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    Summary
    EudraCT Number:2017-004310-25
    Sponsor's Protocol Code Number:ARRAY-797-301
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-05-28
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2017-004310-25
    A.3Full title of the trial
    A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 (PF-07265803) in Patients with Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
    A.4.1Sponsor's protocol code numberARRAY-797-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03439514
    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 SponsorArray BioPharma Inc. (a wholly owned subsidiary of Pfizer Inc.)
    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 supportArray Biopharma Inc.
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportPfizer Inc.
    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 Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post code10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+18007181021
    B.5.6E-mailClinicalTrials.gov_Inquiries@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 nameARRY-371797
    D.3.2Product code PF-07265803
    D.3.4Pharmaceutical form Film-coated 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.3.9.1CAS number 765914-60-1
    D.3.9.2Current sponsor codeARRY-371797
    D.3.9.3Other descriptive namePF-07265803
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboFilm-coated tablet
    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
    Dilated Cardiomyopathy (DCM) with Lamin A/C protein (LMNA) Mutation
    E.1.1.1Medical condition in easily understood language
    Dilated Cardiomyopathy
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10056419
    E.1.2Term Dilated cardiomyopathy
    E.1.2System Organ Class 100000004849
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    NYHA Class II/III patients only: Evaluate the effect of ARRY-371797 (PF-07265803) on functional capacity (as measured by the
    6-minute walk test [6MWT]) compared to placebo
    E.2.2Secondary objectives of the trial
    - Evaluate additional measures of efficacy of ARRY-371797 (PF-07265803) compared to placebo in the randomized period.
    - Evaluate the impact of ARRY-371797 (PF-07265803) on composite of all-cause mortality, or worsening heart failure (WHF).
    - Evaluate the impact of ARRY-371797 (PF-07265803) on overall survival (OS).
    - Evaluate the safety of ARRY-371797 (PF-07265803) compared to placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Selected Key Inclusion Criteria:
    • Provide a signed and dated informed consent document prior to initiation of any study-related procedures. Patients under guardianship or partial guardianship will be eligible unless prohibited by local laws or by local/central ethic committees
    • Age ≥ 18 years at time of informed consent
    • Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class II/III/ or Class IV defined as:
    a. Gene positive for a pathogenic, likely pathogenic or VUS mutation in the LMNA gene as determined by an accredited clinical laboratory
    b. NYHA functional Class II or III that has been stable for at least 3 months
    c. Evidence of cardiac impairment as determined by LVEF≤ 50%
    • Patient will have an implantable cardioverter defibrillator/cardiac resynchronization therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation of study treatment or CRT-D initiated at least 6 months prior to initiation of study intervention and defibrillation function activated at least 4 weeks prior to initiation of study intervention. Devices must have activated pacing capabilities or a separate pacemaker must be present.
    • Class II/III patients must have objective functional impairment evidenced by a reduction in 6-minute walk test (6MWT);
    • Stable medical and/or device therapy consistent with American Heart Association (AHA) / American College of Cardiology (ACC) or European Society of Cardiology (ESC) guidelines
    • Patients must meet acceptable hematology, hepatic and renal laboratory values as specified within 35 days prior Day 1

    Please refer to Protocol, section 5.1 for detailed list of Inclusion Criteria.



    E.4Principal exclusion criteria
    Selected Key Exclusion Criteria:
    • Presence of other form(s) of cardiomyopathy contributing to HF (e.g., inflammatory or infiltrative cardiomyopathy) or clinically significant cardiac anatomic abnormality (e.g., LV aneurysm), clinically significant coronary artery disease (e.g., coronary revascularization, exercise-induced angina) or uncorrected, hemodynamically significant (i.e., moderate-severe) primary structural valvular disease not due to HR, per Investigator judgment.
    • Currently receiving or deemed at high risk of requiring chronic renal replacement therapy (e.g., hemodialysis or peritoneal dialysis) within 6 months.
    • Treatment with any investigational agent(s) for HF within 35 days prior to Day 1.
    • Malignancy that is active or has been diagnosed within 3 years prior to screening, except surgically curatively resected in situ malignancies or surgically cured early breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell carcinoma) thyroid cancer or cervical cancer, or, with prior review by the Medical Monitor, other earlystage surgically curatively resected malignancies with less than a 20% expected 2-year recurrence rate.
    • Non-cardiac condition that limits lifespan to < 1 year.
    • Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human immunodeficiency virus (HIV) at screening.
    • Pregnancy or breastfeeding, or patients who plan to become pregnant during the duration of the trial
    • Patients with an underlying condition that may impact the ability of the 6MWT to reflect changes in cardiovascular function such as: an orthopedic condition that limits walking abilities (e.g., severe arthritis), significant musculoskeletal pathology, significant chronic obstructive pulmonary disease (COPD) that limits exercise tolerance or any other condition that according to the Investigator's assessment significantly limits a patient's performance on the 6MWT independently from the patient's cardiomyopathy.
    • Documented hypersensitivity/allergy or clinically significant intolerance to any component of drug product.

    Please refer to Protocol, section 5.2 for detailed list of Exclusion Criteria.
    E.5 End points
    E.5.1Primary end point(s)
    NYHA Class II/III patients only: Change from baseline in 6MWT at Week 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 24
    E.5.2Secondary end point(s)
    1. NYHA Class II/III patients only: Change from baseline in 6MWT at Weeks 4 and 12
    2. NYHA Class II/III patients only: Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ) Physical Limitation (PL) and Total Symptom Score (TSS) domains at Weeks 12 and 24
    3. NYHA Class II/III patients only: Change from baseline in Patient Global Impression (PGI) scores at Weeks 12 and 24
    - Patient Global Impression of Severity (PGI-S)
    - Patient Global Impression of Change (PGI-C)
    4. NYHA Class II/III patients only: Change from baseline in N-terminal pro-brain natriuretic peptide (NT-proBNP) at Weeks 4, 12 and 24
    5. HFS: defined as the time from randomization to the first occurence of any event in the composite of death due to any cause, or worsening heart failure (HF-related hospitalization or HF-related urgent care visit).
    6. OS
    8. Safety as determined by:
    - Incidence and severity of adverse events (AEs)
    - Changes in clinical safety laboratory tests, vital signs and 12-lead electrocardiograms (ECGs)
    - Incidence and severity of ventricular or atrial arrhythmias detected using existing implantable cardioverter defibrillator (ICD)/cardiac resynchronization therapy defibrillator (CRT-D) or
    other applicable device interrogations at Weeks 12 and 24,
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Weeks 4 and 12
    2. Weeks 12 and 24
    3. Weeks 12 and 24
    4. Weeks 4, 12 and 24
    5. Thoughout the duration of the study
    7. Throughout the duration of the study
    8. Safety endpoints throughout the duration of the study
    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 Yes
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Randomized, double-blind treatment period followed by open-label treatment period
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Mexico
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    Norway
    Spain
    United Kingdom
    Argentina
    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
    The end of the study is reached when all patients enrolled into the randomized portion of the study have had the opportunity to be followed for at least 24 weeks in the open-label or have discontinued from the study, whichever comes first
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    E.8.9.2In all countries concerned by the trial months0
    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: 140
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 123
    F.4.2.2In the whole clinical trial 160
    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)
    Patients who remain on treatment at the end of the study and who may, in the opinion of the Investigator, derive benefit from continued treatment with ARRY-371797 will be provided the opportunity to continue treatment with ARRY-371797 through a protocol amendment or under a separate rollover protocol. Any such continued treatment will, where required by local regulations, occur only after Health Authority approval and Ethics Committee approval.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2018-05-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-08
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2022-08-02
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