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

RecruitingPhase 3

A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

Who can join

Ages 21 to 70 · All sexes

Full 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

About the study

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.

What is being tested

Sponsor: Novartis Pharmaceuticals · Participants: 770 · Started: Mar 24, 2026

Contact the study team

Official record on ClinicalTrials.gov — NCT07326709

Locations in the U.S.

AlabamaUniversity of Alabama Birmingham, Birmingham
ArkansasUniv of Arkansas for Med Sciences, Little Rock
CaliforniaUniversity of CA San Diego, La Jolla
UC Davis Health System, Sacramento
UCSF, San Francisco
Stanford Healthcare, Stanford
ColoradoCenExcel Rocky Mtn Clin Research, Englewood
ConnecticutU Conn Health, Farmington
District of ColumbiaGeorgetown University, Washington D.C.
FloridaUM Department Of Neurology, Miami
University Of South Florida, Tampa
GeorgiaGrady Memorial Hospital and Clinics, Atlanta
IllinoisParkinsons-Movement Disorder Ctr, Chicago
Rush University Medical Center, Chicago
IndianaIndiana University Health, Indianapolis
IowaUniversity of Iowa Health Care, Iowa City
KansasUniversity of Kansas Hospital, Fairway
Hereditary Neurological Disease Centre, Wichita
MarylandJohns Hopkins University, Baltimore
MassachusettsBeth Israel Deaconess Med Ctr, Boston
MichiganHenry Ford Health System, Detroit
Trinity Health Michigan, Grand Rapids
New YorkAlbany Medical College, Albany
UBMD Neurology, Buffalo
Columbia University Irving Medical, New York
Universi of Rochester School of Med, Rochester
North CarolinaUniversity Of NC At Chapel Hill, Chapel Hill
Duke University Health System, Durham
OhioCleveland Clinic Foundation, Cleveland
OregonOregon Health Sciences University, Portland
PennsylvaniaUniversity of Pennsylvania, Philadelphia
University Of Pittsburgh, Pittsburgh
TennesseeVeracity Neuroscience LLC, Memphis
Vanderbilt University Medical CenterX, Nashville
TexasTX Movement Disorder Spec PLLC, Georgetown
U of TX Health Science Ct, Houston
VermontThe University of Vermont MC, Burlington
VirginiaVCU Health Systems MCV, Richmond
WashingtonUniversity of Washington Medical Center, Seattle
WisconsinUni Of Wisconsin Hosp And Clinic, Madison

Conditions

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.