An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia

Sponsors

Lead Sponsor: Celgene

Collaborator: Acceleron Pharma, Inc.

Source Celgene
Brief Summary

This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia.

The study is divided into the following periods:

- Historical Period,

- Screening/Run-in Period,

- Double-blind Treatment Period (48 weeks),

- Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks),

- Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation

- Post-treatment Follow-up Period

Overall Status Active, not recruiting
Start Date May 2, 2016
Completion Date June 7, 2025
Primary Completion Date November 24, 2017
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Percentage of Participants Who Achieved Erythroid Response From Week 13 to Week 24 (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24
Secondary Outcome
Measure Time Frame
Percentage Of Participants Who Achieve >=33% Reduction From Baseline in Transfusion Burden From Week 37 to Week 48 (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48
Percentage Of Participants Who Achieve >=50% Reduction From Baseline in Transfusion Burden From Week 13 to Week 24 (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24
Percentage Of Participants Who Achieve >=50% Reduction From Baseline in Transfusion Burden From Week 37 to Week 48 (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48
Baseline Values and Mean Change From Baseline in Transfusion Burden (RBC Units) to the Fixed Week 13 to Week 24 Interval (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24
Baseline Values and Mean Change From Baseline At Week 48 In Derived Liver Iron Concentration (LIC) By Magnetic Resonance Imaging (MRI) (Data Cut-off Date: 11 May 2018) Baseline: Week -12 to Day -1; Treatment: Week 48
Baseline Values and Mean Change From Baseline At Week 48 In Mean Daily Dose Of Iron Chelation Therapies (ICT) Deferasirox, Deferiprone and Deferoxamine Mesilate/Deferoxamine (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48
Baseline Values and Mean Change From Baseline At Week 48 In Mean Serum Ferritin (Data Cut-off Date: 11 May 2018) Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48
Baseline Values and Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48 By Dual Energy X-Ray Absorptiometry (DXA) (Data Cut-off Date: 11 May 2018) Baseline: Day 1; Treatment: Week 48
Baseline Values and Mean Change From Baseline In Myocardial Iron By T2* Magnetic Resonance Imaging (MRI) at Week 48 (Data Cut-off Date: 11 May 2018) Baseline: Day 1; Treatment: Week 48
Baseline Values and Mean Change From Baseline in the Transfusion-dependent Quality of Life (TranQol) Questionnaire Total Score at Weeks 24 and 48 Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 and 48
Baseline Values and Mean Change From Baseline in the Transfusion-dependent Quality of Life (TranQol) Questionnaire Physical Health Domain at Weeks 24 and 48 (Data Cut-off Date: 11 May 2018) Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 and 48
Baseline Values and Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire Physical Functioning Domain at Weeks 24 and 48 (Data Cut-off Date: 11 May 2018) Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 and 48
Baseline Values and Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire General Health Domain at Weeks 24 and 48 (Data Cut-off Date: 11 May 2018) Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 and 48
Baseline Values and Mean Change From Baseline in the 36-item Short Form Health Survey (SF-36) Questionnaire Physical Component Summary at Weeks 24 and 48 (Data Cut-off Date: 11 May 2018) Baseline: 4 weeks prior to Day 1; Treatment: Weeks 24 and 48
Percentage of Participants Who Utilized Healthcare Resources During Treatment [Healthcare Resource Utilization (HRU)] (Data Cut-off Date: 11 May 2018) HRU data is collected continuously from Week-12 (informed consent) up to 9 weeks post last dose. Up to data cutoff date of 11 May 2018 (48 weeks post last participant enrolled day 1), median exposure is 64.1 weeks (Q1,Q3:52.5,70.0; Min, Max: 3,97)
Number of Days in Higher Care Hospital Units; [Healthcare Resource Utilization (HRU)] (Data Cut-off Date: 11 May 2018) HRU data is collected continuously from Week-12 (informed consent) up to 9 weeks post last dose. Up to data cutoff date of 11 May 2018 (48 weeks post last participant enrolled day 1), median exposure is 64.1 weeks (Q1,Q3:52.5,70.0; Min, Max: 3,97)
Percentage Of Participants Who Were Transfusion Independent For ≥ 8 Weeks During Treatment (Data Cut-off Date: 11 May 2018) Transfusion data is collected continuously at week (wk) -24 (12 weeks pre ICF) up to 9 wks post last dose. Efficacy cutoff = defined as: death, study discontinuation, last dose+20, 11 May 18; median exposure is 64.1 wks (Q1, Q3: 52.5,70.0; Min, Max: 3,97)
Mean Duration of Reduction in Transfusion Burden In Participants With a >= 33% Reduction and >=50% Reduction in Red Blood Cell (RBC) Transfusion Burden During Any Rolling 12-week Interval Up to the Efficacy Cutoff Date (11 May 2018) Transfusion data is collected continuously at week (wk) -24 (12 weeks pre ICF) up to 9 wks post last dose. Efficacy cutoff = defined as: death, study discontinuation, last dose+20, 11 May 18; median exposure is 64.1 wks (Q1, Q3: 52.5,70.0; Min, Max: 3,97)
Kaplan-Meier Estimates for Duration of Transfusion Independence in Participants Who Were Transfusion Independent For ≥ 8 Weeks (Data Cut-off Date: 11 May 2018) Transfusion data is collected continuously at week (wk) -24 (12 weeks pre ICF) up to 9 wks post last dose. Efficacy cutoff = defined as: death, study discontinuation, last dose+20, 11 May 18; median exposure is 64.1 wks (Q1, Q3: 52.5,70.0; Min, Max: 3,97)
Time to Erythroid Response in Participants With ≥ 33% Reduction and ≥ 50% Reduction in RBC Transfusion Burden (Data Cut-off Date: 11 May 2018) Transfusion data is collected continuously at week (wk) -24 (12 weeks pre ICF) up to 9 wks post last dose. Efficacy cutoff = defined as: death, study discontinuation, last dose+20, 11 May 18; median exposure is 64.1 wks (Q1, Q3: 52.5,70.0; Min, Max: 3,97)
Post-Baseline Transfusion Event Frequency (Data Cut-off Date: 11 May 2018) Transfusion data is collected continuously at week (wk) -24 (12 weeks pre ICF) up to 9 wks post last dose. Efficacy cutoff = defined as: death, study discontinuation, last dose+20, 11 May 18; median exposure is 64.1 wks (Q1, Q3: 52.5,70.0; Min, Max: 3,97)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Clearance (CL/F) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Apparent Volume of Distribution of the Central Compartment (V1/F) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Elimination Half-life (t1/2) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Time to Reach Maximum Concentration (Tmax) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration for the Starting Dose (Cmax) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Maximum Concentration at Steady State for the Starting Dose (Cmax,ss) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Pharmacokinetic (PK) Parameters: Bayesian Estimate of Area Under the Concentration-Time Curve at Steady State for the Starting Dose (AUCss) (Data Cut-off Date: 11 May 2018) Blood serum samples taken pre-dose on Days 1, 22, 64, 85, 106, 127, 169, 211, 253, 295, 337. Blood serum samples also taken on Days 135 and 142 (Days 8 + 15 after Dose 6)
Participants With Treatment-Emergent Adverse Events (TEAE) (Data Cut-off Date: 11 May 2018) Day 1 up to 97 weeks (maximum treatment as of data cut-off date)
Participants With Pre-Existing and/or Treatment-Emergent Antidrug Antibodies (ADA) (Data Cut-off Date: 11 May 2018) Timeframe: predose Day 1, Days 22, 64, 106, 148, 232, 316
Enrollment 336
Condition
Intervention

Intervention Type: Drug

Intervention Name: Luspatercept

Description: Subjects will start with luspatercept at 1 mg/kg dose level.

Arm Group Label: Luspatercept (ACE-536) plus Best Supportive Care (BSC)

Other Name: ACE-536

Intervention Type: Other

Intervention Name: Placebo

Description: Placebo, Subcutaneous, every 21 days.

Arm Group Label: Placebo plus Best Supportive Care (BSC)

Eligibility

Criteria:

Inclusion Criteria:

Subjects must satisfy the following criteria to be enrolled in the study:

1. Male or female, ≥18 years of age at the time of signing the informed consent document (ICF).

2. Subject must understand and voluntarily sign an Inform Consent Form prior to any study-related assessments/procedures being conducted.

3. Subject is willing and able to adhere to the study visit schedule and other protocol requirements.

4. Documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia. (β-thalassemia with mutation and/or multiplication of alpha globin is allowed).

5. Regularly transfused, defined as: 6-20 Red Blood Cell (RBC) units* in the 24 weeks prior to randomization and no transfusion-free period for ≥ 35 days during that period.

* Sites who prescribe transfusions and have the transfusion records only in volumes should use for conversion of volume to units the below criteria, in order to obtain number of units within the last 24 weeks to assess the eligibility: 1 unit in this protocol refers to a quantity of packed RBCs approximately 200-350 mL. (i) sites who use transfusion bags within this range, or ≥ 350 mL, the conversion in units should be done by dividing the volume transfused to the patient by 350 mL, (ii) sites who use transfusion bags < 200 mL, the conversion in units should be done by dividing the volume transfused to the patient by 200 mL.

6. Performance status: Eastern Cooperative Oncology Group (ECOG) score of 0 or 1.

7. 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:

1. Have two 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.

2. Either commit to true abstinence** from heterosexual contact (which must be reviewed on a monthly basis and source documented) If a FCBP engages in sexual activity that may result in a pregnancy, she must 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 five times the mean terminal half-life of luspatercept based on multiple-dose Pharmacokinetic PK) data) after discontinuation of study therapy.

- True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.] *** Agreement to use highly effective methods of contraception that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly throughout the course of the study.

Such methods include: Combined (estrogen and progesterone/progestin containing) hormonal contraception: Oral; Intravaginal; Transdermal; Progestogen/progestin only hormonal contraception associated with inhibition of ovulation: Oral; Injectable hormonal contraception; Implantable hormonal contraception; Placement of an intrauterine device (IUD); Placement of an intrauterine hormone-releasing system (IUS); Bilateral tubal occlusion; Vasectomized partner; Sexual Abstinence.

8. Male subjects must:

- Practice true abstinence 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 five 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.

Exclusion Criteria:

The presence of any of the following will exclude a subject from enrollment:

1. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.

2. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.

3. Any condition that confounds the ability to interpret data from the study.

4. A diagnosis of Hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H);

5. Evidence of active hepatitis C (HCV) infection as demonstrated by a positive HCV-RNA test of sufficient sensitivity, or active infectious hepatitis B as demonstrated by the presence of HBsAg and/or HBVDNA-positive,, or known positive human immunodeficiency virus (HIV).

Note: Subjects receiving antiviral therapies should have 2 negative HCVRNA tests 3 months apart.(ie, one test at the end of the antiviral therapy and a second test 3 months following the first test).

6. Deep Vein Thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to randomization.

7. Use of chronic anticoagulant therapy is excluded, unless the treatment stopped at least 28 days prior to randomization. Anticoagulant therapies used for prophylaxis for surgery or high risk procedures as well as low Molecular Weight (LMW) heparin for superficial venous thrombosis and chronic aspirin are allowed.

8. Platelet count > 1000 x 109/L

9. Poorly controlled diabetes mellitus within 24 weeks prior to randomization as defined by short term (eg, hyperosmolar or ketoacidotic crisis) and/or history of diabetic cardiovascular complications (eg, stroke or myocardial infarction).

10. Treatment with another investigational drug or device ≤ 28 days prior to randomization.

11. Prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).

12. Use of an erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to randomization.

13. Iron chelation therapy, if initiated ≤ 24 weeks prior to randomization (allowed if initiated > 24 weeks before or during treatment).

14. Hydroxyurea treatment ≤ 24 weeks prior to randomization.

15. Pregnant or lactating females.

16. Uncontrolled hypertension. Controlled hypertension for this protocol is considered ≤ Grade 1 according to NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (current active minor version).

17. Major organ damage, including:

1. Liver disease with alanine aminotransferase (ALT) > 3 x the upper limit of normal (ULN) or history of evidence of cirrhosis;

2. Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.

3. Lung disease, including pulmonary fibrosis or pulmonary hypertension which are clinically significant ie, ≥ Grade 3 NCI CTCAE version 4.0 (current active minor version).

4. Creatinine clearance < 60 mL/min (per Cockroft-Gault formula).

18. Proteinuria ≥ Grade 3 according to NCI CTCAE version 4.0 (current active minor version).

19. Chronic systemic glucocorticoids ≤ 12 weeks prior to randomization (physiologic replacement therapy for adrenal insufficiency is allowed). Single day glucocorticoid treatment (eg, for prevention or treatment of transfusion reactions, is allowed).

20. Major surgery ≤ 12 weeks prior to randomization (subjects must have completely recovered from any previous surgery prior to randomization).

21. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product (see Investigator Brochure).

22. Cytotoxic agents, immunosuppressants ≤ 28 days prior to randomization (ie, antithymocite globulin (ATG) or cyclosporine)

23. History of malignancy with the exception of:

1. Curatively resected nonmelanoma skin cancer.

2. Curatively treated cervical carcinoma in situ.

3. Other solid tumor with no known active disease in the opinion of the investigator.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Jeevan Shetty, MBCh.B , FRCP Study Director Celgene Corporation
Location
Facility:
Children's Hospital of Los Angeles | Los Angeles, California, 90027, United States
Children's Hospital and Research Center at Oakland | Oakland, California, 94609, United States
Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago, Illinois, 60611, United States
Boston Children's Hospital | Boston, Massachusetts, 02115, United States
Weill Cornell Medical College | New York, New York, 10065, United States
Hospital of the University of Pennsylvania | Philadelphia, Pennsylvania, 19104, United States
Prince of Wales Hospital | Randwick, New South Wales, 2031, Australia
Mater Adult Hospital | South Brisbane, Queensland, 4101, Australia
Royal Adelaide Hospital Institute of Medical and Veterinary Science | Adelaide, South Australia, 5000, Australia
Monash Medical Centre | Clayton, Victoria, 3168, Australia
Sir Charles Gairdner Hospital | Nedlands, Western Australia, 6009, Australia
Royal Prince Alfred Hospital | Camperdown, 2050, Australia
University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD | Plovdiv, 4002, Bulgaria
Specialized Hospital for Active Treatment of Haematological Diseases - Sofia | Sofia, 1756, Bulgaria
Multiprofile Hospital for Active Treatment Sveta Marina EAD | Varna, 9010, Bulgaria
University Health Network | Toronto, Ontario, M5G 2C4, Canada
Hopital Henri Mondor | Creteil, 94010, France
GH de Institut Catholique St. VincentHématologie | Lille, 59000, France
Hôpitaux de La Timone | Marseille Cedex 9, 13385, France
Hospital of Necker | Paris, 75015, France
Laiko General Hospital | Ampelokipi - Athens, 11526, Greece
Childrens' Hospital of Athens Aghoa Sofia | Athens, 115 27, Greece
General Hospital Georgios Gennimatas of Athens | Athens, 11527, Greece
University General Hospital of Patras | Rio Patras, 26500, Greece
Hippokration Hospital | Thessaloniki, 54642, Greece
Soroka University Medical Centre | Beer Sheva, 84101, Israel
Rambam Health Corporation | Haifa, 31096, Israel
HaEmek Medical Center | Haïfa (Afula), 18101, Israel
Shaare Zedek Medical Center | Jerusalem, 91031, Israel
Hadassah Medical Center | Jerusalem, 91120, Israel
Galilee Medical Center | Nahariya, 22100, Israel
Rabin Medical Center | Petah Tikva, 49100, Israel
Presidio Ospedaliero Antonio Perrino | Brindisi, 72100, Italy
Universita degli Studi di Cagliari - ASL8 | Cagliari, 09121, Italy
Azienda Ospedaliera Universitaria Di Ferrara Arcispedale Sanna | Ferrara, 44124, Italy
Ospedale Galliera | Genoa, 16128, Italy
Fondazione Ca Granda IRCCS Ospedale Maggiore | Milan, 20122, Italy
Seconda Universita Degli Studi Di Napoli | Naples, 80131, Italy
AORN A Cardarelli | Napoli, 80131, Italy
Azienda Ospedaliero Universitaria S. Luigi Gonzaga | Orbassano, 10043, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello | Palermo, 90146, Italy
Azienda Ospedaliera Universitaria Integrata Di Verona | Verona, 37134, Italy
Chronic Care Center | Beirut, Lebanon
Hospital Sultanah Aminah | Johor Bahru, Johor, 80100, Malaysia
Hospital Sultanah Bahiyah | Alor Setar, Kedah, 5460, Malaysia
Hospital Raja Permaisuri Bainun | Ipoh, Perak, 30990, Malaysia
Queen Elizabeth Hospital | Kota Kinabalu, Sabah, 88586, Malaysia
Hospital Umum Sarawak | Kuching, Sarawak, 93586, Malaysia
University Malaya Medical Centre | Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, 59100, Malaysia
Hospital Pulau Pinang c/o Penang Medical College | Georgetown, 10990, Malaysia
Changhua Christian Hospital | Changhua City, 500, Taiwan
Kaohsiung Medical University Hospital | Kaohsiung, 807, Taiwan
China Medical University Hospital | Taichung, 40447, Taiwan
National Taiwan University Hospital | Taipei, Zhongzheng Dist., 100, Taiwan
Chulalongkorn University | Bangkok, 10330, Thailand
Siriraj Hospital Mahidol University | Bangkok, 10700, Thailand
Maharaj Nakorn Chiang Mai Chiang Mai University | Chiang Mai, 50200, Thailand
University Hospital Farhat Hached | Sousse, 4031, Tunisia
Bone Marrow Transplant Center | Tunis, 1006, Tunisia
Aziza Othmana Hospital | Tunis, 1008, Tunisia
Military Hospital of Tunis | Tunis, 1089, Tunisia
Acibadem Adana Hospital | Adana, 01130, Turkey
Cukurova University Medical Faculty Balcali Hospital | Adana, 01330, Turkey
Hacettepe Universitesi | Ankara, 01660, Turkey
Antalya Egitim Arastirma | Antalya, 07100, Turkey
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi | Istanbul, 34093, Turkey
Ege Universitesi Tip Fakultesi Hastanesi | Izmir, 35100, Turkey
Mersin University Medical Faculty | Mersin, 33343, Turkey
St James University Hospital | Leeds, LS9 7TF, United Kingdom
University College Hospital Trust | London Bloomsbury, WC1E 6AU, United Kingdom
Barts Health NHS Trust | London, E1 1BB, United Kingdom
Whittington Hospital | London, N19 5NF, United Kingdom
Manchester Royal Infirmary | Manchester, M13 9WL, United Kingdom
Location Countries

Australia

Bulgaria

Canada

France

Greece

Israel

Italy

Lebanon

Malaysia

Taiwan

Thailand

Tunisia

Turkey

United Kingdom

United States

Verification Date

January 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Luspatercept (ACE-536) plus Best Supportive Care (BSC)

Type: Experimental

Description: Luspatercept, subcutaneous(ly) (SC) once every 21 days

Label: Placebo plus Best Supportive Care (BSC)

Type: Placebo Comparator

Description: normal saline solution subcutaneous(ly) (SC) once every 21 days

Acronym BELIEVE
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov