Clinical trial NCT02197130: Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington's Disease
Keywords: Huntington; chorea; total motor score; CAG repeat: total functional capacity; motor cognitive and behavioral symptoms
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2014-07-22
Enrollment: 272
Sex: ALL
Minimum age (years): 30
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* CAG repeat equal or greater than 36;
* Total motor score equal or greater than 10;
* Total functional capacity equal or greater than 7.

Exclusion Criteria:

* Clinically significant neurologic disorder other than Huntington's disease;
* Other severe acute psychiatric conditions, mania and/or psychosis;
* History of neutropenia, and myeloproliferative disorders;

Primary outcomes:
measure: Change From Baseline in the Total Motor Score (TMS) Assessment of the Unified Huntington Disease Rating Scale (UHDRS) After 26 Weeks of Treatment.
timeFrame: Baseline, Week 26
Secondary outcomes:
measure: Number of Participants That Met White Blood Count (WBC) and Absolute Neutrophil Count (ANC) Stopping Criteria
timeFrame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Number of Participants With Adverse Events
timeFrame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Number of Participants With Serious Adverse Events
timeFrame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormalities)
timeFrame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Number of Participants With Laboratory Test Abnormalities (With Normal Baseline)
timeFrame: Screening, Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Absolute Values)
timeFrame: Screening, Day 1, 28, 91, and 182
measure: Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Increase From Baseline)
timeFrame: Screening, Day 1, 28, 91, and 182
measure: Number of Participants With Vital Sign Data That Met Criteria for Potential Clinical Concern (Decrease From Baseline)
timeFrame: Screening, Day 1, 28, 91, and 182
measure: Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Absolute Values)
timeFrame: Screening, Day 1, 28, 91, and 182
measure: Number of Participants With Electrocardiogram (ECG) Data That Met Criteria for Potential Clinical Concern(Increase From Baseline)
timeFrame: Screening, Day 1, 28, 91, and 182
measure: Severity of Adverse Events Related to Extrapyramidal Symptoms (EPS) Including Dystonia and Akathisia
timeFrame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Change From Baseline in the Total Maximum Chorea (TMC) Score of the UHDRS After 13 and 26 Weeks of Treatment.
timeFrame: Baseline, Week 13, Week 26
measure: Number of Participants With Suicidal Ideation or Suicidal Behavior as Assessed by the Columbia Suicide Severity Rating Scale (C-SSRS) From Baseline to Follow-up Visit
timeFrame: Day 1, 7, 14, 28, 56, 91, 133, 182 and follow-up visits (from Day 189 to 192)
measure: Clinical Global Impression of Improvement (CGI-I) Scale Score After 13 and 26 Weeks of Treatment.
timeFrame: Week 13 & Week 26
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Kirklin Clinic of UAB Hospital
state: Alabama
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Los Angeles
country: United States
facility: Ronald Regan UCLA Medical Center Drug Information Center
state: California
city: Los Angeles
country: United States
facility: UCLA Neurology Clinic
state: California
city: Los Angeles
country: United States
facility: UCLA Radiology
state: California
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Center for Movement Disorders and Neurorestoration
state: Florida
city: Indianapolis
country: United States
facility: Indiana University Health Neuroscience Center
state: Indiana
city: Charlestown
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest Baptist Medical Center - Dept. of Neurology
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation Hospital Pharmacy
state: Ohio
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Columbus
country: United States
facility: Davis Medical Center
state: Ohio
city: Columbus
country: United States
facility: The Wexner Medical Center at the Ohio State University
state: Ohio
city: Columbus
country: United States
facility: Wexner Medical Center at the Ohio State University
state: Ohio
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Vancouver
country: Canada
facility: The Centre for Huntington Disease
state: British Columbia
city: Toronto
country: Canada
facility: Centre for Movement Disorders
state: Ontario
city: Montreal
country: Canada
facility: CHUM-Notre-Dame Hospital
state: Quebec
city: Montreal
country: Canada
facility: CHUM Notre Dame, Pharmacie
state: Quebec
city: Munchen
country: Germany
facility: Technische Universität München
state: Bavaria
city: Aachen
country: Germany
facility: Uniklinik RWTH Aachen
city: Berlin
country: Germany
facility: Charite - Universitatsmedizin Berlin
city: Bochum
country: Germany
facility: Prof. Dr. Carsten Saft
city: Erlangen
country: Germany
facility: Friedrich-Alexander-Universität
city: Freiburg
country: Germany
facility: Prof. Dr. med. Stephan Klebe
city: Lubeck
country: Germany
facility: Universitat zu Lubeck
city: Marburg
country: Germany
facility: Philipps Universitat Marburg
city: Münster
country: Germany
facility: George Huntington Institut
city: Taufkirchen
country: Germany
facility: Kbo-Isar-Amper-Klinikum gGmbH
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm
city: Wuerzburg
country: Germany
facility: Universitatsklinikum Wuerzburg
city: Gdansk
country: Poland
facility: Copernicus Podmiot Leczniczy sp. z o.o.
city: Krakow
country: Poland
facility: Krakowska Akademia Neurologii Sp. z o.o.
city: Poznan
country: Poland
facility: Solumed Centrum Medyczne
city: Warszawa
country: Poland
facility: Instytut Psychiatrii i Neurologii
city: Aberdeen
country: United Kingdom
facility: NHS Grampian, Clinical Genetics Centre
state: Aberdeenshire
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
state: Hampshire
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Trust
state: Oxfordshire
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospitals NHS Foundation Trust
state: South Yorkshire
city: Birmingham
country: United Kingdom
facility: Birmingham & Solihull Mental Health NHS Foundation Trust
state: West Midlands
city: Cambridge
country: United Kingdom
facility: John Van Geest Centre for Brain Repair
city: Cardiff
country: United Kingdom
facility: University of Wales Hospital
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
city: London
country: United Kingdom
facility: Clinical Research Facility
city: London
country: United Kingdom
facility: University College London Hospitals Huntington's Disease Research Centre
city: London
country: United Kingdom
facility: University College London Hospitals NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust
city: Manchester
country: United Kingdom
facility: The National Institute for Health Research/Wellcome Trust Clinical Research Facility
city: Newcastle Upon Tyne
country: United Kingdom
facility: St Nicholas Hospital
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle Magnetic Resonance Centre
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: