NCT07326709 · Novartis Pharmaceuticals
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
(INVEST-HD)
What this study is about
The purpose is to assess safety and how well patients handle the treatment of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control treatment group$1. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
View original scientific 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. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- La Jolla, California
- Englewood, Colorado
- Washington D.C., District of Columbia
- Chicago, Illinois
- Fairway, Kansas
- Albany, New York
- Buffalo, New York
- New York, New York
- Cleveland, Ohio
- Philadelphia, Pennsylvania
- Memphis, Tennessee
- Georgetown, Texas
And 2 more locations — see the full list below.
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 23, 2026 · Source of record for eligibility and locations