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    The EU Clinical Trials Register currently displays   44313   clinical trials with a EudraCT protocol, of which   7357   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-003020-32
    Sponsor's Protocol Code Number:A011-16
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2021-10-06
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-003020-32
    A.3Full title of the trial
    Phase 2, Double-blind, Randomized, Placebocontrolled Study to Evaluate the Effects of Sotatercept versus Placebo-Controlled for the Treatment of Combined Postcapillary and Precapillary Pulmonary Hypertension (Cpc-PH) due to Heart Failure with Preserved Ejection Fraction (HFpEF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 Study of Sotatercept for Combined Postcapillary and Precapillary Pulmonary Hypertension Treatment
    A.3.2Name or abbreviated title of the trial where available
    A Phase 2 Study of Sotatercept for Cpc-PH Treatment
    A.4.1Sponsor's protocol code numberA011-16
    A.5.4Other Identifiers
    Name:IND Number:154471
    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 SponsorAcceleron Pharma 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 supportAcceleron Pharma Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcceleron Pharma Inc.
    B.5.2Functional name of contact pointSaraBeth Hahn
    B.5.3 Address:
    B.5.3.1Street Address128 Sidney Street
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number001484767 5150
    B.5.6E-mailshahn@xlrn.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 nameSotatercept
    D.3.2Product code ACE-011
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSOTATERCEPT
    D.3.9.1CAS number 1001080-50-7
    D.3.9.2Current sponsor codeACE-011
    D.3.9.3Other descriptive nameSOTATERCEPT
    D.3.9.4EV Substance CodeSUB179718
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 placeboPowder for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Combined Postcapillary and Precapillary Pulmonary Hypertension (Cpc-PH) due to Heart Failure with Preserved Ejection
    Fraction (HFpEF)
    E.1.1.1Medical condition in easily understood language
    Combined Postcapillary and Precapillary Pulmonary Hypertension due to Heart Failure
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10064911
    E.1.2Term Pulmonary arterial hypertension
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Efficacy: This study is designed to evaluate the efficacy, measured by change from baseline in
    PVR (primary endpoint) and 6MWD (key secondary endpoint) of sotatercept versus placebo in
    adults with Cpc-PH due to HFpEF.
    Safety: The safety and tolerability of sotatercept versus placebo in adults with Cpc-PH due to
    HFpEF will be assessed by the safety endpoints listed below.
    E.2.2Secondary objectives of the trial
    Not Applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants must meet the following criteria to be enrolled in this study:
    1. Age 18 to 85 years
    2. Clinical diagnosis of HFpEF:
    • Left ventricular ejection fraction ≥ 50%, with no history of LVEF below 45%
    3. Demonstrated Cpc-PH by all of the following:
    • Baseline RHC performed within 10 days prior to Visit 1 (during the Screening
    Period) documenting a minimum PVR ≥ 320 dyn•sec/cm5 (4 wood units)
    • Mean pulmonary arterial pressure > 20 mmHg
    • Pulmonary capillary wedge pressure > 15 mmHg but < 30 mmHg
    4. New York Heart Association FC of II or III
    5. Six-Minute Walk Distance ≥ 100 meters repeated twice during Screening and both values
    within 15% of each other, calculated from the highest value
    6. Chronic medication for HF or for any underlying condition, administered at a stable (per
    investigator) dose for ≥ 30 days prior to Visit 1. Diuretics and/or anticoagulants are
    excepted from this rule but should not be newly started or stopped within 30 days of
    Visit 1, and a prescribed dose change should not occur within 7 days of Visit 1.
    Anticoagulation may be suspended for RHC if necessary.
    7. Females of childbearing potential (defined in Appendix 2) must:
    • Have a negative pregnancy test as verified by the investigator prior to starting study
    drug administration; she must agree to ongoing pregnancy testing during the course of
    the study and until 8 weeks after the last dose of the study drug
    • If sexually active, have used, and agree to continue to use highly effective
    contraception without interruption, for at least 28 days prior to starting the
    investigational product, during the study (including dose interruptions), and for
    16 weeks (112 days) after discontinuation of study drug
    • Refrain from breastfeeding a child or donating blood, eggs, or ovum for the duration
    of the study and for at least 16 weeks (112 days) after the last dose of study drug
    See Appendix 2 for additional contraceptive information.
    8. Male participants must:
    • Agree to use a condom, defined as a male latex condom or non-latex condom NOT
    made out of natural (animal) membrane (e.g., polyurethane), during sexual contact
    with a pregnant female or a female of childbearing potential while participating in the
    study, during dose interruptions, and for at least 16 weeks (112 days) following investigational product discontinuation, even if he has undergone a successful
    vasectomy (see Appendix 2 for additional contraceptive information)
    • Refrain from donating blood or sperm for the duration of the study and for 16 weeks
    (112 days) after the last dose of study drug
    9. Ability to adhere to the study visit schedule and understand and comply with all protocol
    requirements
    10. Agreement to not participate in any other trials of investigational drugs/devices while
    enrolled in the A011-16 study
    11. Ability to understand and provide written informed consent for participation
    E.4Principal exclusion criteria
    1. A diagnosis of PH in WHO Group 1, WHO Group 3, WHO Group 4, or WHO Group 5
    2. Significant parenchymal lung disease (e.g., chronic obstructive pulmonary disease with
    Global Initiative for Obstructive Lung Disease (GOLD) criteria III and IV, moderate or
    severe restrictive lung disease, diffuse interstitial fibrosis or alveolitis, pulmonary
    thromboembolism)
    3. Cardiovascular co-morbidities, which include any of the following:
    • Any history of greater than mild mitral regurgitation or aortic regurgitation valvular
    disease or greater than mild aortic or mitral stenosis
    • Acute coronary syndrome, coronary artery bypass graft or percutaneous coronary
    intervention within 180 days of Visit 1
    • Uncontrolled heart rate (> 100 bpm) from atrial fibrillation or atrial flutter
    • History of serious life-threatening or hemodynamically significant arrhythmia
    • History of or anticipated heart transplant or ventricular assist device implantation
    • History of or anticipated implantation of pacemaker or implantable cardioverter
    defibrillator
    • Occurrence of myocardial infarction within 90 days of Visit 1
    • History of known pericardial constriction, hypertrophic cardiomyopathy, sarcoidosis,
    or amyloid cardiomyopathy
    • Uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure
    >1 60 mmHg or sitting diastolic blood pressure > 110 mmHg during Screening after a
    period of rest
    • Systemic hypotension as evidenced by sitting systolic blood pressure < 90 mmHg or
    sitting diastolic blood pressure < 50 mmHg during Screening
    • Resting heart rate of < 45 bpm or > 115 bpm
    • Cerebrovascular accident within 90 days of Visit 1
    • Acutely decompensated HF within 45 days prior to Visit 1, as per investigator
    assessment
    • Electrocardiogram during Screening Period with Fridericia’s corrected QT interval
    (QTcF) > 480 msec or > 500 msec if a ventricular conduction defect (right bundle
    branch block; left bundle branch block; or interventricular conduction delay) is
    present
    • Personal or family history of long corrected QT syndrome or sudden cardiac death in
    first-degree relative
    4. Hospitalization for any indication within 30 days of Visit 1
    5. Received any approved PAH-specific therapies, i.e., endothelin receptor antagonists,
    prostacyclin analogs, phosphodiesterase-5 inhibitors, or a soluble guanylate cyclase
    stimulator, within 30 days prior to Visit 1
    6. Received intravenous inotropes (e.g., dobutamine, dopamine, norepinephrine,
    vasopressin) within 30 days prior to Visit 1
    7. Received erythropoietin within 6 months prior to Visit 1
    8. Known history of chronic liver disease, including untreated hepatitis B and/or hepatitis C
    (with evidence of recent infection and/or active virus replication), defined as mild to
    severe hepatic impairment (Child-Pugh Class A to C)
    9. Prior exposure to sotatercept or luspatercept
    10. Currently enrolled in or have completed any other investigational product study within
    30 days for small-molecule drugs or within 5 half-lives for biologics prior to the date of
    signed informed consent
    11. Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior
    to Visit 1 or planned initiation during the study (participants who are stable in the
    maintenance phase of a program and who will continue for the duration of the study are
    eligible)
    12. Any of the following clinical laboratory values prior to Visit 1 as specified:
    • Hemoglobin above the gender-specific upper limit of normal (ULN) per local
    laboratory test within 28 days of Visit 1 or < 10 g/dL per local laboratory within
    28 days of Visit 1
    • Serum alanine aminotransferase or aspartate aminotransferase levels > 3× ULN or
    total bilirubin > 1.5× ULN within 28 days of Visit 1
    • Estimated glomerular filtration rate < 30 ml/min/1.73 m2
    (4-variable Modification of
    Diet in Renal Disease equation) within 28 days of Visit 1 or required renal
    replacement therapy within 90 days of Visit 1
    • Glycated hemoglobin (HbA1c) > 10% within 28 days of Visit 1
    • Platelet count < 75,000/mm3 within 28 days of Visit 1
    13. History of severe allergic or anaphylactic reaction or hypersensitivity to recombinant
    proteins or excipients in the investigational product
    14. Major surgery within 60 days prior to Visit 1. Participants must have completely
    recovered from any previous surgery prior to Visit 1
    15. Prior heart-lung transplants or life expectancy of < 12 months
    16. Pregnant or breastfeeding females
    17. History of active malignancy, with the exception of fully excised or treated basal cell
    carcinoma, cervical carcinoma in-situ, or ≤ 2 squamous cell carcinomas of the skin
    18. History of clinically significant (as determined by the investigator) endocrine,
    hematologic, hepatic, (auto)immune, metabolic, urologic, pulmonary, neurologic,
    neuromuscular, dermatologic, psychiatric, renal, and/or another disease that may limit
    participation in the study.
    19. Body mass index ≥ 45 kg/m2
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy Endpoints
    Primary Efficacy Endpoint
    • Change in PVR at 24 weeks from baseline
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks from baseline
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoint
    • Change in 6MWD at 24 weeks from baseline
    Secondary Endpoints
    • Number of Clinical Worsening events at 24 weeks and 48 weeks. Clinical Worsening
    events are defined as any of the following:
    − Hospitalization due to a cardiopulmonary indication (a non-elective
    hospitalization lasting at least 24 hours in duration caused by clinical conditions
    directly related to PH and/or HF)
    − Administration of intravenous diuretics
    − Death (all causes)
    − Decrease in 6MWD by ≥ 15% from baseline confirmed by 2 tests at least 4 hours,
    but no more than 1 week, apart
    • Number of participants with first Clinical Worsening event, defined as above, at
    24 and 48 weeks
    • Time to Clinical Worsening, defined as above
    • Change in dyspnea score (assessed by Borg CR10 scale®) at Week 24 from baseline
    • Change in hemodynamic and echocardiography (ECHO) parameters, including but
    not limited to PVR, mean pulmonary arterial pressure (mPAP), pulmonary capillary
    wedge pressure (PCWP), TAPSE, right ventricular fractional area change (RVFAC),
    LVEF, IVRT, E/e' ratio, and ratio of the peak velocity flow of the E wave in early
    diastole to peak velocity flow of the A wave in late diastole (E/A ratio) at 24 weeks
    from baseline
    • Change in NT-proBNP at 24 weeks from baseline
    • Change in NYHA FC at 24 weeks from baseline
    • Change in 6MWD at 48 weeks from baseline
    • Change in hemodynamic and ECHO parameters, including but not limited to PVR, ,
    mPAP, PCWP, TAPSE, RVFAC, LVEF, IVRT, E/e' ratio, and E/A ratio at 48 weeks
    from baseline
    • Change in NT-proBNP at 48 weeks from baseline
    • Change in NYHA FC at 48 weeks from baseline
    • Change in PVR, 6MWD, and NYHA FC at Week 48 from baseline in the Extension
    Period in the placebo-crossed Treatment Group
    • Change in PVR, 6MWD, and NYHA FC from Week 24 to Week 48 in the Extension
    Period in the placebo-crossed Treatment Group
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 and 48 weeks accordingly
    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 No
    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 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 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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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
    Israel
    United States
    Belgium
    France
    Germany
    Italy
    Netherlands
    Spain
    Sweden
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    LPLV
    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 months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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: 45
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 135
    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 state14
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 180
    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)
    None
    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 Decision2021-12-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-06
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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