*CHTT227A12301: Phase 3 study with votoplam in Hungtington's Disease
Research type
Research Study
Full 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
IRAS ID
1013249
Contact name
Fiona Morris
Contact email
Sponsor organisation
Novartis Pharmaceuticals UK Limited
Clinicaltrials.gov Identifier
Research summary
The purpose of this trial is to learn more about the effects of votoplam, also called HTT227, compared to placebo and historical data in people with Huntington’s disease (HD).
HD is a rare condition that is passed down from a parent with a faulty gene to their children, with each child having a 50% chance of inheriting the disease. People with HD make a mutant form of huntingtin (HTT) protein that causes the breakdown of nerve cells that slowly lose their function and die. The loss of nerve cells can affect a person’s movement, ability to think, and their mental health. As the disease gets worse, people may lose the ability to walk, talk, swallow, or take care of themselves and may need full-time support.
Approximately 770 participants aged 21 to 70 years with HD will receive treatment. Participants will be randomly assigned to take votoplam as a tablet by mouth (Group 1) or take placebo as a tablet by mouth (Group 2). Participants are more likely to be placed in Group 1 than in Group 2. Votoplam is being tested for its effects on HD. Placebo looks like the trial drug but does not have any drug in it.
The treatment period will be flexible with a maximum duration of 3 years. None of the participants, trial doctors, or trial staff will know which treatment the participants will receive. Some trials are done this way because knowing what treatment the participants receive can affect the results of the trial. Doing a trial this way helps to make sure that the results are looked at with fairness across all treatments.
Researchers will check participants’ HD and general health throughout the trial and contact the participants 1 month after their last dose of trial treatment for their follow-up health check-up.REC name
London - Westminster Research Ethics Committee
REC reference
26/LO/0024
Date of REC Opinion
3 Mar 2026
REC opinion
Further Information Favourable Opinion