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    Summary
    EudraCT Number:2014-004900-31
    Sponsor's Protocol Code Number:A8241022
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2015-02-13
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2014-004900-31
    A.3Full title of the trial
    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
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    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
    A.4.1Sponsor's protocol code numberA8241022
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1162-4293
    A.5.4Other Identifiers
    Name:US IND NumberNumber:118,646
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPfizer Inc, 235 East 42nd Street, New York, NY 10017
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPfizer Inc, 235 East 42nd Street, New York, NY 10017
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc
    B.5.2Functional name of contact pointClinical Trials.gov Call Center
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1800 718 1021
    B.5.5Fax number+1303 739 1119
    B.5.6E-mailClinicalTrials.govcallcenter@pfizer.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePF-02545920
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot available
    D.3.9.2Current sponsor codePF-02545920
    D.3.9.3Other descriptive namePF-02545920
    D.3.9.4EV Substance CodeSUB26938
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    HUNTINGTON'S DISEASE
    E.1.1.1Medical condition in easily understood language
    HUNTINGTON'S DISEASE
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10020469
    E.1.2Term Huntington's chorea
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To assess long-term safety and tolerability of 20 mg BID of PF-02545920 in subjects with HD.
    E.2.2Secondary objectives of the trial
    - To assess motor function after 6 and 12 month oral dosing with 20 mg BID of PF-02545920 in subjects with HD.
    - To assess the efficacy of 6 and 12 month oral dosing with 20 mg BID of PF-02545920 on chorea severity in subjects with HD.
    - To assess whether 6 and 12 month oral dosing with 20 mg BID of PF-02545920 can improve the overall clinical impression in subjects with HD.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    MRI study:
    At selected sites subjects will undergo brain MRI scans at Month 6 (V6) and Month 12/ET (V8), using a 3-Tesla MRI scanner and standardized image acquisition protocols.
    The MRI session will include three individual MRI acquisitions during the time the subject is in the magnet:
    1. A high resolution T1-weighted structural MRI.
    2. Brain white matter microstructures will be imaged using diffusion tensor imaging (DTI).
    3. Patterns of activation in the brain will be imaged using resting-state functional MRI.

    For objectives please refer to protocol
    E.3Principal inclusion criteria
    1. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
    2. Subjects who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
    3. Subjects must have completed Study A8241021 and, in the opinion of the investigator and sponsor, have been compliant with the STUDY PROCEDURES, Lifestyle Guidelines and study treatment.
    4. Only subjects who were part of the MRI cohort in study A8241021 are eligible for the MRI assessments.
    5. Males or females between the age of 30 years and 66 years (inclusive).
    6. Diagnosis of HD based on characteristic clinical findings, including presence of chorea, and genetic confirmation with the detection of an expansion of ≥36 CAG trinucleotide repeats in the huntingtin gene (Htt).
    7. Male and female subjects of childbearing potential and at risk for pregnancy must agree to use a highly effective method of contraception throughout the study and for at least 28 days (90 days for males) after the last dose of assigned treatment.
    Female subjects who are not of childbearing potential (ie, meet at least 1 of the following criteria):
    - Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    - Have medically confirmed ovarian failure; or
    - Achieved postmenopausal status, defined as follows: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; status may be confirmed by having a serum follicle stimulating hormone (FSH) level within the laboratory’s reference range for postmenopausal women.
    E.4Principal exclusion criteria
    1. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees directly involved in the conduct of the study.
    2. Participation in study(ies) involving investigational drug(s) (Phases 1-4) other than PF-02545920 within the past 9 months before the current study begins and/or during study participation.
    3. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
    4. Evidence or history of:
    a. Clinically significant neurologic disorder other than Huntington’s disease. This also includes subjects with previous history of epilepsy or seizures (except childhood febrile seizures), stroke, head injury with significant neurologic sequelae.
    b. Other severe acute psychiatric condition, mania and/or psychosis.
    c. For subjects who score ≥ 3 on the suicidal ideation item of the Problem Behaviors Assessment or answer “Yes” to the C-SSRS questions 4 or 5, a risk assessment should be done by a qualified mental health professional (eg, a psychiatrist or licensed PhD level clinical psychologist) to assess whether it is safe for the subject to participate in the study (See Suicidality Risk Assessment). In addition, subjects deemed by the investigator to be at significant risk of suicidal or violent behavior should be excluded. Attempted suicide or suicidal ideation with intention or plan, which required hospital admission and/or change of level of care within 15 months prior to Screening should be discussed with medical monitor or clinician before proceeding.
    5. Evidence or history of any clinically significant conditions which affect one of the following systems and which were used as exclusion criteria in preceding A8241021 study:
    a. Renal.
    b. Endocrine.
    c. Pulmonary.
    d. Hematological.
    e. Gastrointestinal (including any condition possibly affecting drug absorption, eg, gastrectomy, gastric bypass).
    f. Immunological, including positivity for human immunodeficiency virus (HIV) and acquired immunodeficiency syndrome (AIDS).
    g. Severe allergic diseases (excluding untreated, asymptomatic, seasonal and environmental allergies at time of dosing).
    h. Any history of malignant tumors and treatment within the previous 15 months.
    6. Subjects with:
    a. WBC ≤ 3500/mm3 OR ANC ≤ 2000/mm3 anytime in study A8241021 which were not approved as acceptable for enrollment in study A8241022 by the sponsor study clinician or sponsor medical monitor. These cases should be discussed with the sponsor medical monitor or clinician prior to submitting subject for an eligibility approval.
    b. History of neutropenia, including benign ethnic neutropenia, clozapine induced agranulocytosis or granulocytopenia.
    c. History of myeloproliferative disorders (primary myelofibrosis, polycythemia vera, essential thrombocythemia, chronic eosinophilic leukemia/hypereosinophilic syndrome, systemic mastocytosis).
    7. Evidence or history of clinically significant cardiovascular disease, including:
    a. Uncontrolled hypertension (sitting or supine diastolic blood pressure >95 mmHg and/or sitting or supine systolic blood pressure >170 mmHg with or without treatment).
    b. Evidence of orthostatic symptoms (eg, dizziness upon standing) or systolic blood pressure (BP) drop ≥ 20 mm Hg or diastolic BP drop ≥ 10 mmHg from supine to standing assessment at screening.
    c. Any 12-lead ECG with repeated demonstration of QTc >450 msec or a QRS interval >120 msec.
    d. Coronary bypass surgery.
    e. History of myocardial infarction or ischemic heart disease.
    f. Heart failure.
    g. Non clinically significant ECG findings including sinus bradycardia and tachycardia will not exclude subjects from the study.
    8. Subjects with laboratory test results reviewed at screening that deviate from the upper or lower limits of the reference range, as assessed by the study-specific laboratory and confirmed by a single repeat, if deemed necessary, except for non-clinically significant values, as determined by the investigator.
    a. AST or ALT must be ≤ 2 X upper limit of reference range.
    b. Total bilirubin must be within 1.5 X of the upper limit of reference range at screening; subjects with a history of Gilbert's syndrome may have a direct bilirubin measured and would be eligible for this study provided the direct bilirubin is ≤ ULN.
    ...
    (For further exclusion criteria, please refer to protocol.)
    E.5 End points
    E.5.1Primary end point(s)
    - Adverse events, weight, vital signs (pulse, blood pressure and body temperature), physical examination, neurological examination, electrocardiogram (ECG) and clinical laboratory findings (hematology, biochemistry and urinalysis).

    The endpoints are:
    - The number and proportion of subjects with adverse events.
    - Assessment of clinical laboratory parameters.
    - Assessment of vital signs.
    - Assessment of ECG parameters.
    - White blood count (WBC) and absolute neutrophil count (ANC) at each visit.
    - Abnormal laboratory findings from baseline.
    - Frequency and severity of adverse events related to extrapyramidal symptoms (EPS) including dystonia and akathisia, as assessed by the investigator.
    - C-SSRS (suicide severity assessment).
    E.5.1.1Timepoint(s) of evaluation of this end point
    - adverse events: v3-v9
    - Assessment of clinical laboratory parameters: v2-v9
    - Assessment of vital signs: v3, v4, v5, v7, v9
    - Assessment of ECG parameters: v2, v5-v8
    - White blood count (WBC) and absolute neutrophil count (ANC) at each visit
    - Abnormal laboratory findings from baseline: v2-v9
    - Frequency and severity of adverse events related to extrapyramidal symptoms (EPS) including dystonia and akathisia, as assessed by the investigator: v3-v9
    - C-SSRS (suicide severity assessment): v2-v9
    E.5.2Secondary end point(s)
    - Change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 6 and 12 months of treatment.
    - Change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 6 and 12 months of treatment.
    - Clinical Global Impression-Improvement score after 6 and 12 months of treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Change from baseline in the Total Motor Score (TMS) assessment of the Unified Huntington Disease Rating Scale (UHDRS) after 6 and 12 months of treatment.
    - Change from baseline in the Total Maximum Chorea (TMC) score of the UHDRS after 6 and 12 months of treatment.
    - Clinical Global Impression-Improvement score after 6 and 12 months of treatment.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned12
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA28
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Germany
    Poland
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 250
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state80
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 130
    F.4.2.2In the whole clinical trial 260
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    no difference from expected normal treatment
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation CHDI Foundation, Inc.
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-04-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-05-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2017-02-06
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