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Clinical Trials in the UK / NCT07326709
Recruiting Phase 3

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

NCT07326709 · tracked via the Priya Life Science UK tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2026-03-24
Last updated
2026-08-31

Condition(s) studied

Huntington Disease

Investigational drug(s) / intervention(s)

Votoplam (blinded)Placebo

Votoplam (blinded): Votoplam (blinded) active treatment

Placebo: Placebo

Study 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.

Eligibility

Sex
ALL
Min age
21 Years
Max age
70 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (82)

FacilityCityRegionStatus
University of Alabama Birmingham Birmingham Alabama Recruiting
Univ of Arkansas for Med Sciences Little Rock Arkansas Recruiting
University of CA San Diego La Jolla California Recruiting
UC Davis Health System Sacramento California Recruiting
Stanford Healthcare Stanford California Recruiting
CenExcel Rocky Mtn Clin Research Englewood Colorado Recruiting
U Conn Health Farmington Connecticut Recruiting
Georgetown University Washington D.C. District of Columbia Recruiting
University Of South Florida Tampa Florida Recruiting
Grady Memorial Hospital and Clinics Atlanta Georgia Recruiting
Parkinsons-Movement Disorder Ctr Chicago Illinois Recruiting
Indiana University Health Indianapolis Indiana Recruiting
University of Iowa Health Care Iowa City Iowa Recruiting
University of Kansas Hospital Fairway Kansas Recruiting
Beth Israel Deaconess Med Ctr Boston Massachusetts Recruiting
Henry Ford Health System Detroit Michigan Recruiting
Trinity Health Michigan Grand Rapids Michigan Recruiting
Albany Medical College Albany New York Recruiting
UBMD Neurology Buffalo New York Recruiting
Columbia University Irving Medical New York New York Recruiting
Universi of Rochester School of Med Rochester New York Recruiting
University Of NC At Chapel Hill Chapel Hill North Carolina Recruiting
Duke University Health System Durham North Carolina Recruiting
Cleveland Clinic Foundation Cleveland Ohio Recruiting
University of Pennsylvania Philadelphia Pennsylvania Recruiting
University Of Pittsburgh Pittsburgh Pennsylvania Recruiting
Veracity Neuroscience LLC Memphis Tennessee Recruiting
Vanderbilt University Medical CenterX Nashville Tennessee Recruiting
TX Movement Disorder Spec PLLC Georgetown Texas Recruiting
U of TX Health Science Ct Houston Texas Recruiting
The University of Vermont MC Burlington Vermont Recruiting
VCU Health Systems MCV Richmond Virginia Recruiting
University of Washington Medical Center Seattle Washington Recruiting
Uni Of Wisconsin Hosp And Clinic Madison Wisconsin Recruiting
Novartis Investigative Site CABA Buenos Aires Recruiting
Novartis Investigative Site CABA Buenos Aires Recruiting
Novartis Investigative Site Ciudad de Buenos Aires Recruiting
Novartis Investigative Site Westmead New South Wales Recruiting
Novartis Investigative Site Caulfield South Victoria Recruiting
Novartis Investigative Site Leuven Vlaams Brabant Recruiting

+ 42 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07326709 on ClinicalTrials.gov ↗ ← All trials in the UK