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Recruiting NCT07326709

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

Phase III Interventional Huntington Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Votoplam (blinded), Placebo.
Who it may be relevant to
Registry conditions: Huntington Disease. Basic parameters: 21 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Argentina, Australia, Canada, China +6
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Detailed description

This study will have a variable double-blind treatment duration of up to 36 months. As part of the study design, not every participant will complete 36 months of treatment.

The study consists of 3 periods:

* Screening Period: A period of up to 42-days to assess participants eligibility * Double-blind Treatment Period: This period will have variable individual treatment duration, up to 36 months. The double-blind treatment period concludes when ≥50% patients complete Month 36. The maximum treatment duration for an individual participant is 36 months. * Safety Follow-up Period: A period consisting of one safety follow-up visit, conducted on site or by phone call, for all participants not continuing treatment in the separate open-label extension study or discontinuing early. The visit/phone call will take place 30 days after End of Study (EOS)

Interventions

  • Drug Votoplam (blinded)
    Votoplam (blinded) active treatment
  • Drug Placebo
    Placebo

Primary outcome measures

  • Change from Baseline in cUHDRS score [Time frame: Baseline, Month 36]
Secondary outcome measures (9)
  • Change from Baseline in UHDRS-TFC [Time frame: Baseline, Month 36]
  • Change from Baseline in UHDRS-IS [Time frame: Baseline, Month 36]
  • The time to decline in TFC score by at least one or IS score by at least 10 [Time frame: Baseline to end of treatment up to 36 months]
  • Change from Baseline in UHDRS-TMS [Time frame: Baseline, Month 36]
  • Change from Baseline in SDMT [Time frame: Baseline, Month 36]
  • Change from Baseline in SWRT [Time frame: Baseline, Month 36]
  • Percent change from Baseline in blood mHTT protein [Time frame: Baseline to steady state and up to 36 months in blood mHTT protein]
  • Change from Baseline in serum NfL [Time frame: Baseline, Month 36]
  • Incidence and severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and TEAEs leading to participant withdrawal [Time frame: Baseline to up to 36 months]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 25 centers
  • University of CA San Diego — La Jolla
  • Stanford Healthcare — Stanford
  • CenExcel Rocky Mtn Clin Research — Englewood
  • Georgetown University — Washington D.C.
  • University Of South Florida — Tampa
  • Parkinsons-Movement Disorder Ctr — Chicago
  • Indiana University Health — Indianapolis
  • University of Iowa Health Care — Iowa City
  • … and 17 more centers
United Kingdom · 8 centers
  • Novartis Investigative Site — Exeter
  • Novartis Investigative Site — Aberdeen
  • Novartis Investigative Site — Cambridge
  • Novartis Investigative Site — Cardiff
  • Novartis Investigative Site — Glasgow
  • University College Hospital — London
  • Novartis Investigative Site — Oxford
  • Southampton General Hospital — Southampton
Canada · 3 centers
  • North York General Hospital — North York
  • Centre de recherche du CHUM — Montreal
  • CUSM Montreal Neurological Institute — Montreal
South Korea · 3 centers
  • Novartis Investigative Site — Dongjak Gu
  • Novartis Investigative Site — Seoul
  • Novartis Investigative Site — Busan
Argentina · 2 centers
  • Novartis Investigative Site — CABA
  • Novartis Investigative Site — CABA
China · 2 centers
  • West China Hospital of Sichuan Uni — Chengdu
  • Novartis Investigative Site — Hangzhou
France · 2 centers
  • Novartis Investigative Site — Bordeaux
  • Novartis Investigative Site — Montpellier
Switzerland · 2 centers
  • Novartis Investigative Site — Basel
  • Novartis Investigative Site — Muri bei Bern
Taiwan · 2 centers
  • Novartis Investigative Site — Changhua
  • Novartis Investigative Site — Taipei
Australia · 1 center
  • Novartis Investigative Site — Caulfield South
Israel · 1 center
  • Tel Aviv Sourasky Med Ctr Ichilov — Tel Aviv

Identifiers

NCT: NCT07326709 · CHTT227A12301

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗