Tests votoplam in people with early symptomatic Huntington's disease
Compares votoplam with placebo in adults with early Huntington's disease.
Plain-language title generated from the registry record
Registry title: A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Key facts
- Where
- Leuven, BelgiumSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 21 and 70 years old?
Source: NCT07326709, who can take part
Where in Belgium
- UZ LeuvenLeuven, BelgiumTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharmaceuticals
- Trial code
- NCT07326709EU 2025-522227-99-00
- Record updated
- 22 September 2026
- Original description
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.Read full original descriptionShow less
A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
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.
Inclusion: 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: 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; 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; Pregnant or nursing (breastfeeding) women; Other protocol defined inclusion/exclusion criteria may apply.