Votoplam for adults with early symptomatic Huntington's disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3, randomized, double-blind, placebo-controlled trial tests votoplam (HTT227) in participants with early symptomatic Huntington's disease with a variable individual treatment duration of up to 36 months. After a screening period of up to 42 days, eligible participants meeting genetic and clinical criteria (including CAG repeat ≥40 and specified UHDRS and CAP100 scores) are randomized to blinded votoplam or placebo. The double-blind period concludes when at least 50% of participants reach month 36, and participants not entering an open-label extension have a single safety follow-up about 30 days after end of study. The main aims are to characterize safety and tolerability and to see if votoplam slows disease progression compared to placebo.

Why it matters

Who can join: the full rules

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

Where it's running

• University of Alabama Birmingham
  Birmingham, Alabama, United States Recruiting
• Univ of Arkansas for Med Sciences
  Little Rock, Arkansas, United States Recruiting
• University of CA San Diego
  La Jolla, California, United States Recruiting
• UC Davis Health System
  Sacramento, California, United States Recruiting
• UCSF
  San Francisco, California, United States Recruiting
• Stanford Healthcare
  Stanford, California, United States Recruiting

+67 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.