Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
A Phase 2, Randomized, Placebo Controlled, Double Blind Proof-of-concept Study Of The Efficacy And Safety Of Pf-02545920 In Subjects With Huntington's Disease
2 other identifiers
interventional
272
5 countries
57
Brief Summary
This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease. A total of approximately 260 subjects are planned to be randomized in the study. Primary endpoint is the change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 26 weeks of treatment. secondary endpoints will include change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 13 and 26 weeks of treatment and Clinical Global Impression-Improvement score after 13 and 26 weeks of treatment.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Sep 2014
57 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 16, 2014
CompletedFirst Posted
Study publicly available on registry
July 22, 2014
CompletedStudy Start
First participant enrolled
September 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2016
CompletedResults Posted
Study results publicly available
November 17, 2017
CompletedNovember 17, 2017
October 1, 2017
2 years
July 16, 2014
September 7, 2017
October 13, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.
The UHDRS was a clinical rating scale which has been developed by the Huntington Disease Study Group (HSG) to provide a uniform assessment of the clinical features and course of Huntington's Disease (HD). The components of the full UHDRS assess motor function, cognition, behavior and functional abilities. The total motor score (TMS) assessed motor features of HD with standardized ratings of oculomotor function, dysarthria, chorea, dystonia, gait, and postural stability. Some items (such as chorea and dystonia) required grading each extremity (face, bucco-oral-lingual, and trunk) separately. Eye movements require both horizontal and vertical grades. The total motor impairment scores was the sum of all the individual 31 motor sub-items (each rated from 0 to 4), with higher scores indicating more severe motor impairment than lower scores. The range of TMS is 0-124.
Baseline, Week 26
Secondary Outcomes (14)
Number of Participants That Met White Blood Count (WBC) and Absolute Neutrophil Count (ANC) Stopping Criteria
Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
Number of Participants With Adverse Events
Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
Number of Participants With Serious Adverse Events
Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormalities)
Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
Number of Participants With Laboratory Test Abnormalities (With Normal Baseline)
Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
- +9 more secondary outcomes
Study Arms (3)
20 mg PF-02545920 BID
EXPERIMENTAL20 mg PF-02545920 BID
5 mg PF-02545920 BID
EXPERIMENTAL5 mg PF-02545920 BID
Placebo BID
PLACEBO COMPARATORMatching placebo
Interventions
20 mg twice a day (BID) for 26 weeks. Each 20 mg dose will be taken as 4 tablets of 5 mg. The 20mg dose will be titrated as follow: 5mg BID for 7 days, 10mg BID for 7 days, 15 mg BID for 7 days and 20 mg BID to week 26. Study drug will be provided in weekly blister cards.
Matching Placebo twice a day (BID) for 26 weeks. Each placebo dose will be taken as 4 tablets of matching Placebo. The placebo dose will not be titrated. Matching placebo will be provided in weekly blister cards.
Eligibility Criteria
You may qualify if:
- CAG repeat equal or greater than 36;
- Total motor score equal or greater than 10;
- Total functional capacity equal or greater than 7.
You may not qualify if:
- Clinically significant neurologic disorder other than Huntington's disease;
- Other severe acute psychiatric conditions, mania and/or psychosis;
- History of neutropenia, and myeloproliferative disorders;
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Pfizerlead
Study Sites (57)
The Kirklin Clinic of UAB Hospital
Birmingham, Alabama, 35233, United States
The University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Mayo Clinic Arizona
Phoenix, Arizona, 85054, United States
Mayo Clinic Arizona
Scottsdale, Arizona, 85259, United States
University of California, Irvine
Irvine, California, 92697, United States
Ronald Regan UCLA Medical Center Drug Information Center
Los Angeles, California, 90095, United States
UCLA Neurology Clinic
Los Angeles, California, 90095, United States
UCLA Radiology
Los Angeles, California, 90095, United States
Rocky Mountain Movement Disorders Center
Englewood, Colorado, 80113, United States
University of Florida Center for Movement Disorders and Neurorestoration
Gainesville, Florida, 32607, United States
Indiana University Health Neuroscience Center
Indianapolis, Indiana, 46202, United States
Massachusetts General Hospital
Charlestown, Massachusetts, 02129, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Albany Medical College
Albany, New York, 12208, United States
Wake Forest Baptist Medical Center - Dept. of Neurology
Winston-Salem, North Carolina, 27157, United States
Cleveland Clinic Foundation Hospital Pharmacy
Cleveland, Ohio, 44195, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Davis Medical Center
Columbus, Ohio, 43210, United States
The Wexner Medical Center at the Ohio State University
Columbus, Ohio, 43221, United States
Wexner Medical Center at the Ohio State University
Columbus, Ohio, 43221, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
The Centre for Huntington Disease
Vancouver, British Columbia, V6T 2B5, Canada
Centre for Movement Disorders
Toronto, Ontario, M3B 2S7, Canada
CHUM-Notre-Dame Hospital
Montreal, Quebec, H2L 4M1, Canada
CHUM Notre Dame, Pharmacie
Montreal, Quebec, H4L 4M1, Canada
Technische Universität München
München, Bavaria, 81675, Germany
Uniklinik RWTH Aachen
Aachen, 52074, Germany
Charite - Universitatsmedizin Berlin
Berlin, 10117, Germany
Prof. Dr. Carsten Saft
Bochum, 44791, Germany
Friedrich-Alexander-Universität
Erlangen, 91054, Germany
Prof. Dr. med. Stephan Klebe
Freiburg im Breisgau, 79106, Germany
Universitat zu Lubeck
Lübeck, 23562, Germany
Philipps Universitat Marburg
Marburg, 35043, Germany
George Huntington Institut
Münster, 48149, Germany
Kbo-Isar-Amper-Klinikum gGmbH
Taufkirchen, 84416, Germany
Universitätsklinikum Ulm
Ulm, 89081, Germany
Universitatsklinikum Wuerzburg
Würzburg, 97080, Germany
Copernicus Podmiot Leczniczy sp. z o.o.
Gdansk, 80462, Poland
Krakowska Akademia Neurologii Sp. z o.o.
Krakow, 31505, Poland
Solumed Centrum Medyczne
Poznan, 60529, Poland
Instytut Psychiatrii i Neurologii
Warsaw, 02957, Poland
NHS Grampian, Clinical Genetics Centre
Aberdeen, Aberdeenshire, AB25 2ZA, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, Hampshire, SO16 6YD, United Kingdom
Oxford University Hospitals NHS Trust
Oxford, Oxfordshire, OX3 9DU, United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
Sheffield, South Yorkshire, S10 2JF, United Kingdom
Birmingham & Solihull Mental Health NHS Foundation Trust
Birmingham, West Midlands, B15 2FG, United Kingdom
John Van Geest Centre for Brain Repair
Cambridge, CB2 0PY, United Kingdom
University of Wales Hospital
Cardiff, CF14 4XW, United Kingdom
NHS Greater Glasgow and Clyde
Glasgow, G51 4TF, United Kingdom
Clinical Research Facility
London, SE1 9RT, United Kingdom
University College London Hospitals Huntington's Disease Research Centre
London, WC1B 5EH, United Kingdom
University College London Hospitals NHS Foundation Trust
London, WC1N 3BG, United Kingdom
Central Manchester University Hospitals NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
The National Institute for Health Research/Wellcome Trust Clinical Research Facility
Manchester, M13 9WL, United Kingdom
St Nicholas Hospital
Newcastle upon Tyne, NE3 3XT, United Kingdom
Newcastle Magnetic Resonance Centre
Newcastle upon Tyne, NE4 5PL, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Pfizer ClinicalTrials.gov Call Center
- Organization
- Pfizer, Inc.
Study Officials
- STUDY DIRECTOR
Pfizer CT.gov Call Center
Pfizer
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 16, 2014
First Posted
July 22, 2014
Study Start
September 1, 2014
Primary Completion
September 1, 2016
Study Completion
October 1, 2016
Last Updated
November 17, 2017
Results First Posted
November 17, 2017
Record last verified: 2017-10