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

RecruitingPhase 3

Sponsor: Novartis Pharmaceuticals

Conditions: Huntington Disease

Interventions: Votoplam (blinded), Placebo

Countries: United States, Argentina, Canada, China, Israel, South Korea, Switzerland, Taiwan

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 overview

Sex: ALL

Age: 21 Years to 70 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (41)

La Jolla, California, United States
Englewood, Colorado, United States
Washington D.C., District of Columbia, United States
Tampa, Florida, United States
Chicago, Illinois, United States
Indianapolis, Indiana, United States
Iowa City, Iowa, United States
Fairway, Kansas, United States
Grand Rapids, Michigan, United States
Albany, New York, United States
Buffalo, New York, United States
New York, New York, United States
Rochester, New York, United States
Cleveland, Ohio, United States
Philadelphia, Pennsylvania, United States
Memphis, Tennessee, United States
Nashville, Tennessee, United States
Georgetown, Texas, United States
Houston, Texas, United States
Burlington, Vermont, United States
Seattle, Washington, United States
CABA, Buenos Aires, Argentina
North York, Ontario, Canada
Montreal, Quebec, Canada
Montreal, Quebec, Canada
Chengdu, Sichuan, China
Tel Aviv, Israel
Dongjak Gu, Seoul, South Korea
Seoul, Seoul, South Korea
Busan, South Korea
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