This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
499
Pridopidine hard gelatin capsule
Pridopidine-matching placebo hard gelatin capsule
Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT)
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.
Time frame: From baseline to Week 65
Change From Baseline to Week 65 in the UHDRS TFC Score (ITT)
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.
Time frame: From baseline to Week 65.
Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT)
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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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 D.C., 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
...and 48 more locations
Time frame: From baseline to Week 65