PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 499
Not provided
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.
- 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 061)
🇺🇸Davis, California, United States
Prilenia Investigational Site (Site 051)
🇺🇸San Diego, California, United States
Prilenia Investigational Site (Site 343)
🇺🇸Englewood, Colorado, United States
Prilenia Investigational Site (Site 333)
🇺🇸Washington, District of Columbia, United States
Prilenia Investigational Site (Site 160)
🇺🇸Gainesville, Florida, United States
Prilenia Investigational Site (Site 019)
🇺🇸Tampa, Florida, United States
Prilenia Investigational Site (Site 032)
🇺🇸Atlanta, Georgia, 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 083)
🇺🇸Wichita, Kansas, United States
Scroll for more (48 remaining)Prilenia Investigational Site (Site 061)🇺🇸Davis, California, United States