Clinical trial NCT02342548: Open Label Extension Study To Investigate Long Term Safety, Tolerability And Efficacy Of Pf-02545920 In Subjects With Huntington's Disease Who Completed Study A8241021
Official title: An Open Label Extension Study To Investigate The Long Term Safety, Tolerability And Efficacy Of Pf-02545920 In Subjects With Huntington's Disease Who Previously Completed Study A8241021
Study type: INTERVENTIONAL
Overall status: TERMINATED
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: 2015-01-21
Enrollment: 188
Sex: ALL
Minimum age (years): 30
Maximum age (years): 66
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study is a 12 month open label extension study of PF-02545920 20 mg dosed BID following study A8241021 in subjects with HD. Primary endpoints will be to assess long-term safety and tolerability of 20 mg BID of PF-02545920. Secondary endpoints will be the change from baseline in the Total Motor Score (TMS)assessment, and/ior the Total maximum Chorea (TMC) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 6 and 12 months of treatment, and Clinical Global Impression-Improvement score after 6 and 12 months of treatment. Subjects, who were assigned to the 20 mg PF-02545920 dose group in the preceding A8241021 study, will receive 20 mg PF-02545920 without any titration. All other subjects will be titrated to the 20 mg BID dose as follows: 5 mg BID for 7 days, 10 mg BID for 7 days, 15 mg BID for 7 days, then 20 mg BID for the remainder of the treatment phase. Up to 260 subjects may take part in this open label extension


Eligibility criteria: Inclusion Criteria:

* Subjects must have completed study A8241021
* Diagnosis of HD, including ≥36 CAG repeats.

Exclusion Criteria:

* Significant neurological disorder other than Huntington's disease.
* WBC ≤ 3500/mm3 AND/OR ANC ≤ 2000/mm3 and history of neutropenia or myeolo-proliferative disorders.
* Any drug related SAE experienced during study A8241021 which were not approved as acceptable for enrollment in A8241022.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events and Serious Adverse Events
timeFrame: 1 year
measure: Number of Participants With Laboratory Test Abnormalities (Without Regard to Baseline Abnormality)
timeFrame: 1 year
measure: Number of Participants With Vital Signs Data Meeting Categorical Summarization Criteria
timeFrame: 1 year
measure: Number of Participants With Electrocardiogram (ECG) Data Meeting Categorical Summarization Criteria
timeFrame: 1 year
measure: Number of Participants With Abnormal White Blood Cell Count and Absolute Neutrophil Count (Without Regard to Baseline Abnormality)
timeFrame: 1 year
measure: Number of Participants With Laboratory Test Abnormalities (Normal Baseline)
timeFrame: 1 year
measure: Number of Participants With Adverse Events Related to Extrapyramidal Symptoms by Severity
timeFrame: 1 year
measure: Number of Participants in Each Columbia Classification Algorithm of Suicide Assessment (C-CASA) Category
timeFrame: Baseline (Day 1), Weeks 2 and 4, Months 3, 6, 9 and 12, follow-up visit (7-14 days after the last dose of Month 12)
Secondary outcomes:
measure: Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score
timeFrame: Baseline (Day 1), Month 6, and Month 12
measure: Change From Baseline in Unified Huntington's Disease Rating Scale (UHDRS) Total Maximum Chorea (TMC) Score
timeFrame: Baseline (Day 1), Month 6, and Month 12
measure: Absolute Value in Clinical Global Impression of Improvement (CGI-I) Score
timeFrame: Month 6 and Month 12
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: The Kirkland Clinic of UAB Hospital
state: Alabama
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
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 Reagan 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 & Neurorestoration
state: Florida
city: Indianapolis
country: United States
facility: Indiana University Health Neuroscience Center
state: Indiana
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
state: North Carolina
city: Columbus
country: United States
facility: The Ohio State University
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: The Wright Center of Innovation- 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, The University of British Columbia
state: British Columbia
city: Toronto
country: Canada
facility: Center 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: Aachen
country: Germany
facility: Uniklinik RWTH Aachen
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Bochum
country: Germany
facility: St. Josef Hospital
city: Erlangen
country: Germany
facility: Friedrich-Alexander-Universität
city: Lübeck
country: Germany
facility: Universität zu Lübeck
city: Marburg
country: Germany
facility: Philipps Universitat Marburg
city: Munchen
country: Germany
facility: Technische Universität München
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: Universitaetsklinikum Wuerzburg
city: Gdansk
country: Poland
facility: Copernicus Podmiot Leczniczy sp.zo.o
city: Krakow
country: Poland
facility: Krakowska Akademia Neurologii Sp. zo.o
city: Poznan
country: Poland
facility: Solumed Centrum Medyczne
city: Warszawa
country: Poland
facility: Instytut Psychiatrii i Neurologii, I Klinika Neurologiczna
city: Oxford Road
country: United Kingdom
facility: Central Manchester University Hospitals NHS Foundation Trust
state: Manchester
city: Gosforth
country: United Kingdom
facility: St Nicholas Hospital
state: Newcastle UPON TYNE
city: Birmingham
country: United Kingdom
facility: Bimingham & Solihull Mental Health NHS Foundation Trust Department of Neuropsychiatry
state: WEST Midlands
city: Aberdeen
country: United Kingdom
facility: NHS Grampian, Aberdeen Royal Infirmary, Clinical Genetics Centre
city: Cardiff
country: United Kingdom
facility: Institute of Psychological Medicine and Clinical Neurosciences
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
city: London
country: United Kingdom
facility: Guy's and St. Thomas' NHS Foundation Trust
city: London
country: United Kingdom
facility: University College London Hospitals Huntington's Diesease
city: London
country: United Kingdom
facility: University College London 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: Newcastle Magnetic Resonance Centre
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Trust
city: Sheffield
country: United Kingdom
facility: Sheffield Teaching Hospital NHS Foundation Trust
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust, Wessex Neurological Centre
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: