Clinical trial NCT04556656: PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients With Early Stage of Huntington Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Huntington Disease
Keywords: Huntington Disease
Lead sponsor: Prilenia
Sponsor class: INDUSTRY
First posted: 2020-09-21
Enrollment: 499
Sex: ALL
Minimum age (years): 25
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: INCLUSION CRITERIA MAIN STUDY

1. Diagnosis of HD based on clinical features and the presence of ≥36 CAG repeats in the huntingtin gene
2. Diagnostic confidence level (DCL) of 4
3. Adult-onset HD with onset of signs and symptoms ≥18 years of age
4. Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of ≥7, at screening

EXCLUSION CRITERIA

1. Use of pridopidine within 12 months before the baseline visit.
2. Gene therapy at any time
3. 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
4. History of epilepsy or seizures within the last 5 years
5. Pregnant or breastfeeding, or intention to become pregnant during the study

Primary outcomes:
measure: Change From Baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity (UHDRS-TFC) Score (mITT)
timeFrame: From baseline to Week 65
measure: Change From Baseline to Week 65 in the UHDRS TFC Score (ITT)
timeFrame: From baseline to Week 65.
Secondary outcomes:
measure: Change From Baseline to Week 65 in Composite UHDRS (cUHDRS) Total Score (mITT)
timeFrame: From baseline to Week 65
Other outcomes:
measure: Change From Baseline in cUHDRS Total Score - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78
measure: Change From Baseline in Q-Motor Finger Tapping Inter-Onset Interval (IOI) Mean - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 52, Week 65, and Week 78.
measure: Change From Baseline in Q-Motor Pronation/Supination Inter-Tap-Interval (ITI) Mean - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 52, Week 65, and Week 78.
measure: Change in Q-Motor Pronation/Supination Inter-Onset-Interval (IOI) Mean - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 52, Week 65, and Week 78.
measure: Change From Baseline in the UHDRS TFC Score - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78
measure: Change From Baseline in Stroop Word Reading (SWR) - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78
measure: Change From Baseline in Symbol Digit Modalities Test (SDMT) - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78.
measure: Change From Baseline in Total Motor Score (TMS) - Patients Off ADMs (mITT)
timeFrame: Time course from baseline to Week 26, Week 39, Week 52, Week 65, and Week 78.
Locations:
city: Davis
country: United States
facility: Prilenia Investigational Site (Site 061)
state: California
city: San Diego
country: United States
facility: Prilenia Investigational Site (Site 051)
state: California
city: Englewood
country: United States
facility: Prilenia Investigational Site (Site 343)
state: Colorado
city: Washington
country: United States
facility: Prilenia Investigational Site (Site 333)
state: District of Columbia
city: Gainesville
country: United States
facility: Prilenia Investigational Site (Site 160)
state: Florida
city: Tampa
country: United States
facility: Prilenia Investigational Site (Site 019)
state: Florida
city: Atlanta
country: United States
facility: Prilenia Investigational Site (Site 032)
state: Georgia
city: Chicago
country: United States
facility: Prilenia Investigational Site (Site 088)
state: Illinois
city: Kansas City
country: United States
facility: Prilenia Investigational Site (Site 029)
state: Kansas
city: Wichita
country: United States
facility: Prilenia Investigational Site (Site 083)
state: Kansas
city: Louisville
country: United States
facility: Prilenia Investigational Site (Site 087)
state: Kentucky
city: Baltimore
country: United States
facility: Prilenia Investigational Site (Site 028)
state: Maryland
city: Boston
country: United States
facility: Prilenia Investigational Site (Site 017)
state: Massachusetts
city: Boston
country: United States
facility: Prilenia Investigational Site (Site 076)
state: Massachusetts
city: Saint Louis
country: United States
facility: Prilenia Investigational Site (Site 027)
state: Missouri
city: Albany
country: United States
facility: Prilenia Investigational Site (Site 037)
state: New York
city: New York
country: United States
facility: Prilenia Investigational Site (Site 002)
state: New York
city: Durham
country: United States
facility: Prilenia Investigational Site (Site 119)
state: North Carolina
city: Cincinnati
country: United States
facility: Prilenia Investigational Site (Site 089)
state: Ohio
city: Columbus
country: United States
facility: Prilenia Investigational Site (Site 020)
state: Ohio
city: Portland
country: United States
facility: Prilenia Investigational Site (Site 006)
state: Oregon
city: Philadelphia
country: United States
facility: Prilenia Investigational Site (Site 018)
state: Pennsylvania
city: Nashville
country: United States
facility: Prilenia Investigational Site (Site 031)
state: Tennessee
city: Houston
country: United States
facility: Prilenia Investigational Site (Site 199)
state: Texas
city: Richmond
country: United States
facility: Prilenia Investigational Site (Site 326)
state: Virginia
city: Seattle
country: United States
facility: Prilenia Investigational Site (Site 096)
state: Washington
city: Innsbruck
country: Austria
facility: Prilenia Investigational site (Site 291)
city: Calgary
country: Canada
facility: Prilenia Investigational Site (Site 030)
state: Alberta
city: Vancouver
country: Canada
facility: Prilenia Investigational Site (Site 048)
state: British Columbia
city: Montréal
country: Canada
facility: Prilenia Investigational Site (Site 098)
state: Quebec
city: Halifax
country: Canada
facility: Prilenia Investigational Site (Site 232)
city: Prague
country: Czechia
facility: Prilenia Investigational Site (Site 388)
city: Lille
country: France
facility: Prilenia Investigational Site (Site 385)
city: Marseille
country: France
facility: Prilenia Investigational Site (Site 384)
city: Paris
country: France
facility: Prilenia Investigational Site (Site 392)
city: Aachen
country: Germany
facility: Prilenia Investigational Site (Site 234)
city: Bochum
country: Germany
facility: Prilenia Investigational Site (Site 379)
city: Lübeck
country: Germany
facility: Prilenia Investigational Site (Site 377)
city: Münster
country: Germany
facility: Prilenia Investigational Site (Site 376)
city: Taufkirchen
country: Germany
facility: Prilenia Investigational site (Site 292)
city: Ulm
country: Germany
facility: Prilenia Investigational Site (Site 175)
city: Bari
country: Italy
facility: Prilenia Investigational Site (Site 249)
city: Bologna
country: Italy
facility: Prilenia Investigational Site (Site 394)
city: Milano
country: Italy
facility: Prilenia Investigational Site (Site 239)
city: Napoli
country: Italy
facility: Prilenia Investigational Site (Site 393)
city: Roma
country: Italy
facility: Prilenia Investigational Site (Site 228)
city: Leiden
country: Netherlands
facility: Prilenia Investigational Site (Site 044)
city: Maastricht
country: Netherlands
facility: Prilenia Investigational Site (Site 387)
city: Gdańsk
country: Poland
facility: Prilenia Investigational Site (Site 386)
city: Kraków
country: Poland
facility: Prilenia Investigational Site (Site 244)
city: Warsaw
country: Poland
facility: Prilenia Investigational Site (Site 246)
city: Barcelona
country: Spain
facility: Prilenia Investigational Site (Site 380)
city: Burgos
country: Spain
facility: Prilenia Investigational Site (Site 381)
city: Madrid
country: Spain
facility: Prilenia Investigational Site (Site 176)
city: Valencia
country: Spain
facility: Prilenia Investigational Site (Site 382)
city: Aberdeen
country: United Kingdom
facility: Prilenia Investigational Site (Site 180)
city: Cardiff
country: United Kingdom
facility: Prilenia Investigational Site (Site 390)
city: Newcastle
country: United Kingdom
facility: Prilenia Investigational Site (Site 378)
Study officials:
affiliation: Prilenia
name: Yael Cohen
role: STUDY_DIRECTOR
References: