: Zach LamsonTaking people
  • University of Washington Medical CenterSeattle, United StatesContact: Debra Del CastilloTaking people
  • Stanford HealthcareStanford, United StatesContact: Jessica YankulovaTaking people
  • University of South FloridaTampa, United StatesContact: Ruth CajusteTaking people
  • Georgetown UniversityWashington D.C., United StatesContact: Robin KuprewiczTaking people
  • Hereditary Neurological Disease CentreWichita, United StatesContact: Gregory SuterTaking people

About the trial

What the trial wants to find out

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.

Read full original descriptionShow less

HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.

The 2 groups in the trial

  • VotoplamTreatment being tested

    Votoplam (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)

    Treatment: Votoplam (blinded)

  • PlaceboPlacebo, for comparison

    Placebo (blinded) taken orally, randomized in a 3:2 ratio (Votoplam: Placebo)

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
Official title
A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Plain-language title
Tests votoplam in people with early symptomatic Huntington's diseaseGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Fit questions
Built from NCT07326709, who can take part
Record updated
22 September 2026