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    Summary
    EudraCT Number:2017-000322-35
    Sponsor's Protocol Code Number:ACE-536-MF-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-06-28
    Trial 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 ConcernedUK - MHRA
    A.2EudraCT number2017-000322-35
    A.3Full title of the trial
    A Phase 2, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Luspatercept (ACE-536) in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis and Anemia With and Without Red Blood Cell-Transfusion Dependence
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate the benefit and safety of Luspatercept (ACE-536) in adults with myeloproliferative neoplasm-associated myelofibrosis and anemia with and without red blood cell-transfusion dependence
    A.4.1Sponsor's protocol code numberACE-536-MF-001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03194542
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1197-1202
    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 SponsorCelgene Corporation
    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 supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCelgene Corporation
    B.5.2Functional name of contact pointClinical Trial Disclosure
    B.5.3 Address:
    B.5.3.1Street Address86 Morris Avenue
    B.5.3.2Town/ citySummit, New Jersey
    B.5.3.3Post code07901
    B.5.3.4CountryUnited States
    B.5.4Telephone number+19137096862
    B.5.6E-mailClinicalTrialDisclosure@bms.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 Yes
    D.2.1.1.1Trade name Reblozyl
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameLuspatercept
    D.3.2Product code ACE-536
    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 INNLuspatercept
    D.3.9.1CAS number 1373715-00-4
    D.3.9.2Current sponsor codeACE-536
    D.3.9.3Other descriptive nameLUSPATERCEPT
    D.3.9.4EV Substance CodeSUB179621
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number37.5
    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 Yes
    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.IMP: 2
    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 Yes
    D.2.1.1.1Trade name Reblozyl
    D.2.1.1.2Name of the Marketing Authorisation holderCelgene Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameLuspatercept
    D.3.2Product code ACE-536
    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 INNLuspatercept
    D.3.9.1CAS number 1373715-00-4
    D.3.9.2Current sponsor codeACE-536
    D.3.9.3Other descriptive nameLUSPATERCEPT
    D.3.9.4EV Substance CodeSUB179621
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number87.5
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Anemia associated with myeloproliferative neoplasm (MPN)-associated
    myelofibrosis
    E.1.1.1Medical condition in easily understood language
    Anemia due to a bone marrow disorder resulting in abnormal production of blood cells
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077161
    E.1.2Term Primary myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074689
    E.1.2Term Post polycythemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074690
    E.1.2Term Post essential thrombocythemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074692
    E.1.2Term Post essential thrombocythaemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10074691
    E.1.2Term Post polycythaemia vera myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10028537
    E.1.2Term Myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy and safety of luspatercept for the treatment of anemia in subjects
    with MPN-associated myelofibrosis with and without RBC-transfusion dependence.
    E.2.2Secondary objectives of the trial
    - To evaluate the safety of luspatercept in MPN-associated myelofibrosis
    - To evaluate the effect of luspatercept in MPN-associated myelofibrosis:
    -- on the time to and duration of anemia response
    -- on frequency of RBC transfusions and transfusion dependence
    -- on symptom response improvement via the Myeloproliferative Neoplasms Symptom Assessment Form (Total Symptom Score (MPN-SAF TSS)
    -- on health-related quality of life (HRQoL) via the EQ-5D-5L and Functional Assessment of Cancer Therapy – Anemia (FACT-An) questionnaires
    --on the changes in hemoglobin and mean hemoglobin increase
    -- To evaluate population pharmacokinetics of luspatercept in subjects with MPN-associated myelofibrosis with and without RBC-transfusion dependence
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject is ≥18 years of age at the time of signing the informed consent form (ICF).
    2. Subject has MPN-associated myelofibrosis (PMF, post-PV MF, and/or post-ET MF) as confirmed from the most recent local bone marrow biopsy report according to the World Health organization 2016 criteria (Arber, 2016).
    3. Subject has anemia defined as:
    a. Cohorts 1 and 3A
    i. Obtain ≥ 3 Hgb levels of ≤ 9.5 g/dL recorded on ≥ 3 different days, including the day of dosing, in the 84-day period immediately up to the C1D1 date. There must be ≥ 14 days in between each Hgb measurement. No subjects with an interval ≥ 42 days between hemoglobin measurements will be enrolled.
    ii. There must not be any RBC transfusions within the 84-day period immediately up to the C1D1 date.
    b. Cohorts 2 and 3B
    i. Average RBC-transfusion frequency: 4 to 12 RBC units/84 days immediately up to the C1D1 date. There must be no interval > 56 days without ≥ 1 RBC transfusion.
    ii. Subjects must have a Hgb value of < 13 g/dL on C1D1 prior to luspatercept administration.
    iii. Only RBC transfusions given when the Hgb ≤ 9.5 g/dL are scored in determining eligibility.
    iv. RBC transfusions given because of bleeding, infection, or chemotherapy-induced anemia are not scored in determining eligibility.
    4. Subject has an Eastern Cooperative Oncology Group (ECOG) performance score of ≤ 2.
    5. Subject is not anticipated during the 6 months from the C1D1 date to receive a hematopoietic cell transplant.
    6. A female of childbearing potential (FCBP) for this study is defined as a female who: 1)
    has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). FCBP participating in the study must:
    a. Have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact.
    b. Either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, effective contraception without interruption, 28 days prior to starting investigational product, during the study therapy (including dose interruptions), and for 12 weeks (approximately 5 times the mean terminal halflife of luspatercept based on multiple-dose pharmacokinetics [PK] data) after discontinuation of study therapy.
    7. Male subjects must:
    a. Practice true abstinence (which must be reviewed on a monthly basis) or agree to use a condom 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 12 weeks (approximately 5 times the mean terminal half-life of luspatercept based on multiple-dose PK data) following investigational product discontinuation, even if he has undergone a successful vasectomy
    8. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
    9. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
    E.4Principal exclusion criteria
    1. Subject (sbj) use of hydroxyurea or other drugs with potential effects on hematopoiesis (refer to Section 8.2 for prohibited medications) or ongoing AEs from previous treatment ≤ 112 days immediately up to the enrollment date
    a. Systemic corticosteroids are permitted for nonhematological conditions providing the sbjt is receiving a stable or decreasing dose for ≥ 84 days immediately up to enrollment and is receiving a constant dose equivalent to ≤ 10 mg prednisone for the 28 days immediately up to enrollment
    2. Cohort 1 and 2 only: sbjs treated with JAK2 inhibitors ≤ 112 days immediately up to the enrollment date or if anticipated/substantial likelihood for sbj to receive ruxolitinib within the first 168 days on the study
    3. Cohort 3 only: subjects not receiving ruxolitinib: a.for at least 280 days (40 weeks) without interruptions exceeding 2 consecutive weeks; b. on a stable daily dose for at least 112 days (16 weeks) immediately up to the enrollment date as part of their standard-of-care therapy;
    4. Sbj use of ESAs or androgenic steroids ≤ 112 days immediately up to the enrollment date
    5. Starting iron chelation therapy (ICT) or changing the ICT dose within ≤ 112 days up to the enrollment date
    6. Sbj with anemia from iron deficiency, B12 and folate deficiencies, hemolytic anemia, infection, or bleeding
    7. Pregnant or breastfeeding females
    8. Sbj with blood myeloblasts ≥ 5%
    9. Sbj with major surgery within 8 weeks up to the enrollment date. Sbj must have completely recovered from any previous surgery immediately up to the enrollment date
    10. Sbj with prior history of malignancies, other than disease under study, unless the sbj has been free of the disease for ≥ 5 years. However, sbj with the following history/concurrent conditions is allowed: - Basal or squamous cell carcinoma of the skin;- Carcinoma in situ of the cervix;- Carcinoma in situ of the breast;- Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)
    11. Sbj with prior therapy of luspatercept or sotatercept
    12. Sbj participation in any other clinical protocol or investigational trial that involves administration of experimental therapy and/or therapeutic devices within 30 days immediately up to the enrollment date
    13. Sbj with prior hematopoietic cell transplant
    14. Sbj with any of the following lab. abnormalities at screening: - Neutrophils < 1 x 10^9/L;- White blood count (WBC) > 100 x 10^9/L;
    - Platelets
    i. Cohorts 1 and 2: < 25 x 10^9/L
    ii. Cohort 3A and 3B: < 50 x 10^9/L
    iii. All Cohorts: > 1000 x 10^9/L
    - Estimated glomerular filtration rate < 45 mL/min/1.73 m2 (via the 4-variable modification of diet in renal disease MDRD formula)
    - Aspartate aminotransferase (AST) or alanine transaminase (ALT) > 3.0 x upper limit of normal (ULN)
    - Direct bilirubin ≥ 2 x ULN
    i.higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (ie, ineffective erythropoiesis)
    - Uncontrolled hyperthyroidism or hypothyroidism
    15. Sbj with stroke, deep venous thrombosis, pulmonary or arterial embolism within 6 months immediately up to the enrollment date.
    16. Sbj with diastolic blood pressure ≥ 90 mmHg or systolic blood pressure ≥ 140 mHg measured during the Screening Period despite appropriate treatment.
    17. Sbj with inadequately controlled heart disease and/or have a known left ventricular ejection fraction <35%.
    18. Sbj with history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product (see IB).
    19. Sbj with uncontrolled systemic fungal, bacterial, or viral infection (ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
    20. Sbj with human immunodeficiency virus (HIV), evidence of active infectious Hepatitis B (HepB), and/or evidence of active HepC.
    21. Sbj with any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations that would prevent the subject from participating in the study.
    22. Sbj with any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
    23. Sbj with any condition or concomitant medication that confounds the ability to interpret data from the study.
    24. Subject on anticoagulant therapy not under appropriate control or subject not on a stable dose of anticoagulant therapy for ≥ 8 weeks up to the enrollment date.
    25. Subject on anagrelide within 28 days immediately up to the enrollment date
    26. Subject with a major bleeding event (defined as symptomatic bleeding in a critical area or organ and/or bleeding causing a decrease in Hgb of ≥ 2 g/dL or leading to transfusion of ≥ 2 units of packed red cells) in the last 6 months prior to enrollment.
    E.5 End points
    E.5.1Primary end point(s)
    1) Anemia response as it relates to hemoglobin (Hgb) increase
    2) Anemia response as it relates to increased red blood cell (RBC) – transfusion independence
    E.5.1.1Timepoint(s) of evaluation of this end point
    1.) Cohorts 1 and 3A: Any consecutive “rolling” 84-day period from Day 1 through
    and including Day 168

    2.) Cohorts 2 and 3B: Any consecutive “rolling” 84-day period from Day 1 through
    and including Day 168
    E.5.2Secondary end point(s)
    1. Time to anemia response
    2. Duration of anemia response
    3. Frequency of RBC transfusions
    4. Frequency of RBC-transfusion dependence
    5. Proportion of subjects who achieve ≥ 50% reduction in fatigue symptom as measured by the Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
    6. The proportion of subjects who achieve ≥ 50% reduction in total symptom score (TSS)
    7. Health-related quality of life (HRQoL)
    8. Functional Assessment of Cancer Therapy – Anemia (FACTAn)
    9. EQ-5D-5L questionnaires
    10. Adverse Events (AEs)
    11. Antidrug antibodies (ADA)
    12. Pharmacokinetic - AUC
    13. Pharmacokinetic - Cmax
    14. Changes in hemoglobin in the absence of RBC transfusions (Cohorts 1 and 3A)
    15 Mean hemoglobin increase
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. from Day 1 through and including Day 168
    2. Day 1 through end of treatment.
    3, 4, 5, 6, 7, 8, 9, 14 Day 1 through and including Day 168 and Day 1 through end of treatment
    10. Screening through 42 days post last dose
    11. Day 1 up to 1 year for ADA-negative subjects; Day 1 up to 2 years for ADA-positive subjects
    12, 13. Up to 1 year
    15. Any consecutive “rolling” 84-day period from Day 1 through and including Day 168; Day 1 through end 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 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 Yes
    E.6.13.1Other scope of the trial description
    Biomarkers
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    France
    Italy
    United Kingdom
    United States
    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
    The End of Trial is defined as either the date of the last visit of the last subject to complete the Posttreatment Follow-up Period, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date.
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years6
    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: 17
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 86
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 47
    F.4.2.2In the whole clinical trial 103
    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)
    Subjects will be treated according to normal SOC. The Sponsor may end the trial when key endpoints and objectives have been analyzed. An Extension Phase of Treatment Period up to 2 years or more from the first dose is available if the subject continues to receive clinical benefit. A roll-over protocol also exists into which subjects have access to luspatercept and/or complete posttreatment follow up. Such protocol would only be written for a compound that would not yet be commercially available
    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 Decision2017-07-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-08-22
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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    The status of studies in GB is no longer updated from 1.1.2021
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