MedDataX Logo

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

NCT ID: NCT04556656

Last Updated: 2025-03-12

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

499 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-16

Study Completion Date

2024-03-21

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

NCT02006472

Huntington's Disease
COMPLETED PHASE2

A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease

NCT02494778

Huntington's Disease
TERMINATED PHASE2

A Study to Evaluate Sigma-1 and Dopamine-2 Receptor Occupancy by Pridopidine in the Human Brain of Healthy Volunteers and in Patients With Huntington's Disease

NCT03019289

Health Volunteers, Huntington Disease
COMPLETED PHASE1

A Study of Treatment With Pridopidine (ACR16) in Participants With Huntington's Disease

NCT00665223

Huntington's Disease
COMPLETED PHASE3

Open-label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington Disease

NCT01306929

Huntington Disease
COMPLETED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Huntington Disease

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Pridopidine

45 mg pridopidine twice daily (BID)

Group Type EXPERIMENTAL

Pridopidine

Intervention Type DRUG

Pridopidine hard gelatin capsule

Placebo

Matching placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Pridopidine-matching placebo hard gelatin capsule

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Pridopidine

Pridopidine hard gelatin capsule

Intervention Type DRUG

Placebo

Pridopidine-matching placebo hard gelatin capsule

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

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
Minimum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Prilenia

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Yael Cohen

Role: STUDY_DIRECTOR

Prilenia

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Prilenia Investigational Site (Site 061)

Davis, California, United States

Site Status

Prilenia Investigational Site (Site 051)

San Diego, California, United States

Site Status

Prilenia Investigational Site (Site 343)

Englewood, Colorado, United States

Site Status

Prilenia Investigational Site (Site 333)

Washington D.C., District of Columbia, United States

Site Status

Prilenia Investigational Site (Site 160)

Gainesville, Florida, United States

Site Status

Prilenia Investigational Site (Site 019)

Tampa, Florida, United States

Site Status

Prilenia Investigational Site (Site 032)

Atlanta, Georgia, United States

Site Status

Prilenia Investigational Site (Site 088)

Chicago, Illinois, United States

Site Status

Prilenia Investigational Site (Site 029)

Kansas City, Kansas, United States

Site Status

Prilenia Investigational Site (Site 083)

Wichita, Kansas, United States

Site Status

Prilenia Investigational Site (Site 087)

Louisville, Kentucky, United States

Site Status

Prilenia Investigational Site (Site 028)

Baltimore, Maryland, United States

Site Status

Prilenia Investigational Site (Site 017)

Boston, Massachusetts, United States

Site Status

Prilenia Investigational Site (Site 076)

Boston, Massachusetts, United States

Site Status

Prilenia Investigational Site (Site 027)

St Louis, Missouri, United States

Site Status

Prilenia Investigational Site (Site 037)

Albany, New York, United States

Site Status

Prilenia Investigational Site (Site 002)

New York, New York, United States

Site Status

Prilenia Investigational Site (Site 119)

Durham, North Carolina, United States

Site Status

Prilenia Investigational Site (Site 089)

Cincinnati, Ohio, United States

Site Status

Prilenia Investigational Site (Site 020)

Columbus, Ohio, United States

Site Status

Prilenia Investigational Site (Site 006)

Portland, Oregon, United States

Site Status

Prilenia Investigational Site (Site 018)

Philadelphia, Pennsylvania, United States

Site Status

Prilenia Investigational Site (Site 031)

Nashville, Tennessee, United States

Site Status

Prilenia Investigational Site (Site 199)

Houston, Texas, United States

Site Status

Prilenia Investigational Site (Site 326)

Richmond, Virginia, United States

Site Status

Prilenia Investigational Site (Site 096)

Seattle, Washington, United States

Site Status

Prilenia Investigational site (Site 291)

Innsbruck, , Austria

Site Status

Prilenia Investigational Site (Site 030)

Calgary, Alberta, Canada

Site Status

Prilenia Investigational Site (Site 048)

Vancouver, British Columbia, Canada

Site Status

Prilenia Investigational Site (Site 098)

Montreal, Quebec, Canada

Site Status

Prilenia Investigational Site (Site 232)

Halifax, , Canada

Site Status

Prilenia Investigational Site (Site 388)

Prague, , Czechia

Site Status

Prilenia Investigational Site (Site 385)

Lille, , France

Site Status

Prilenia Investigational Site (Site 384)

Marseille, , France

Site Status

Prilenia Investigational Site (Site 392)

Paris, , France

Site Status

Prilenia Investigational Site (Site 234)

Aachen, , Germany

Site Status

Prilenia Investigational Site (Site 379)

Bochum, , Germany

Site Status

Prilenia Investigational Site (Site 377)

Lübeck, , Germany

Site Status

Prilenia Investigational Site (Site 376)

Münster, , Germany

Site Status

Prilenia Investigational site (Site 292)

Taufkirchen, , Germany

Site Status

Prilenia Investigational Site (Site 175)

Ulm, , Germany

Site Status

Prilenia Investigational Site (Site 249)

Bari, , Italy

Site Status

Prilenia Investigational Site (Site 394)

Bologna, , Italy

Site Status

Prilenia Investigational Site (Site 239)

Milan, , Italy

Site Status

Prilenia Investigational Site (Site 393)

Napoli, , Italy

Site Status

Prilenia Investigational Site (Site 228)

Roma, , Italy

Site Status

Prilenia Investigational Site (Site 044)

Leiden, , Netherlands

Site Status

Prilenia Investigational Site (Site 387)

Maastricht, , Netherlands

Site Status

Prilenia Investigational Site (Site 386)

Gdansk, , Poland

Site Status

Prilenia Investigational Site (Site 244)

Krakow, , Poland

Site Status

Prilenia Investigational Site (Site 246)

Warsaw, , Poland

Site Status

Prilenia Investigational Site (Site 380)

Barcelona, , Spain

Site Status

Prilenia Investigational Site (Site 381)

Burgos, , Spain

Site Status

Prilenia Investigational Site (Site 176)

Madrid, , Spain

Site Status

Prilenia Investigational Site (Site 382)

Valencia, , Spain

Site Status

Prilenia Investigational Site (Site 180)

Aberdeen, , United Kingdom

Site Status

Prilenia Investigational Site (Site 390)

Cardiff, , United Kingdom

Site Status

Prilenia Investigational Site (Site 378)

Newcastle, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Austria Canada Czechia France Germany Italy Netherlands Poland Spain United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Reilmann R, Feigin A, Rosser AE, Kostyk SK, Saft C, Cohen Y, Schuring H, Hand R, Tan AM, Chen K, Feng W, Navon-Perry L, Cruz-Herranz A, Syltevik C, Boot D, Squitieri F, Kayson E, Mehra M, Goldberg YP, Geva M, Hayden MR; PROOF-HD study investigators. Pridopidine in early-stage manifest Huntington's disease: a phase 3 trial. Nat Med. 2025 Sep 5. doi: 10.1038/s41591-025-03920-3. Online ahead of print.

Reference Type DERIVED
PMID: 40913168 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2020-002822-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PL101-HD301

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

A Study of Pridopidine (ACR16) for the Treatment of Participants With Huntington's Disease
NCT00724048 COMPLETED PHASE2
Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease
NCT01806896 COMPLETED PHASE2
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
NCT05358717 COMPLETED PHASE2
Safety and Efficacy of Propranolol in the Treatment of Tardive Dyskinesia
NCT03254186 WITHDRAWN PHASE2/PHASE3
Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
NCT02197130 COMPLETED PHASE2
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
NCT01590888 COMPLETED PHASE2
An Extension Study to Evaluate the Long-Term Safety and Efficacy of Votoplam in Participants With Huntington's Disease (HD)
NCT06254482 ACTIVE_NOT_RECRUITING PHASE2
A Trial of Memantine as Symptomatic Treatment for Early Huntington Disease
NCT01458470 COMPLETED PHASE2
A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease
NCT06585449 RECRUITING PHASE1
Efficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing Chorea
NCT01019473 TERMINATED PHASE2
A Comparative Phase 2 Study Assessing the Efficacy of Triheptanoin, an Anaplerotic Therapy in Huntington's Disease
NCT02453061 COMPLETED PHASE2
A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington Disease
NCT01485965 COMPLETED PHASE1
A Safety and Efficacy Study of Dimebon in Patients With Huntington Disease
NCT00920946 COMPLETED PHASE3
A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
NCT03761849 COMPLETED PHASE3
A Placebo- and Active Controlled Study of Preladenant in Subjects With Moderate to Severe Parkinson's Disease (P04938)
NCT01155466 COMPLETED PHASE3
PF-06669571 In Subjects With Idiopathic Parkinson's Disease
NCT02565628 COMPLETED PHASE1
A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod
NCT02215616 COMPLETED PHASE2
A Study to Evaluate the Efficacy and Safety of Intravenous Prasinezumab in Participants With Early Parkinson's Disease
NCT04777331 ACTIVE_NOT_RECRUITING PHASE2
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
NCT05107128 COMPLETED PHASE2
A Study to Assess the Safety and Effectiveness of Pridopidine Compared to Placebo in the Treatment of Levodopa-Induced Dyskinesia in Patients With Parkinson's Disease
NCT03922711 TERMINATED PHASE2
A Phase I Clinical Study of ER2001 Injection for the Treatment of Early Manifest Huntington's Disease.
NCT07339514 ACTIVE_NOT_RECRUITING PHASE1/PHASE2
Cooperative Huntington's Observational Research Trial
NCT00313495 UNKNOWN
Efficacy and Safety of SOM3355 in Huntington's Disease Chorea
NCT03575676 COMPLETED PHASE2
A Study to Evaluate the Efficacy and Safety of Intravenous (IV) Prasinezumab in Participants With Early-Stage Parkinson's Disease
NCT07174310 RECRUITING PHASE3
28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease
NCT05358821 COMPLETED PHASE2