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

Recruiting now · Phase 3 · Has a placebo group

Conditions studied: Huntington Disease

In brief

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.

Key facts

Study ID

NCT07326709

Run by

Novartis Pharmaceuticals

People needed

770

Starts

2026-03-24

Expected to finish

2030-04-30

Last updated by the study team

2026-07-29

Who can join

Age: 21 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Stanford Healthcare — Stanford, California, United States (enrolling)
• CenExcel Rocky Mtn Clin Research — Englewood, Colorado, United States (enrolling)
• Georgetown University — Washington D.C., District of Columbia, United States (enrolling)
• University Of South Florida — Tampa, Florida, United States (enrolling)
• Parkinsons-Movement Disorder Ctr — Chicago, Illinois, United States (enrolling)
• Indiana University Health — Indianapolis, Indiana, United States (enrolling)
• University of Iowa Health Care — Iowa City, Iowa, United States (enrolling)
• University of Kansas Hospital — Fairway, Kansas, United States (enrolling)
• Henry Ford Health System — Detroit, Michigan, United States (enrolling)
• Trinity Health Michigan — Grand Rapids, Michigan, United States (enrolling)
• Albany Medical College — Albany, New York, United States (enrolling)
• UBMD Neurology — Buffalo, New York, United States (enrolling)
• Columbia University Irving Medical — New York, New York, United States (enrolling)
• Universi of Rochester School of Med — Rochester, New York, United States (enrolling)
• University Of NC At Chapel Hill — Chapel Hill, North Carolina, United States (enrolling)
• Cleveland Clinic Foundation — Cleveland, Ohio, United States (enrolling)
• University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)
• Veracity Neuroscience LLC — Memphis, Tennessee, United States (enrolling)
• Vanderbilt University Medical CenterX — Nashville, Tennessee, United States (enrolling)
• TX Movement Disorder Spec PLLC — Georgetown, Texas, United States (enrolling)
• U of TX Health Science Ct — Houston, Texas, United States (enrolling)
• The University of Vermont MC — Burlington, Vermont, United States (enrolling)
• University of Washington Medical Center — Seattle, Washington, United States (enrolling)
• Uni Of Wisconsin Hosp And Clinic — Madison, Wisconsin, United States (enrolling)
• University of CA San Diego — La Jolla, California, United States (enrolling)

Full record on ClinicalTrials.gov

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