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

Primary Purpose

Huntington Disease

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Pridopidine
Placebo
Sponsored by
Prilenia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington Disease focused on measuring Huntington Disease

Eligibility Criteria

25 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Prilenia Investigational Site (Site 061)
  • Prilenia Investigational Site (Site 051)
  • Prilenia Investigational Site (Site 343)
  • Prilenia Investigational Site (Site 333)
  • Prilenia Investigational Site (Site 160)
  • Prilenia Investigational Site (Site 019)
  • Prilenia Investigational Site (Site 032)
  • Prilenia Investigational Site (Site 088)
  • Prilenia Investigational Site (Site 029)
  • Prilenia Investigational Site (Site 083)
  • Prilenia Investigational Site (Site 087)
  • Prilenia Investigational Site (Site 028)
  • Prilenia Investigational Site (Site 017)
  • Prilenia Investigational Site (Site 076)
  • Prilenia Investigational Site (Site 027)
  • Prilenia Investigational Site (Site 037)
  • Prilenia Investigational Site (Site 002)
  • Prilenia Investigational Site (Site 119)
  • Prilenia Investigational Site (Site 089)
  • Prilenia Investigational Site (Site 020)
  • Prilenia Investigational Site (Site 006)
  • Prilenia Investigational Site (Site 018)
  • Prilenia Investigational Site (Site 031)
  • Prilenia Investigational Site (Site 199)
  • Prilenia Investigational Site (Site 326)
  • Prilenia Investigational Site (Site 096)
  • Prilenia Investigational site (Site 291)
  • Prilenia Investigational Site (Site 030)
  • Prilenia Investigational Site (Site 048)
  • Prilenia Investigational Site (Site 098)
  • Prilenia Investigational Site (Site 232)
  • Prilenia Investigational Site (Site 388)
  • Prilenia Investigational Site (Site 385)
  • Prilenia Investigational Site (Site 384)
  • Prilenia Investigational Site (Site 392)
  • Prilenia Investigational Site (Site 234)
  • Prilenia Investigational Site (Site 379)
  • Prilenia Investigational Site (Site 377)
  • Prilenia Investigational Site (Site 376)
  • Prilenia Investigational site (Site 292)
  • Prilenia Investigational Site (Site 175)
  • Prilenia Investigational Site (Site 249)
  • Prilenia Investigational Site (Site 394)
  • Prilenia Investigational Site (Site 239)
  • Prilenia Investigational Site (Site 393)
  • Prilenia Investigational Site (Site 228)
  • Prilenia Investigational Site (Site 044)
  • Prilenia Investigational Site (Site 387)
  • Prilenia Investigational Site (Site 386)
  • Prilenia Investigational Site (Site 244)
  • Prilenia Investigational Site (Site 246)
  • Prilenia Investigational Site (Site 380)
  • Prilenia Investigational Site (Site 381)
  • Prilenia Investigational Site (Site 176)
  • Prilenia Investigational Site (Site 382)
  • Prilenia Investigational Site (Site 180)
  • Prilenia Investigational Site (Site 390)
  • Prilenia Investigational Site (Site 378)

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Pridopidine

Placebo

Arm Description

45 mg pridopidine twice daily (BID)

Matching placebo

Outcomes

Primary Outcome Measures

Change from baseline in the Unified Huntington Disease Rating Scale-Total functional capacity (UHDRS-TFC) score

Secondary Outcome Measures

Full Information

First Posted
September 14, 2020
Last Updated
December 10, 2022
Sponsor
Prilenia
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1. Study Identification

Unique Protocol Identification Number
NCT04556656
Brief Title
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

2. Study Status

Record Verification Date
December 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 16, 2020 (Actual)
Primary Completion Date
March 3, 2023 (Anticipated)
Study Completion Date
April 7, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prilenia

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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. Eligible patients who completed the Main Study (65 to 78 weeks) will have the option to enroll into an open-label extension.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington Disease
Keywords
Huntington Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
499 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pridopidine
Arm Type
Experimental
Arm Description
45 mg pridopidine twice daily (BID)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Matching placebo
Intervention Type
Drug
Intervention Name(s)
Pridopidine
Intervention Description
Pridopidine hard gelatin capsule
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Pridopidine-matching placebo hard gelatin capsule
Primary Outcome Measure Information:
Title
Change from baseline in the Unified Huntington Disease Rating Scale-Total functional capacity (UHDRS-TFC) score
Time Frame
Baseline, 65 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Prilenia Investigational Site (Site 061)
City
Davis
State/Province
California
ZIP/Postal Code
95616
Country
United States
Facility Name
Prilenia Investigational Site (Site 051)
City
San Diego
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
Prilenia Investigational Site (Site 343)
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Prilenia Investigational Site (Site 333)
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20057
Country
United States
Facility Name
Prilenia Investigational Site (Site 160)
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32611
Country
United States
Facility Name
Prilenia Investigational Site (Site 019)
City
Tampa
State/Province
Florida
ZIP/Postal Code
33620
Country
United States
Facility Name
Prilenia Investigational Site (Site 032)
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Prilenia Investigational Site (Site 088)
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Prilenia Investigational Site (Site 029)
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66103
Country
United States
Facility Name
Prilenia Investigational Site (Site 083)
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67226
Country
United States
Facility Name
Prilenia Investigational Site (Site 087)
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40292
Country
United States
Facility Name
Prilenia Investigational Site (Site 028)
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21218
Country
United States
Facility Name
Prilenia Investigational Site (Site 017)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Prilenia Investigational Site (Site 076)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Prilenia Investigational Site (Site 027)
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63130
Country
United States
Facility Name
Prilenia Investigational Site (Site 037)
City
Albany
State/Province
New York
ZIP/Postal Code
12208
Country
United States
Facility Name
Prilenia Investigational Site (Site 002)
City
New York
State/Province
New York
ZIP/Postal Code
10027
Country
United States
Facility Name
Prilenia Investigational Site (Site 119)
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27708
Country
United States
Facility Name
Prilenia Investigational Site (Site 089)
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45221
Country
United States
Facility Name
Prilenia Investigational Site (Site 020)
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Prilenia Investigational Site (Site 006)
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Prilenia Investigational Site (Site 018)
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Prilenia Investigational Site (Site 031)
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Prilenia Investigational Site (Site 199)
City
Houston
State/Province
Texas
ZIP/Postal Code
77004
Country
United States
Facility Name
Prilenia Investigational Site (Site 326)
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23284
Country
United States
Facility Name
Prilenia Investigational Site (Site 096)
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Prilenia Investigational site (Site 291)
City
Innsbruck
Country
Austria
Facility Name
Prilenia Investigational Site (Site 030)
City
Calgary
State/Province
Alberta
ZIP/Postal Code
AB T2N 1N4
Country
Canada
Facility Name
Prilenia Investigational Site (Site 048)
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
BC V6T 1Z4
Country
Canada
Facility Name
Prilenia Investigational Site (Site 098)
City
Montréal
State/Province
Quebec
ZIP/Postal Code
QC H2X 3E4
Country
Canada
Facility Name
Prilenia Investigational Site (Site 232)
City
Halifax
ZIP/Postal Code
NS B3S 1L8
Country
Canada
Facility Name
Prilenia Investigational Site (Site 388)
City
Prague
Country
Czechia
Facility Name
Prilenia Investigational Site (Site 385)
City
Lille
Country
France
Facility Name
Prilenia Investigational Site (Site 384)
City
Marseille
Country
France
Facility Name
Prilenia Investigational Site (Site 392)
City
Paris
Country
France
Facility Name
Prilenia Investigational Site (Site 234)
City
Aachen
Country
Germany
Facility Name
Prilenia Investigational Site (Site 379)
City
Bochum
Country
Germany
Facility Name
Prilenia Investigational Site (Site 377)
City
Lübeck
Country
Germany
Facility Name
Prilenia Investigational Site (Site 376)
City
Münster
Country
Germany
Facility Name
Prilenia Investigational site (Site 292)
City
Taufkirchen
Country
Germany
Facility Name
Prilenia Investigational Site (Site 175)
City
Ulm
Country
Germany
Facility Name
Prilenia Investigational Site (Site 249)
City
Bari
Country
Italy
Facility Name
Prilenia Investigational Site (Site 394)
City
Bologna
Country
Italy
Facility Name
Prilenia Investigational Site (Site 239)
City
Milano
Country
Italy
Facility Name
Prilenia Investigational Site (Site 393)
City
Napoli
Country
Italy
Facility Name
Prilenia Investigational Site (Site 228)
City
Roma
Country
Italy
Facility Name
Prilenia Investigational Site (Site 044)
City
Leiden
Country
Netherlands
Facility Name
Prilenia Investigational Site (Site 387)
City
Maastricht
Country
Netherlands
Facility Name
Prilenia Investigational Site (Site 386)
City
Gdańsk
Country
Poland
Facility Name
Prilenia Investigational Site (Site 244)
City
Kraków
Country
Poland
Facility Name
Prilenia Investigational Site (Site 246)
City
Warsaw
Country
Poland
Facility Name
Prilenia Investigational Site (Site 380)
City
Barcelona
Country
Spain
Facility Name
Prilenia Investigational Site (Site 381)
City
Burgos
Country
Spain
Facility Name
Prilenia Investigational Site (Site 176)
City
Madrid
Country
Spain
Facility Name
Prilenia Investigational Site (Site 382)
City
Valencia
Country
Spain
Facility Name
Prilenia Investigational Site (Site 180)
City
Aberdeen
Country
United Kingdom
Facility Name
Prilenia Investigational Site (Site 390)
City
Cardiff
Country
United Kingdom
Facility Name
Prilenia Investigational Site (Site 378)
City
Newcastle
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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