Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT03761849) involved people with Huntington's disease and tested a medicine called tovorafenib (also referred to as "Tomi") as well as an earlier investigational medicine called RO7234292, compared against placebo (a dummy treatment with no active ingredient). In total, 899 people were enrolled across six groups. Three of those groups — all receiving either RO7234292 or its placebo — did not complete the study as a group (0 completions recorded), while in the tovorafenib and its placebo groups, between 207 and 216 out of roughly 263–264 participants in each group completed the trial. The trial primarily measured changes in a combined Huntington's disease rating scale (called the cUHDRS, which bundles together scores for physical function, movement, and two thinking tasks into a single number) and a separate measure of day-to-day functioning called the Total Functional Capacity (TFC) score. The reported data shows that for the main combined rating scale (cUHDRS), scores changed from the starting point (baseline) as follows: the placebo group in the tovorafenib part of the trial showed a change of −0.630, the tovorafenib every-8-weeks group showed −0.793, and the tovorafenib every-16-weeks group showed −1.173. For context, the trial description notes that a worsening of 1.2 or more on this scale is considered a meaningful decline. For the TFC functioning score (where lower is worse), the reported changes from baseline were −0.883 for the placebo group, −0.921 for the tovorafenib every-16-weeks group, and −1.284 for the tovorafenib every-8-weeks group. Secondary measures — including motor (movement) scores, two thinking/cognitive tests, and a clinician-rated severity scale — were also reported across the groups, with changes in various directions across all groups; the full numbers for each group are recorded on ClinicalTrials.gov. For the clinician severity scale, data was only reported for the tovorafenib and its placebo groups, and was not available for the RO7234292 groups. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 42032152) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Huntington's Disease Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with manifest (confirmed, active) Huntington's Disease, based on a specific scoring system used by doctors (called a DCL score of 4)
- You are largely independent in your daily life, scoring 70 or above on an Independence Scale used to measure how well someone can function on their own
- Your Huntington's Disease has been confirmed through genetic (DNA) testing, with a specific genetic measurement (CAP score) above 400
- A doctor has assessed that you are able to take care of yourself and carry out everyday activities on your own at the start of the trial
Who may not be able to join:
- You have a serious medical condition, an important abnormality in lab test results or vital signs (such as blood pressure or heart rate), or a fear of enclosed spaces (claustrophobia) that the trial doctor believes would make it unsafe for you to participate
- You are pregnant, breastfeeding, or planning to become pregnant during the trial or within 5 months after your last dose of the trial medication
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Clinical Trials, Hoffmann-La Roche
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Change From Baseline in the Composite Unified Huntington's Disease Rating Scale (cUHDRS) Score-Z Score; Change From Baseline in the Total Functional Capacity (TFC) Score
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.