Phase 3
Completed N=499
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
Huntington Disease
Source: ClinicalTrials.gov NCT04556656 ↗
Enrolled (actual)
499
Serious AEs
11.0%
Results posted
Mar 2025
Primary outcomePrimary: Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) — -1.18; -0.95 score on a scale — p=0.1670
◆ Published Evidence
Emerging
10citations · ~10 / year
Pridopidine in early-stage manifest Huntington's disease: a phase 3 trial.
Summary
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
Linked Publications
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Pridopidine in early-stage manifest Huntington's disease: a phase 3 trial.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT) |
-1.18; -0.95 | 0.1670 |
| PRIMARY Change From Baseline to Week 65 in the UHDRS TFC Score (ITT) |
-1.17; -0.94 | 0.1598 |
| SECONDARY Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT) |
-0.99; -0.88 | 0.4544 |
Eligibility Criteria
INCLUSION CRITERIA MAIN STUDY
- Diagnosis of HD based on clinical features and the presence of ≥36 CAG repeats in the huntingtin gene
- Diagnostic confidence level (DCL) of 4
- Adult-onset HD with onset of signs and symptoms ≥18 years of age
- Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of ≥7, at screening
EXCLUSION CRITERIA
- Use of pridopidine within 12 months before the baseline visit.
- Gene therapy at any time
- Any serious medical condition or clinically significant laboratory, or vital sign abnormality that precludes the patient's safe participation in and completion of the study e.g. significant heart disease within 12 weeks before baseline or history of certain cardiac arrhythmias
- History of epilepsy or seizures within the last 5 years
- Pregnant or breastfeeding, or intention to become pregnant during the study
Data sourced from ClinicalTrials.gov (NCT04556656) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.