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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 10/30/2020.

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Subjects Who Require Regular RBC Transfusions Due to Beta (β)-Thalassemia.

Clinicaltrials.gov identifier NCT04143724

Recruitment Status Recruiting

First Posted October 29, 2019

Last update posted July 23, 2020

Study Description

Brief summary:

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric subjects who require regular RBC transfusions due to β-thalassemia. At least 12 subjects are planned to be enrolled per each of the 4 age-group strata at 2 different dose levels. Any subject benefiting from the study treatment (at the Investigator's discretion), at the completion of the Treatment Period, will be offered the opportunity to continue luspatercept and long-term follow-up in the Long-term Treatment and Follow-up Period. Subjects entering the Long-term Treatment and Follow-up Period will continue to receive luspatercept at the same dose level as during the Treatment Period for up to 5 years from their first dose (Cycle 1 Day 1) or when the drug is approved for pediatric patients (whichever comes first). Subjects who receive treatment for the full 5 years will then have an End of Treatment visit and then a Post End of Treatment visit (12 weeks from last dose). Any subject that discontinues treatment prior to 5 years from Cycle 1 Day 1 will be have an End of Treatment visit, a Post End of Treatment visit (12 weeks from last dose), and then will continue in Long-Term Posttreatment Follow-up until 5 years from Cycle 1 Day 1 or until they withdraw consent/assent, are lost to follow-up, or the End of Trial, whichever occurs first.

  • Condition or Disease:Beta-Thalassemia
  • Intervention/Treatment: Drug: ACE-536
    Drug: ACE-536
  • Phase: Phase 2
Detailed Description

This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric subjects who require regular red blood cell (RBC) transfusions due to β-thalassemia and to determine the recommended dose (RD) to be used in the planned confirmatory pediatric study. The primary endpoints are the determination of the RD and the development of a PK model that describes the PK exposure data of luspatercept and associated variability. The secondary endpoints include change in RBC transfusion burden, change in hemoglobin levels, safety, frequency of antidrug antibodies and its effect on safety and Exposure-response relationships for measures of activities and toxicity. The study will consist of the following periods: - Screening/Run-in Period - Treatment Period - Posttreatment Follow-up Period - Long-term Treatment and Follow-up Period Subject screening procedures will occur during the Screening/Run-in Period, within 12 weeks prior to the start of study treatment. Subjects who meet the study eligibility criteria will be enrolled into the Treatment Period. Patients enrolled into the study should have β-thalassaemia (including Hemoglobin E/β-thalassaemia) with ≥ 4 RBC transfusions in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period. Subjects in all planned dose escalation cohorts will receive luspatercept, administered as a subcutaneous (SC) injection, every 3 weeks (21 days) for up to 4 cycles (84 days) in the Treatment Period. Dose delay(s) and dose reduction(s) may be required for individual subjects. Each dose escalation cohort will consist of up to 6 subjects. Subjects will be treated at one of 2 dose levels, 0.75 mg/kg or 1.0 mg/kg, and enrollment will be sequential in descending order of age: - Cohort 1: 12 to < 18 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 2: 12 to < 18 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 3: 6 to < 12 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 4: 6 to < 12 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 5: 2 to < 6 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 6: 2 to < 6 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 7: 6 months to < 2 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 8: 6 months to < 2 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles). The first dose of luspatercept should be administered at least 14 days after the last transfusion prior to enrollment. Subject enrollment via the Integrated Response Technology (IRT) system and study treatment administration should be on the same day (Cycle 1 Day 1), provided that the eligibility criteria are met. Otherwise, the subject will need to be rescreened. Once all 6 subjects in a cohort have completed the first cycle (Study Day 22), a Dose Review Team (DRT), consisting of the Celgene Medical Monitor, Celgene lead safety physician, Celgene biostatistician, other Celgene functional area representatives or designees, as appropriate, and all active site Investigators and/or designees (at sites with a subject who has received study drug), will review all safety data, including dose-limiting toxicities (DLTs), adverse events (AEs), serious adverse events (SAEs), and laboratory results (including hematology and chemistry) reported during Cycle 1 of each dose level. A DLT, using the current active version of the National Cancer Institute Common Toxicity Criteria for Adverse Events, version 5.0 (NCI-CTCAE v.5.0), is defined as any of the following toxicities at any dose level occurring within 21 days of the first administered dose: - Treatment-related SAE of ≥ Grade 3 - Treatment-related nonhematologic AE of ≥ Grade 3 - Treatment-related hematologic AE of ≥ Grade 4 The DRT recommendations to enroll the next cohort at the next planned dose level and/or proceed to the next lower age group will be based in part upon the following criteria: - If a DLT occurs in ≤ 1 subject (out of 6) in a cohort within 21 days following the initial dose of luspatercept, the next planned dose level and/or next lower age group dose may proceed; - If a DLT occurs in ≥ 2 subjects (out of 6) in a cohort within 21 days following the initial dose of luspatercept, the next planned dose level and/or the next lower age group should not proceed; - If a hemoglobin increase of ≥ 2.0 g/dL (confirmed within 14 days after initial study treatment administration and not attributable to RBC transfusion) occurs in ≥ 2 subjects (out of 6) in a cohort, the decision to proceed to the next planned dose level and/or the next lower age group will need to be evaluated by the DRT. At least 12 subjects are planned to be enrolled with up to 12 subjects per age-group strata eligible for the Dose Determining Sets (DDS). With up to 4 age groups, a total of up to 48 subjects are to be included in the DDS. This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric subjects who require regular red blood cell (RBC) transfusions due to β-thalassemia and to determine the recommended dose (RD) to be used in the planned confirmatory pediatric study. The primary endpoints are the determination of the RD and the development of a pharmacokinetic (PK) model that describes the PK exposure data of luspatercept and associated variability. The secondary endpoints include change in RBC transfusion burden, change in hemoglobin levels, safety, frequency of antidrug antibodies and its effect on safety and Exposure-response relationships for measures of activities and toxicity. The study will consist of the following periods: - Screening/Run-in Period - Treatment Period - Posttreatment Follow-up Period - Long-term Treatment and Follow-up Period Subject screening procedures will occur during the Screening/Run-in Period, within 12 weeks prior to the start of study treatment. Subjects who meet the study eligibility criteria will be enrolled into the Treatment Period. Patients enrolled into the study should have β-thalassaemia (including Hemoglobin E/β-thalassaemia) with ≥ 4 RBC transfusions in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period. Subjects in all planned dose escalation cohorts will receive luspatercept, administered as a subcutaneous (SC) injection, every 3 weeks (21 days) for up to 4 cycles (84 days) in the Treatment Period. Dose delay(s) and dose reduction(s) may be required for individual subjects. Each dose escalation cohort will consist of up to 6 subjects. Subjects will be treated at one of 2 dose levels, 0.75 mg/kg or 1.0 mg/kg, and enrollment will be sequential in descending order of age: - Cohort 1: 12 to < 18 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 2: 12 to < 18 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 3: 6 to < 12 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 4: 6 to < 12 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 5: 2 to < 6 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 6: 2 to < 6 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 7: 6 months to < 2 years: Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles) - Cohort 8: 6 months to < 2 years: Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles) The first dose of luspatercept should be administered at least 14 days after the last transfusion prior to enrollment. Subject enrollment via the Integrated Response Technology (IRT) system and study treatment administration should be on the same day (Cycle 1 Day 1), provided that the eligibility criteria are met. Otherwise, the subject will need to be rescreened. Once all 6 subjects in a cohort have completed the first cycle (Study Day 22), a Dose Review Team (DRT), consisting of the Celgene Medical Monitor, Celgene lead safety physician, Celgene biostatistician, other Celgene functional area representatives or designees, as appropriate, and all active site Investigators and/or designees (at sites with a subject who has received study drug), will review all safety data, including dose-limiting toxicities (DLTs), adverse events (AEs), serious adverse events (SAEs), and laboratory results (including hematology and chemistry) reported during Cycle 1 of each dose level. A DLT, using the current active version of the National Cancer Institute Common Toxicity Criteria for Adverse Events, version 5.0 (NCI-CTCAE v.5.0), is defined as any of the following toxicities at any dose level occurring within 21 days of the first administered dose: - Treatment-related SAE of ≥ Grade 3 - Treatment-related nonhematologic AE of ≥ Grade 3 - Treatment-related hematologic AE of ≥ Grade 4 The DRT recommendations to enroll the next cohort at the next planned dose level and/or proceed to the next lower age group will be based in part upon the following criteria: - If a DLT occurs in ≤ 1 subject (out of 6) in a cohort within 21 days following the initial dose of luspatercept, the next planned dose level and/or next lower age group dose may proceed; - If a DLT occurs in ≥ 2 subjects (out of 6) in a cohort within 21 days following the initial dose of luspatercept, the next planned dose level and/or the next lower age group should not proceed; - If a hemoglobin increase of ≥ 2.0 g/dL (confirmed within 14 days after initial study treatment administration and not attributable to RBC transfusion) occurs in ≥ 2 subjects (out of 6) in a cohort, the decision to proceed to the next planned dose level and/or the next lower age group will need to be evaluated by the DRT. At least 12 subjects are planned to be enrolled with up to 12 subjects per age-group strata eligible for the Dose Determining Sets (DDS). With up to 4 age groups, a total of up to 48 subjects are to be included in the DDS. To minimize safety risk to subjects, best supportive care will be available, including RBC transfusions, iron-chelating agents, use of antibiotic therapy, antiviral and antifungal therapy, and/or nutritional support as needed. Dose interruptions/delays and dose reductions may be used to manage toxicities. Subjects can receive study treatment for up to 4 cycles during the Treatment Period, until study treatment becomes intolerable, or the subject wishes to discontinue study treatment for any reason. The decision to discontinue a subject from study treatment, is the responsibility of the treating physician; however, prior to discontinuing a subject, it is recommended that the Investigator contact the Medical Monitor and forward appropriate supporting documents for review and discussion. Subjects are to undergo end-of-treatment evaluations when study treatment is discontinued. The reason for treatment discontinuation will be recorded in the electronic case report form (eCRF) pages and in the source document. During the Treatment Period, any subjects discontinued from study treatment will continue to be assessed in the Posttreatment Follow-up Period for AEs, concomitant medications, concomitant procedures, transfusions, hematologic improvement, and subsequent therapies. Any subject benefiting from the study treatment (at the Investigator's discretion), at the completion of the Treatment Period, will be offered the opportunity to continue luspatercept and long-term follow-up in the Long-term Treatment and Follow-up Period. Subjects entering the Long-term Treatment and Follow-up Period will continue to receive luspatercept at the same dose level as during the Treatment Period for up to 5 years from their first dose (Cycle 1 Day 1) or when the drug is approved for pediatric patients (whichever comes first). Subjects who receive treatment for the full 5 years will then have an End of Treatment (EOT) visit and then a Post End of Treatment visit (12 weeks from last dose). Any subject that discontinues treatment prior to 5 years from Cycle 1 Day 1 will be have an End of Treatment visit, a Post End of Treatment visit (12 weeks from last dose), and then will continue in Long-Term Posttreatment Follow-up until 5 years from Cycle 1 Day 1.

Study Design
  • Study Type: Interventional
  • Estimated Enrollment: 48 participants
  • Allocation: Non-Randomized
  • Intervention Model: Sequential Assignment
  • Masking: None (Open Label) ()
  • Primary Purpose: Treatment
  • Official Title: A Phase 2A Open-label Multicenter Study to Evaluate the Safety and Pharmacokinetics of Luspatercept in Pediatric Subjects Who Require Regular Red Blood Cell Transfusions Due to B-Thalassemia
  • Actual Study Start Date: November 2019
  • Estimated Primary Completion Date: December 2021
  • Estimated Study Completion Date: September 2026
Arms and interventions
Arm Intervention/treatment
Experimental: Cohort 1: 12 to < 18 years - Luspatercept 0.75 mg/kg
Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 0.75mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 3: 6 to < 12 years: Luspatercept 0.75 mg/kg
Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 0.75mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 5: 2 to < 6 years: Luspatercept 0.75 mg/kg
Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 0.75mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 7: 6 months to < 2 years: Luspatercept 0.75 mg/kg
Luspatercept 0.75 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 0.75mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 2: 12 to < 18 years: Luspatercept 1.0 mg/kg,
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 1.0mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 4: 6 to < 12 years: Luspatercept 1.0 mg/kg
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 1.0mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 6: 2 to < 6 years: Luspatercept 1.0 mg/kg
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 1.0mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Experimental: Cohort 8: 6 months to < 2 years: Luspatercept 1.0 mg/kg
Luspatercept 1.0 mg/kg, administered SC once every 21 days (for up to 4 cycles)
Drug: ACE-536
Subjects will start with luspatercept at 1.0mg/kg and the Dose review team (DRT) will evaluate all toxicities of each subject after Cycle 1 and determine whether or not to enroll the next cohort at the next planned dose level and/or next lower age group
Outcome Measures
  • Primary Outcome Measures: 1. Determination of the Recommended Dose [ Time Frame: At the end of Cycle 1 (each Cycle is 21 days) ]
    Determine the recommended dose of luspatercept that is safe and tolerable in pediatric subjects with transfusion-dependent B-thalassemia.
  • 2. Pharmacokinetics - Cmax [ Time Frame: Up to Day 148 (+/-) 7 days ]
    Maximum plasma concentration of drug
  • 3. Pharmacokinetics - AUC [ Time Frame: Up to Day 148 (+/-) 7 days ]
    Area under the plasma concentration-time curve from time zero extrapolated to infinity
  • Secondary Outcome Measures: 1. To evaluate the change in RBC transfusion burden [ Time Frame: Up to 15 weeks (Up to 12 weeks for the Treatment Period plus 3 weeks post last dose (End of Treatment), thus a total of 15 weeks) ]
    Change from baseline as continuous variable
  • 2. To evaluate the change in hemoglobin levels over the course of the study [ Time Frame: Up to 15 weeks (Up to 12 weeks for the Treatment Period plus 3 weeks post last dose (End of Treatment), thus a total of 15 weeks) ]
    Change from baseline as continuous variable
  • 3. Safety Adverse Events (AEs) [ Time Frame: up to 5 years ]
    Frequency/severity and seriousness of AEs, defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the subject's health, including laboratory test values, regardless of etiology. Any worsening (ie, any clinically significant adverse change in the frequency or intensity of a preexisting condition) should be considered an AE.
  • 4. Frequency of antidrug antibodies and its effect on safety [ Time Frame: up to 2 years ]
    ADA measurements; key AEs and safety endpoints by ADA results (positive versus negative)
  • 5. Exposure-response relationships for measures of activities and toxicity [ Time Frame: Up to 15 weeks (Up to 12 weeks for the Treatment Period plus 3 weeks post last dose (End of Treatment), thus a total of 15 weeks) ]
    Exposure-response assessed by the time the subject is exposed to treatment to the change in response (e.g. either transfusion, Hb or AE).
Eligibility Criteria
  • Ages Eligible for Study: 6 to 18 Months (Child)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Criteria

Inclusion Criteria:

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

1. Subject must be 6 months to < 18 years of age at the time of signing the informed consent form (ICF)/informed assent form (IAF). 2. Subject (and when applicable, parent/legal representative) must understand and voluntarily sign an ICF/IAF prior to conducting any study-related assessments/procedures. 3. Subject (and when applicable, parent/legal representative) is willing and able to adhere to the study visit schedule and other protocol requirements. 4. Subject must have documented diagnosis of β-thalassemia or Hemoglobin E/β-thalassemia. 5. Subject is regularly transfused, defined as: ≥ 4 RBC transfusions in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period. Note: For the purpose of the study, transfusions administered over 2 or 3 consecutive days are considered as part of a single transfusion event. 6. Subject has Karnofsky (age ≥16 years) or Lansky (age 1000 x 109/L.

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

10. Subject has treatment with another investigational drug or device ≤ 28 days prior to
enrollment.

11. Subject has prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).

12. Subject has used an erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to
enrollment.

13. Subject use of iron chelation therapy (ICT), if initiated ≤ 8 weeks prior to
enrollment (allowed if initiated > 8 weeks before or during treatment).

14. Subject use of hydroxyurea treatment ≤ 24 weeks prior to enrollment.

15. Subject is pregnant or breastfeeding female.

16. Subject has uncontrolled hypertension. Controlled hypertension for this protocol is
considered ≤ Grade 1 according to NCI CTCAE version 5.0.

17. Subject has major organ damage, including:

1. Symptomatic splenomegaly

2. Liver disease with alanine aminotransferase (ALT)/aspartate aminotransferase
(AST) > 3X the upper limit of normal (ULN) for age

3. 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 enrollment

4. Lung disease, including pulmonary fibrosis or pulmonary hypertension which are
clinically significant

5. Renal insufficiency defined as:

- A serum creatinine based on age/gender based on threshold derived from
Schwartz formula for estimating GFR utilizing child length and stature data
published by the Centers for Disease Control

18. Subject has proteinuria ≥ Grade 3 according to NCI CTCAE version 5.0.

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

20. Subject has major surgery ≤ 12 weeks prior to enrollment (subjects must have
completely recovered from any previous surgery prior to enrollment).

21. Subject has history of severe allergic or anaphylactic reactions or hypersensitivity
to recombinant proteins or excipients in the IP (see IB).

22. Subject use of cytotoxic agents, immunosuppressants ≤ 28 days prior to enrollment (ie,
antithymocite globulin (ATG) or cyclosporine).

23. Subject has 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.

Contacts and Locations
Contacts

Contact: Associate Director Clinical Trial Disclosure 1-888-260-1599 clinicaltrialdisclosure@celgene.com

Locations

United States, California
Children's Hospital of Los Angeles
Los Angeles

Germany
Universitatsklinikum Ulm
Ulm

Greece
General Children's Hospital "Agia Sophia"
Athens

Italy
Ente Ospedaliero Ospedali Galliera - Centro della Microcitemia e delle Anemie Congenite
Genoa

Italy
Azienda Ospedaliera Universitaria "Federico II"
Napoli

Italy
Azienda Ospedaliero Universitaria S. Luigi Gonzaga
Orbassano

Thailand
Siriraj Hospital Mahidol University
Bangkok

Turkey
Ege Universitesi Tip Fakultesi Hastanesi
Izmir

Sponsors and Collaborators

Celgene

Acceleron Pharma, Inc.

Investigators

Study Director: Bouchra Benettaib, MD Celgene Corporation

More Information
  • Responsible Party: Celgene
  • ClinicalTrials.gov Identifier: NCT04143724 History of Changes
  • Other Study ID Numbers: ACE-536-B-THAL-004, U1111-1241-4168, 2019-000208-13
  • First Posted: October 29, 2019 Key Record Dates
  • Last Update Posted: July 23, 2020
  • Last Verified: July 2020
  • Individual Participant
    Data (IPD) Sharing
    Statement:
  • Plan to Share IPD: Yes
  • Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/
  • Supporting Materials: Study Protocol, Statistical Analysis Plan (SAP), Informed Consent Form (ICF), Clinical Study Report (CSR), Analytic Code
  • Time Frame: See Plan Description
  • Access Criteria: See Plan Description
  • URL: https://www.celgene.com/research-development/clinical-trials/clinical-trials-data-sharing/
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Keywords provided by Celgene: ACE-536
    Luspatercept
    Pharmacokinetics
    Beta-Thalassemia
    Red Blood Cell Transfusion
  • Additional relevant MeSH terms: Thalassemia beta-Thalassemia