Phase 3 N=499 Randomized Quadruple-blind Treatment

PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD

Huntington Disease

Bottom Line

View on ClinicalTrials.gov: NCT04556656 ↗

Enrolled (actual)

499

Serious AEs

11.0%

Results posted

Mar 2025

Primary outcome: Primary: 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

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Pridopidine (Drug); Placebo (Drug)
Age: Adult, Older Adult · 25+ yrs
Sex: All
Sponsor: Prilenia
Primary completion: Mar 2023

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

Summary

This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04556656). 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.