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A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants With Alpha (α)-Thalassemia

Primary Purpose

Anemia

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Luspatercept
Placebo
Best Supportive Care
Sponsored by
Bristol-Myers Squibb
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Anemia focused on measuring Luspatercept, BMS-986346, ACE-536, α-thalassemia, Alpha Thalassemia, Reblozyl, Transfusions, Non-transfusion dependent, RBC transfusion dependent

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria: Participant has documented diagnosis of α-thalassemia hemoglobin H (HbH) disease (electrophoresis⎯ or high-performance liquid chromatography [HPLC]⎯based methods for Hb variant analyses are accepted), with or without transfusion dependence; compounded combination with β-thalassemia is allowed if at least 1 non-mutated β-chain gene is present. TD: i) TD participant: (1) ≥ 6 RBC units during the 24 weeks prior to randomization; and (2) No transfusion-free period for > 56 days during the 24 weeks prior to randomization; ii) NTD participant: (1) < 6 Red blood cell (RBC) units during the 24 weeks prior to randomization; and (2) RBC transfusion-free during at least 8 weeks prior to randomization; and (3) Mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1. Investigators shall counsel women of childbearing potential (WOCBP), and male participants who are sexually active with WOCBP, on the importance of pregnancy prevention, the implications of an unexpected pregnancy, and the potential of fetal toxicity occurring due to transmission of study intervention, present in seminal fluid, to a developing fetus, even if the participant has undergone a successful vasectomy or if the partner is pregnant. Key Exclusion Criteria: Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participant from participating in the study. Any condition, including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he/she were to participate in the study History of deep venous thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to randomization Diagnosis of α-thalassemia Trait, Hb Bart hydrops, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias (for example, severe G6PD deficiency, pyruvate kinase deficiency, etc.). Bleeding disorders manifested by frequent bleeding episodes (e.g., menorrhagia, epistaxis, clotting disorders). Undergone episodes of hemolysis not related to alpha-thalassemia, for example, after use of hemolysis-predisposing drugs (for example, antimalarial, nonsteroidal anti-inflammatory drug [NSAID]), within the 8 weeks prior to randomization. Prior exposure to gene therapy to treat α-thalassemia. Use of an erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to randomization. Note: Other protocol-defined inclusion/exclusion criteria apply.

Sites / Locations

  • Local Institution - 0008
  • Local Institution - 0013Recruiting
  • Local Institution - 0015Recruiting
  • Local Institution - 0017Recruiting
  • Local Institution - 0014Recruiting
  • Local Institution - 0012Recruiting
  • Local Institution - 0011Recruiting
  • Local Institution - 0002Recruiting
  • Local Institution - 0023Recruiting
  • Local Institution - 0016Recruiting
  • Local Institution - 0005Recruiting
  • Local Institution - 0007Recruiting
  • Local Institution - 0018Recruiting
  • Local Institution - 0006Recruiting
  • Local Institution - 0009Recruiting
  • Local Institution - 0025
  • Local Institution - 0024
  • Local Institution - 0026Recruiting
  • Local Institution - 0020Recruiting
  • Local Institution - 0022
  • Local Institution - 0028Recruiting
  • Local Institution - 0019Recruiting
  • Local Institution - 0010Recruiting
  • Local Institution - 0004Recruiting
  • Local Institution - 0003Recruiting
  • Local Institution - 0001Recruiting
  • Local Institution - 0027
  • Local Institution - 0021

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Arm Label

Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC)

TD: Placebo + BSC

Non-transfusion Dependent (NTD): Luspatercept + BSC

NTD: Placebo + BSC

Arm Description

Outcomes

Primary Outcome Measures

TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Week 13-48 compared to 12-week interval immediately prior to date of first dose
NTD Cohort: Number of participants with an increase from baseline of ≥ 1.0 grams (g)/decilitre (dL) in mean hemoglobin (Hb) values over the continuous 12-week interval from Week 13 to Week 24 in the absence of transfusion

Secondary Outcome Measures

TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 24-week interval on treatment compared to 24-week interval immediately prior to date of first dose
TD Cohort: The longest duration with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units
TD Cohort: Number of RBC transfusion units from week 1 to week 48
NTD Cohort: Change from baseline in hemoglobin in the absence of transfusion at Week 24
NTD Cohort: The longest duration of an increase from baseline of ≥ 1.0 g/dL in mean hemoglobin values starting from Week 13 in the absence of transfusion
NTD Cohort: Time Duration with an increase from baseline of ≥ 1.0 g/dL in hemoglobin values in the absence of transfusion within 48 weeks
NTD Cohort: Number of participants with an increase from baseline of ≥1.0 g/dL in mean Hb values over the continuous 12- week interval in the absence of transfusion
NTD Cohort: ≥ 3 Increase from Baseline in Functional Assessment of Cancer Therapy Anemia Fatigue Subscale (FACT-An FS) Score from Baseline to Week 13 to Week 24
TD and NTD Cohorts: Number of participants with adverse events (AEs)
TD and NTD Cohorts: Number of participants with serious AEs (SAEs)
TD and NTD Cohorts: Number of participants with antidrug antibody (ADA) (positive or negative)
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden during any continuous 24-week interval within 48 weeks compared to the 24-week interval immediately prior to the date of first dose
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden during any continuous 12-week interval compared to the 12-week interval immediately prior to the date of first dose
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden from Week 13 to Week 24 and Week 37 to Week 48 compared to the 12-week interval immediately prior to the date of first dose
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden from Week 1 to Week 24 and Week 25 to Week 48 compared to the 24-week interval immediately prior to the date of first dose
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden from Week 13 to Week 24 and Week 37 to Week 48 compared to the 12-week interval immediately prior to the date of first dose
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden from Week 1 to Week 24 and Week 25 to Week 48 compared to the 24-week interval immediately prior to the date of first dose
TD Cohort: Change from baseline in total RBC units transfused from Week 1 to Week 24, Week 25 to Week 48, and Week 1 to Week 48
TD Cohort: The longest duration of RBC transfusion-free period for participants who achieve transfusion-free period of ≥ 12 weeks
TD Cohort: The longest duration of reduction in transfusion burden for participants who achieve a response (rolling 12-week and 24-week response, both for ≥ 33% and ≥ 50% reduction)
TD Cohort: Time from first dose to first day of response (rolling 12-week and 24-week response, both for ≥ 33% and ≥ 50% reduction)
TD Cohort: Change from baseline in number of transfusion events at Week 48
TD Cohort: Number of participants who achieve RBC transfusion-free period of any continuous ≥ 12 weeks during treatment
NTD Cohort: Number of participants who achieve RBC transfusion-free period of any continuous ≥ 24 weeks during treatment
NTD Cohort: Time to first transfusion
NTD Cohort: Number of transfusions
NTD Cohort: Number of transfusion visits/units
TD and NTD Cohorts: Change from baseline in mean of hemoglobin values over the continuous 12-week interval from Week 13 to Week 24 and Week 37 to Week 48 in the absence of transfusions
NTD Cohort: Number of participants achieving an increase from baseline of ≥1.0g/dL or ≥1.5g/dL in mean Hb values in absence of transfusions from Week 13 to Week 24, Week 37 to Week 48 and during any continuous 12-week window within 24 weeks and 48 weeks
NTD Cohort: Time from first to last Hb measurement with increase from baseline by ≥ 1.0 g/dL
NTD Cohort: Number of participants who achieve an increase in mean Hb of >10g/dL values during any continuous 12-week and 24-week interval within 48 weeks in the absence of transfusions
TD and NTD Cohorts: Change from baseline in self-reported health-related quality of life (HRQoL) assessed by physical component summary (PCS) and mental component summary (MCS) of 36-item short-form health survey version2 (SF-36v2) at Week 24 and Week 48
NTD Cohort: Change from baseline in non-transfusion dependent β-thalassemia patient-reported outcome (NTDT-PRO) Tiredness/weakness (T/W) and shortness of breath (SoB) domain scores from Week 13 to Week 24 and from Week 37 to Week 48
NTD Cohort: Change from baseline in FACT-An FS Score at Week 24 and Week 48
NTD Cohort: Change from baseline in Functional Assessment of Cancer Therapy Anemia Anemia Subscale (FACT-An AS) at Week 24 and Week 48
TD and NTD Cohorts: Number of participants with at least one hemolytic crisis
TD and NTD Cohorts: Rate of hemolytic crises
TD and NTD Cohorts: Time to first hemolytic crisis
TD and NTD Cohorts: Time to second hemolytic crisis
TD and NTD Cohorts: Change from baseline in hemolysis markers at Week 24 and Week 48
NTD Cohort: Change from baseline in the 6-minute walk test (6MWT) distance at Week 24 and Week 48
TD and NTD Cohorts: Pharmacokinetics (PK): Serum concentration of Luspatercept
TD and NTD Cohorts: Percent Change from Baseline in Biomarkers for Erythropoiesis at Week 84
The biomarkers for erythropoiesis to be evaluated include Hb variants including hemoglobin H (HbH), sTfR1, erythropoietin (EPO), growth differentiation factor (GDF11), GDF8, GDF15. The change will be measured as a percentage of change from baseline for all the biomarkers.
TD and NTD Cohorts: Percent Change from Baseline in Biomarkers and Parameters for Iron Homeostasis at Week 84
The biomarkers and parameters for iron homeostasis to be evaluated include hepcidin, erythroferrone (ERFE), serum ferritin, liver iron concentration (LIC), myocardial iron, iron chelation therapy (ICT). The change will be measured as a percentage of change from baseline for all the biomarkers.
TD and NTD Cohorts: Change in mean corpuscular volume (MCV) at Week 48
TD and NTD Cohorts: Change in mean corpuscular hemoglobin (MCH) at Week 48
TD and NTD Cohorts: Change in nucleated red blood cells (nRBC) at Week 48
TD and NTD Cohorts: Change in red blood cells (RBC) at Week 48

Full Information

First Posted
December 9, 2022
Last Updated
October 11, 2023
Sponsor
Bristol-Myers Squibb
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1. Study Identification

Unique Protocol Identification Number
NCT05664737
Brief Title
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants With Alpha (α)-Thalassemia
Official Title
A Phase 2, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) for the Treatment of Anemia in Adults With Alpha (α)-Thalassemia
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 9, 2022 (Actual)
Primary Completion Date
November 5, 2025 (Anticipated)
Study Completion Date
June 18, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bristol-Myers Squibb

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of the study is to determine the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in participants with α-thalassemia hemoglobin H (HbH) disease.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia
Keywords
Luspatercept, BMS-986346, ACE-536, α-thalassemia, Alpha Thalassemia, Reblozyl, Transfusions, Non-transfusion dependent, RBC transfusion dependent

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
177 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC)
Arm Type
Experimental
Arm Title
TD: Placebo + BSC
Arm Type
Placebo Comparator
Arm Title
Non-transfusion Dependent (NTD): Luspatercept + BSC
Arm Type
Experimental
Arm Title
NTD: Placebo + BSC
Arm Type
Placebo Comparator
Intervention Type
Biological
Intervention Name(s)
Luspatercept
Other Intervention Name(s)
BMS-986346, ACE-536, REBLOZYL
Intervention Description
Specified dose on specified days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Specified dose on specified days
Intervention Type
Other
Intervention Name(s)
Best Supportive Care
Intervention Description
Specified dose on specified days
Primary Outcome Measure Information:
Title
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 12 weeks during Week 13-48 compared to 12-week interval immediately prior to date of first dose
Time Frame
Up to Week 48
Title
NTD Cohort: Number of participants with an increase from baseline of ≥ 1.0 grams (g)/decilitre (dL) in mean hemoglobin (Hb) values over the continuous 12-week interval from Week 13 to Week 24 in the absence of transfusion
Time Frame
Up to Week 24
Secondary Outcome Measure Information:
Title
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units during any continuous 24-week interval on treatment compared to 24-week interval immediately prior to date of first dose
Time Frame
Up to Week 108
Title
TD Cohort: The longest duration with ≥ 50% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units
Time Frame
Up to Week 48
Title
TD Cohort: Number of RBC transfusion units from week 1 to week 48
Time Frame
Up to Week 48
Title
NTD Cohort: Change from baseline in hemoglobin in the absence of transfusion at Week 24
Time Frame
Baseline, Week 24
Title
NTD Cohort: The longest duration of an increase from baseline of ≥ 1.0 g/dL in mean hemoglobin values starting from Week 13 in the absence of transfusion
Time Frame
From Week 13 up to Week 48
Title
NTD Cohort: Time Duration with an increase from baseline of ≥ 1.0 g/dL in hemoglobin values in the absence of transfusion within 48 weeks
Time Frame
Up to Week 48
Title
NTD Cohort: Number of participants with an increase from baseline of ≥1.0 g/dL in mean Hb values over the continuous 12- week interval in the absence of transfusion
Time Frame
Up to Week 24
Title
NTD Cohort: ≥ 3 Increase from Baseline in Functional Assessment of Cancer Therapy Anemia Fatigue Subscale (FACT-An FS) Score from Baseline to Week 13 to Week 24
Time Frame
Baseline, Up to Week 24
Title
TD and NTD Cohorts: Number of participants with adverse events (AEs)
Time Frame
Up to 5 years
Title
TD and NTD Cohorts: Number of participants with serious AEs (SAEs)
Time Frame
Up to 5 years
Title
TD and NTD Cohorts: Number of participants with antidrug antibody (ADA) (positive or negative)
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden during any continuous 24-week interval within 48 weeks compared to the 24-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden during any continuous 12-week interval compared to the 12-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden from Week 13 to Week 24 and Week 37 to Week 48 compared to the 12-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 33% reduction from baseline in RBC transfusion burden from Week 1 to Week 24 and Week 25 to Week 48 compared to the 24-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden from Week 13 to Week 24 and Week 37 to Week 48 compared to the 12-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Number of participants with ≥ 50% reduction from baseline in RBC transfusion burden from Week 1 to Week 24 and Week 25 to Week 48 compared to the 24-week interval immediately prior to the date of first dose
Time Frame
Up to Week 48
Title
TD Cohort: Change from baseline in total RBC units transfused from Week 1 to Week 24, Week 25 to Week 48, and Week 1 to Week 48
Time Frame
Baseline, Up to Week 48
Title
TD Cohort: The longest duration of RBC transfusion-free period for participants who achieve transfusion-free period of ≥ 12 weeks
Time Frame
Up to Week 48
Title
TD Cohort: The longest duration of reduction in transfusion burden for participants who achieve a response (rolling 12-week and 24-week response, both for ≥ 33% and ≥ 50% reduction)
Time Frame
Up to Week 48
Title
TD Cohort: Time from first dose to first day of response (rolling 12-week and 24-week response, both for ≥ 33% and ≥ 50% reduction)
Time Frame
Up to Week 48
Title
TD Cohort: Change from baseline in number of transfusion events at Week 48
Time Frame
Baseline, Up to Week 48
Title
TD Cohort: Number of participants who achieve RBC transfusion-free period of any continuous ≥ 12 weeks during treatment
Time Frame
Up to Week 48
Title
NTD Cohort: Number of participants who achieve RBC transfusion-free period of any continuous ≥ 24 weeks during treatment
Time Frame
Up to Week 48
Title
NTD Cohort: Time to first transfusion
Time Frame
Up to Week 48
Title
NTD Cohort: Number of transfusions
Time Frame
Up to Week 48
Title
NTD Cohort: Number of transfusion visits/units
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Change from baseline in mean of hemoglobin values over the continuous 12-week interval from Week 13 to Week 24 and Week 37 to Week 48 in the absence of transfusions
Time Frame
Baseline, Up to Week 48
Title
NTD Cohort: Number of participants achieving an increase from baseline of ≥1.0g/dL or ≥1.5g/dL in mean Hb values in absence of transfusions from Week 13 to Week 24, Week 37 to Week 48 and during any continuous 12-week window within 24 weeks and 48 weeks
Time Frame
Up to Week 48
Title
NTD Cohort: Time from first to last Hb measurement with increase from baseline by ≥ 1.0 g/dL
Time Frame
Up to Week 48
Title
NTD Cohort: Number of participants who achieve an increase in mean Hb of >10g/dL values during any continuous 12-week and 24-week interval within 48 weeks in the absence of transfusions
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Change from baseline in self-reported health-related quality of life (HRQoL) assessed by physical component summary (PCS) and mental component summary (MCS) of 36-item short-form health survey version2 (SF-36v2) at Week 24 and Week 48
Time Frame
Baseline, Up to Week 48
Title
NTD Cohort: Change from baseline in non-transfusion dependent β-thalassemia patient-reported outcome (NTDT-PRO) Tiredness/weakness (T/W) and shortness of breath (SoB) domain scores from Week 13 to Week 24 and from Week 37 to Week 48
Time Frame
Baseline, Up to Week 48
Title
NTD Cohort: Change from baseline in FACT-An FS Score at Week 24 and Week 48
Time Frame
Baseline, Up to Week 48
Title
NTD Cohort: Change from baseline in Functional Assessment of Cancer Therapy Anemia Anemia Subscale (FACT-An AS) at Week 24 and Week 48
Time Frame
Baseline, Up to Week 48
Title
TD and NTD Cohorts: Number of participants with at least one hemolytic crisis
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Rate of hemolytic crises
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Time to first hemolytic crisis
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Time to second hemolytic crisis
Time Frame
Up to Week 48
Title
TD and NTD Cohorts: Change from baseline in hemolysis markers at Week 24 and Week 48
Time Frame
Baseline, Up to Week 48
Title
NTD Cohort: Change from baseline in the 6-minute walk test (6MWT) distance at Week 24 and Week 48
Time Frame
Baseline, Up to Week 48
Title
TD and NTD Cohorts: Pharmacokinetics (PK): Serum concentration of Luspatercept
Time Frame
Predose and post dose Day 1 through Day 48
Title
TD and NTD Cohorts: Percent Change from Baseline in Biomarkers for Erythropoiesis at Week 84
Description
The biomarkers for erythropoiesis to be evaluated include Hb variants including hemoglobin H (HbH), sTfR1, erythropoietin (EPO), growth differentiation factor (GDF11), GDF8, GDF15. The change will be measured as a percentage of change from baseline for all the biomarkers.
Time Frame
Baseline, Week 84
Title
TD and NTD Cohorts: Percent Change from Baseline in Biomarkers and Parameters for Iron Homeostasis at Week 84
Description
The biomarkers and parameters for iron homeostasis to be evaluated include hepcidin, erythroferrone (ERFE), serum ferritin, liver iron concentration (LIC), myocardial iron, iron chelation therapy (ICT). The change will be measured as a percentage of change from baseline for all the biomarkers.
Time Frame
Baseline, Week 84
Title
TD and NTD Cohorts: Change in mean corpuscular volume (MCV) at Week 48
Time Frame
Baseline, Week 48
Title
TD and NTD Cohorts: Change in mean corpuscular hemoglobin (MCH) at Week 48
Time Frame
Baseline, Week 48
Title
TD and NTD Cohorts: Change in nucleated red blood cells (nRBC) at Week 48
Time Frame
Baseline, Week 48
Title
TD and NTD Cohorts: Change in red blood cells (RBC) at Week 48
Time Frame
Baseline, Week 48

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Participant has documented diagnosis of α-thalassemia hemoglobin H (HbH) disease (electrophoresis⎯ or high-performance liquid chromatography [HPLC]⎯based methods for Hb variant analyses are accepted), with or without transfusion dependence; compounded combination with β-thalassemia is allowed if at least 1 non-mutated β-chain gene is present. TD: i) TD participant: (1) ≥ 6 RBC units during the 24 weeks prior to randomization; and (2) No transfusion-free period for > 56 days during the 24 weeks prior to randomization; ii) NTD participant: (1) < 6 Red blood cell (RBC) units during the 24 weeks prior to randomization; and (2) RBC transfusion-free during at least 8 weeks prior to randomization; and (3) Mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. Participant has Eastern Cooperative Oncology Group (ECOG) score of 0 or 1. Investigators shall counsel women of childbearing potential (WOCBP), and male participants who are sexually active with WOCBP, on the importance of pregnancy prevention, the implications of an unexpected pregnancy, and the potential of fetal toxicity occurring due to transmission of study intervention, present in seminal fluid, to a developing fetus, even if the participant has undergone a successful vasectomy or if the partner is pregnant. Key Exclusion Criteria: Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the participant from participating in the study. Any condition, including the presence of laboratory abnormalities, which places the participant at unacceptable risk if he/she were to participate in the study History of deep venous thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to randomization Diagnosis of α-thalassemia Trait, Hb Bart hydrops, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias (for example, severe G6PD deficiency, pyruvate kinase deficiency, etc.). Bleeding disorders manifested by frequent bleeding episodes (e.g., menorrhagia, epistaxis, clotting disorders). Undergone episodes of hemolysis not related to alpha-thalassemia, for example, after use of hemolysis-predisposing drugs (for example, antimalarial, nonsteroidal anti-inflammatory drug [NSAID]), within the 8 weeks prior to randomization. Prior exposure to gene therapy to treat α-thalassemia. Use of an erythropoiesis-stimulating agent (ESA) ≤ 24 weeks prior to randomization. Note: Other protocol-defined inclusion/exclusion criteria apply.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
BMS Study Connect Contact Center www.BMSStudyConnect.com
Phone
855-907-3286
Email
Clinical.Trials@bms.com
First Name & Middle Initial & Last Name or Official Title & Degree
First line of the email MUST contain the NCT# and Site #.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bristol-Myers Squibb
Organizational Affiliation
Bristol-Myers Squibb
Official's Role
Study Director
Facility Information:
Facility Name
Local Institution - 0008
City
Halifax
State/Province
Nova Scotia
ZIP/Postal Code
B3K 6R8
Country
Canada
Individual Site Status
Withdrawn
Facility Name
Local Institution - 0013
City
Foshan
State/Province
Guangdong
ZIP/Postal Code
528000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0013
Facility Name
Local Institution - 0015
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0015
Facility Name
Local Institution - 0017
City
Maoming Shi
State/Province
Guangdong
ZIP/Postal Code
525000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0017
Facility Name
Local Institution - 0014
City
Nanning Shi
State/Province
Guangxi
ZIP/Postal Code
530021
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0014
Facility Name
Local Institution - 0012
City
Kunming
State/Province
Yunnan
ZIP/Postal Code
650032
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0012
Facility Name
Local Institution - 0011
City
Haikou
ZIP/Postal Code
570203
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0011
Facility Name
Local Institution - 0002
City
Haikou
ZIP/Postal Code
570311
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0002
Facility Name
Local Institution - 0023
City
Liuzhou
ZIP/Postal Code
545006
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0023
Facility Name
Local Institution - 0016
City
Nanning
ZIP/Postal Code
530021
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0016
Facility Name
Local Institution - 0005
City
Thessaloniki
State/Province
B
ZIP/Postal Code
546 42
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0005
Facility Name
Local Institution - 0007
City
Larissa
State/Province
E
ZIP/Postal Code
412 21
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0007
Facility Name
Local Institution - 0018
City
Rio
State/Province
G
ZIP/Postal Code
265 04
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0018
Facility Name
Local Institution - 0006
City
Athens
ZIP/Postal Code
115 27
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0006
Facility Name
Local Institution - 0009
City
Goudi
ZIP/Postal Code
11527
Country
Greece
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0009
Facility Name
Local Institution - 0025
City
Hong Kong
State/Province
HK
Country
Hong Kong
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0025
Facility Name
Local Institution - 0024
City
Hong Kong Island
Country
Hong Kong
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0024
Facility Name
Local Institution - 0026
City
Genova
State/Province
GE
ZIP/Postal Code
16128
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0026
Facility Name
Local Institution - 0020
City
Orbassano
State/Province
TO
ZIP/Postal Code
10043
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0020
Facility Name
Local Institution - 0022
City
Cagliari
ZIP/Postal Code
9121
Country
Italy
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0022
Facility Name
Local Institution - 0028
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0028
Facility Name
Local Institution - 0019
City
Napoli
ZIP/Postal Code
80138
Country
Italy
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0019
Facility Name
Local Institution - 0010
City
Kaohsiung
State/Province
KHH
ZIP/Postal Code
807
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0010
Facility Name
Local Institution - 0004
City
Nan Gang Qu
State/Province
TPE
ZIP/Postal Code
10002
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0004
Facility Name
Local Institution - 0003
City
Taichung
State/Province
TXG
ZIP/Postal Code
40447
Country
Taiwan
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0003
Facility Name
Local Institution - 0001
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0001
Facility Name
Local Institution - 0027
City
Altındağ
ZIP/Postal Code
06230
Country
Turkey
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0027
Facility Name
Local Institution - 0021
City
Topkapı
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Not yet recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Site 0021

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myers Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosurecommitment.html
IPD Sharing Time Frame
See Plan Description
IPD Sharing Access Criteria
See Plan Description
IPD Sharing URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html
Links:
URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
Description
BMS Clinical Trial Information
URL
http://www.BMSStudyConnect.com
Description
BMS Clinical Trial Patient Recruiting
URL
https://www.fda.gov/Safety/MedWatch/SafetyInformation/default.htm
Description
FDA Safety Alerts and Recalls
URL
https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
Description
Investigator Inquiry Form

Learn more about this trial

A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants With Alpha (α)-Thalassemia

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