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

Phase 3

Completed

• Conditions: Huntington Disease
• Interventions: Drug: Pridopidine; Drug: Placebo

• Registration Number: NCT04556656
• Lead Sponsor: Prilenia

Brief Summary

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

Detailed Description

This is a phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pridopidine 45 mg BID in patients with early stage HD.

Study Timeline

• Study First Submitted: 2020-09-14
• Study First Submitted QC: 2020-09-14
• Study First Posted: 2020-09-21
• Study Start: 2020-10-16
• Primary Completion: 2023-03-14
• Study Completion: 2024-03-21
• Results First Submitted: 2024-06-13
• Status Verified: 2025-02
• Results First Submitted QC: 2025-02-21
• Last Update Submitted: 2025-02-21
• Results First Posted: 2025-03-12
• Last Update Posted: 2025-03-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 499

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Pridopidine	Pridopidine	45 mg pridopidine twice daily (BID)
Placebo	Placebo	Matching placebo

Primary Outcome Measures

Name	Time	Method
Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT)	From baseline to Week 65	The primary efficacy endpoint for this study was the change from baseline to Week 65 in the TFC (defined as the sum of all TFC 5-items ratings \[domestic chores, activities of daily living, finances, care level, and occupation\]). The TFC is the standard and well-accepted clinical scale for staging and tracking the progression of HD using functional capacity. Scores range from 0 to 13, with 13 as the least affected and 0 as complete incapacity.
Change From Baseline to Week 65 in the UHDRS TFC Score (ITT)	From baseline to Week 65.	The primary efficacy endpoint for this study was the change from baseline to Week 65 in the TFC (defined as the sum of all TFC 5-items ratings \[domestic chores, activities of daily living, finances, care level, and occupation\]). The TFC is the standard and well-accepted clinical scale for staging and tracking the progression of HD using functional capacity. Scores range from 0 to 13, with 13 as the least affected and 0 as complete incapacity.
Change from baseline in the Unified Huntington Disease Rating Scale-Total functional capacity (UHDRS-TFC) score	Baseline, 65 weeks

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT)	From baseline to Week 65	The composite Unified Huntington Disease Rating Scale (cUHDRS) uses 4 components: Total Motor Score (TMS) assesses motor features (oculomotor, dysarthria, chorea, dystonia, gait, postural stability). Higher score = worse outcome. Best score=0. Worst score= 124.; Stroop Word Reading (SWR) measures attention and mental flexibility. Pat. reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome. Best score=100. Worst score=0.; Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Participant has 90 sec to match numbers with symbols. Scores = correct answers in 90 sec. Higher score = better outcome. Best score=120. Worst score=0.; Total Functional Capacity (TFC) tests the capacity to maintain daily living, finances, care level, occupation. Higher score = better outcome. Best score=13. Worst score=0.; Total integrated cUHDRS scale range: -7.6 to 24.8. The higher, the better.

Trial Locations

Locations (58)

Prilenia Investigational site (Site 291); 🇦🇹 Innsbruck, Austria

Prilenia Investigational Site (Site 385); 🇫🇷 Lille, France

Prilenia Investigational Site (Site 384); 🇫🇷 Marseille, France

Prilenia Investigational Site (Site 376); 🇩🇪 Münster, Germany

Prilenia Investigational Site (Site 394); 🇮🇹 Bologna, Italy

Prilenia Investigational Site (Site 333); 🇺🇸 Washington, District of Columbia, United States

Prilenia Investigational Site (Site 032); 🇺🇸 Atlanta, Georgia, United States

Prilenia Investigational Site (Site 028); 🇺🇸 Baltimore, Maryland, United States

Prilenia Investigational Site (Site 037); 🇺🇸 Albany, New York, United States

Prilenia Investigational Site (Site 017); 🇺🇸 Boston, Massachusetts, United States

Prilenia Investigational Site (Site 076); 🇺🇸 Boston, Massachusetts, United States

Prilenia Investigational Site (Site 088); 🇺🇸 Chicago, Illinois, United States

Prilenia Investigational Site (Site 029); 🇺🇸 Kansas City, Kansas, United States

Prilenia Investigational Site (Site 061); 🇺🇸 Davis, California, United States

Prilenia Investigational Site (Site 343); 🇺🇸 Englewood, Colorado, United States

Prilenia Investigational Site (Site 002); 🇺🇸 New York, New York, United States

Prilenia Investigational Site (Site 027); 🇺🇸 Saint Louis, Missouri, United States

Prilenia Investigational Site (Site 020); 🇺🇸 Columbus, Ohio, United States

Prilenia Investigational Site (Site 018); 🇺🇸 Philadelphia, Pennsylvania, United States

Prilenia Investigational Site (Site 030); 🇨🇦 Calgary, Alberta, Canada

Prilenia Investigational Site (Site 388); 🇨🇿 Prague, Czechia

Prilenia Investigational Site (Site 232); 🇨🇦 Halifax, Canada

Prilenia Investigational Site (Site 392); 🇫🇷 Paris, France

Prilenia Investigational Site (Site 234); 🇩🇪 Aachen, Germany

Prilenia Investigational site (Site 292); 🇩🇪 Taufkirchen, Germany

Prilenia Investigational Site (Site 239); 🇮🇹 Milano, Italy

Prilenia Investigational Site (Site 379); 🇩🇪 Bochum, Germany

Prilenia Investigational Site (Site 377); 🇩🇪 Lübeck, Germany

Prilenia Investigational Site (Site 175); 🇩🇪 Ulm, Germany

Prilenia Investigational Site (Site 044); 🇳🇱 Leiden, Netherlands

Prilenia Investigational Site (Site 390); 🇬🇧 Cardiff, United Kingdom

Prilenia Investigational Site (Site 228); 🇮🇹 Roma, Italy

Prilenia Investigational Site (Site 387); 🇳🇱 Maastricht, Netherlands

Prilenia Investigational Site (Site 386); 🇵🇱 Gdańsk, Poland

Prilenia Investigational Site (Site 382); 🇪🇸 Valencia, Spain

Prilenia Investigational Site (Site 380); 🇪🇸 Barcelona, Spain

Prilenia Investigational Site (Site 244); 🇵🇱 Kraków, Poland

Prilenia Investigational Site (Site 246); 🇵🇱 Warsaw, Poland

Prilenia Investigational Site (Site 180); 🇬🇧 Aberdeen, United Kingdom

Prilenia Investigational Site (Site 381); 🇪🇸 Burgos, Spain

Prilenia Investigational Site (Site 176); 🇪🇸 Madrid, Spain

Prilenia Investigational Site (Site 051); 🇺🇸 San Diego, California, United States

Prilenia Investigational Site (Site 019); 🇺🇸 Tampa, Florida, United States

Prilenia Investigational Site (Site 119); 🇺🇸 Durham, North Carolina, United States

Prilenia Investigational Site (Site 089); 🇺🇸 Cincinnati, Ohio, United States

Prilenia Investigational Site (Site 006); 🇺🇸 Portland, Oregon, United States

Prilenia Investigational Site (Site 031); 🇺🇸 Nashville, Tennessee, United States

Prilenia Investigational Site (Site 199); 🇺🇸 Houston, Texas, United States

Prilenia Investigational Site (Site 096); 🇺🇸 Seattle, Washington, United States

Prilenia Investigational Site (Site 249); 🇮🇹 Bari, Italy

Prilenia Investigational Site (Site 393); 🇮🇹 Napoli, Italy

Prilenia Investigational Site (Site 378); 🇬🇧 Newcastle, United Kingdom

Prilenia Investigational Site (Site 087); 🇺🇸 Louisville, Kentucky, United States

Prilenia Investigational Site (Site 048); 🇨🇦 Vancouver, British Columbia, Canada

Prilenia Investigational Site (Site 098); 🇨🇦 Montréal, Quebec, Canada

Prilenia Investigational Site (Site 160); 🇺🇸 Gainesville, Florida, United States

Prilenia Investigational Site (Site 083); 🇺🇸 Wichita, Kansas, United States

Prilenia Investigational Site (Site 326); 🇺🇸 Richmond, Virginia, United States