Trial Results
A plain-English summary of what published Huntington's disease clinical trial results actually show. It is based only on outcomes reported in the trial registries Fenix tracks — not on news, announcements or opinion.
Results announced — not yet independently verified
These results were announced by the trial sponsor, but have not yet been independently verified by Fenix through a peer-reviewed publication or trial-registry results record. They are therefore not included in the verified results summary below.
AMT-130 (uniQure)
Sponsor announcementNot independently verifiedSponsor-reported topline results
Announced 24 September 2025
uniQure announced topline results from its ongoing gene therapy programme for Huntington's disease, reporting that people who received the higher dose of AMT-130 declined more slowly than an external comparison group after three years. The company also reported that the therapy was generally well tolerated. These figures come from the company itself and have not yet been published in a peer-reviewed journal or posted to a trial registry, so Fenix has not been able to check them independently.
Read the uniQure company announcement
Other studies
A few completed trials showed statistically significant reductions in involuntary movements (chorea) or in the huntingtin protein measured in body fluids. Many other trials reported measurements without p-values or only biomarker changes; those cannot be taken as proof of clinical benefit. Biomarker changes (blood or spinal fluid huntingtin, brain scans) are not the same as improved daily function or slowed disease.
Key results
- Positive findingTreatment for symptoms
Valbenazine reduced involuntary movements compared with placebo as measured by a score for involuntary movements (Unified Huntington's Disease Rating Scale Total Maximal Chorea).
Main measure of the trial
Reported figures: p < 0.0001; average change -4.60 vs -1.44 points; more people were judged improved on Clinical Global Impression of Change (p = 0.0007) and Patient Global Impression of Change (p = 0.0062).
- Positive findingTreatment for symptoms
Deutetrabenazine reduced involuntary movements compared with placebo as measured by the involuntary movements score (Unified Huntington's Disease Rating Scale Total Maximal Chorea).
Main measure of the trial
Reported figures: p < 0.0001; average change -4.42 vs -1.93 points.
- InconclusiveLaboratory measure only
PTC518 lowered the amount of total huntingtin protein measured in blood (geometric mean) at Month 3.
Laboratory measure, not symptoms
Reported figures: p < 0.0001; percent changes reported across groups: -1.40%, -17.36%, -28.67%.
- Mixed findingTreatment for symptoms
SOM3355 reduced involuntary movements in the participants who were not taking neuroleptic medicines, as measured by the involuntary movements score (Unified Huntington's Disease Rating Scale Total Maximal Chorea).
Only in part of the participants
Reported figures: p = 0.045; reported changes -2.19, -2.42, -3.46 points across groups.
- Mixed findingTreatment for symptoms
SAGE-718 did not show a statistically significant difference on its main thinking test (Symbol Digit Modalities Test) at Day 84, but one secondary home-function measure differed.
Additional measure only — main measure not confirmed
Reported figures: Primary test (Symbol Digit Modalities Test) p = 0.1675; change 2.0 vs 0.8 points. A secondary home subdomain score (Huntington's Disease Everyday Functioning Home Subdomain) showed p = 0.0291; changes -4.6 vs -0.7 points.
- Negative findingAims to slow the disease
Pridopidine did not show a statistically significant difference on the trial's main measure of everyday function (Total Functional Capacity) at Week 65.
Main measure of the trial
Reported figures: p = 0.1670; change -1.18 vs -0.95 points.
What a "statistically significant" result means
A statistically significant result only means a measured difference is unlikely to be due to chance. It does not automatically mean the change improves everyday life or slows Huntington's disease.
What the evidence cannot yet tell us
- Many registry entries list numeric changes without p-values or full reports; Fenix cannot judge statistical significance or clinical meaning for those results.
- Changes in biomarkers (blood or spinal fluid huntingtin, brain scans) are not proof that symptoms improved or the disease slowed.
- Trial completion or a reported measurement alone is not evidence that a treatment worked; clinical benefit needs the right outcomes and clear statistical reporting.
View the clinical trial results and sources used to generate this summary.
Based on 109 evidence records · Updated 11 Aug 2026
Last updated: 11 August 2026 at 09:03
This summary is generated from publicly available clinical trial results and is intended to help explain trial results in plain English. It is not medical advice.