Trial · NCT01749540
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
NCT01749540, Phase 2, Completed, Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA; results first posted 2024-07-18
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ConductScience Library. Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002). Trials, record NCT01749540. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01749540
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
- NCT number
- NCT01749540
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 64 participants, actual
- Lead sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Conditions
- B-Thalassemia
- Interventions
- luspatercept
- First posted
- 2012-12-13
- Last updated
- 2024-07-18
- Other registries' IDs
- eudract:2012-002499-15
- Results first posted
- 2024-07-18
Source ClinicalTrials.gov
Registered outcomes58
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Up to approximately 20 weeks | An erythroid response in NTD participants was defined as a mean hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days in the absence of blood transfusion. Baseline hemoglobin for NTD participants was the average of two or more measurements performed during the screening period. Hemoglobin measurements within 2 weeks following red blood cell (RBC) transfusion or 56 days after the last dose were excluded from analysis. NTD participants were participants who had received \<4 units of RBCs within 8 weeks prior to the first dose of study drug. The percentage of participants with a hemoglobin increase of ≥1.5 g/dL from baseline for ≥14 days is presented. |
| Primary | Percentage of Transfusion Dependent (TD) Participants With a ≥20% Reduction in Red Blood Cell (RBC) Transfusion Burden From Baseline During a Rolling 12-week Interval | Any 12-week interval during the study (up to approximately 20 weeks) | Transfusion burden for TD participants was defined as the ratio of RBC transfusion units (or milliliters) transfused during a 12-week interval divided by the duration of that interval compared to the ratio of RBC transfusion units 12 weeks prior to the start of treatment (baseline). The interval during the pretreatment period was defined as the 12 weeks prior to the first dose of study drug. An interval during the treatment plus follow-up period was defined as any 12-week interval after the first dose of study drug. TD participants were participants who had received ≥4 units of RBCs every 8 weeks (confirmed over 6 months prior to the first dose of study drug). The percentage of participants with a ≥20% reduction in RBC transfusion burden from baseline is presented. |
| Secondary | Number of Participants Who Experienced an Adverse Event (AE) | Up to approximately 20 weeks | An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who experienced an AE is presented. |
| Secondary | Number of Participants Who Experienced a Serious Adverse Event (SAE) | Up to approximately 20 weeks | An SAE was any adverse event, occurring at any dose level/regimen and regardless of causality that: resulted in death; was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; was a congenital anomaly/birth defect; or was an important medical event. The number of participants who experienced an SAE is presented. |
| Secondary | Number of Participants Who Experienced an Adverse Event (AE) of Grade 3 or Greater | Up to approximately 20 weeks | AEs were graded using the National Cancer Institute Common Toxicity Criteria for Adverse Events, Version 4.0 (NCI-CTCAE v4.0) and graded as follows: Grade 1=mild; Grade 2=moderate; Grade 3=severe or medically significant but not immediately life-threatening; Grade 4=life-threatening consequences; and Grade 5=death related to AE. An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who experienced an AE of ≥Grade 3 is presented. |
| Secondary | Number of Participants Who Discontinued Study Treatment Due To an Adverse Event (AE) | Up to approximately 12 weeks | An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a study drug, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study drug whether or not it is considered related to the study drug. The number of participants who discontinued study treatment due to an AE is presented. |
| Secondary | Number of Participants Who Experienced a Dose-Limiting Toxicity (DLT) | Up to 28 days | DLTs were determined using the National Cancer Institute Common Toxicity Criteria for Adverse Events, Version 4.0 (NCI-CTCAE v4.0) and graded as follows: Grade 1=mild; Grade 2=moderate; Grade 3=severe or medically significant but not immediately life-threatening; Grade 4=life-threatening consequences; and Grade 5=death related to AE. The occurrence of any of the following toxicities occurring up to 28 days after the first dose was considered a DLT: treatment-related serious adverse event (SAE) ≥ Grade 3; treatment related non-hematologic adverse event (AE) ≥ Grade 3; and treatment related hematologic AE ≥ Grade 4. The number of participants who experienced a DLT is presented. |
| Secondary | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.0 g/dL From Baseline During a Rolling 8-week Interval | Any 8-week interval during the study (up to approximately 20 Weeks) | A modified erythroid response in NTD participants was defined as a mean hemoglobin increase of ≥1.0 g/dL from baseline during an 8-week interval compared to baseline. Baseline hemoglobin for NTD participants was the average of two or more measurements performed during the screening period. Hemoglobin measurements within 2 weeks following red blood cell (RBC) transfusion or 56 days after the last dose were excluded from analysis. NTD participants were participants who had received \<4 units of red blood cells (RBCs) within 8 weeks prior to the first dose of study drug. An 8-week interval was defined as any consecutive 8 weeks during the study. The percentage of participants with a hemoglobin increase of ≥1.0 g/dL from baseline is presented. |
| Secondary | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline During a Rolling 8-week Interval | Any 8-week interval during the study (up to approximately 20 Weeks) | A modified erythroid response in NTD participants was defined as a mean hemoglobin increase of ≥1.5 g/dL from baseline during an 8-week interval compared to baseline. Baseline hemoglobin for NTD participants was the average of two or more measurements performed during the screening period. Hemoglobin measurements within 2 weeks following red blood cell (RBC) transfusion or 56 days after the last dose were excluded from analysis. NTD participants were participants who had received \<4 units of red blood cells (RBCs) within 8 weeks prior to the first dose of study drug. An 8-week interval was defined as any consecutive 8 weeks during the study. The percentage of participants with a hemoglobin increase of ≥1.5 g/dL from baseline is presented. |
| Secondary | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.0 g/dL From Baseline During a Rolling 12-week Interval | Any 12-week interval during the study (up to approximately 20 Weeks) | A modified erythroid response in NTD participants was defined as a mean hemoglobin increase of ≥1.0 g/dL from baseline during a 12-week interval compared to baseline. Baseline hemoglobin for NTD participants was the average of two or more measurements performed during the screening period. Hemoglobin measurements within 2 weeks following red blood cell (RBC) transfusion or 56 days after the last dose were excluded from analysis. NTD participants were participants who had received \<4 units of red blood cells (RBCs) within 8 weeks prior to the first dose of study drug. A 12-week interval was defined as any consecutive 12 weeks during the study. The percentage of participants with a hemoglobin increase of ≥1.0 g/dL from baseline is presented. |
1 to 10 of 58Source ClinicalTrials.gov
See all 58 registered outcomesOutcomes58
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Up to approximately 20 weeks | All participants who received ≥1 dose of study treatment and received \<4 units of RBCs within 8 weeks prior to the first dose of study drug and had data available for the analyses | Percentage of participants | NUMBER |
| PRIMARY | Percentage of Transfusion Dependent (TD) Participants With a ≥20% Reduction in Red Blood Cell (RBC) Transfusion Burden From Baseline During a Rolling 12-week Interval | Any 12-week interval during the study (up to approximately 20 weeks) | All participants who received ≥1 dose of study treatment and received ≥4 units of RBCs every 8 weeks (confirmed over 6 months prior to the first dose of study drug) and had data available for the analyses | Percentage of participants | NUMBER |
| SECONDARY | Number of Participants Who Experienced an Adverse Event (AE) | Up to approximately 20 weeks | All participants who received ≥1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Experienced a Serious Adverse Event (SAE) | Up to approximately 20 weeks | All participants who received ≥1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Experienced an Adverse Event (AE) of Grade 3 or Greater | Up to approximately 20 weeks | All participants who received ≥1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Discontinued Study Treatment Due To an Adverse Event (AE) | Up to approximately 12 weeks | All participants who received ≥1 dose of study treatment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Who Experienced a Dose-Limiting Toxicity (DLT) | Up to 28 days | The DLT analysis population consisted of all participants who received ≥1 dose of study treatment and were observed for DLTs for 28 days after the first dose. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.0 g/dL From Baseline During a Rolling 8-week Interval | Any 8-week interval during the study (up to approximately 20 Weeks) | All participants who received ≥1 dose of study treatment and received \<4 units of RBCs within 8 weeks prior to the first dose of study drug and had data available for the analyses | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline During a Rolling 8-week Interval | Any 8-week interval during the study (up to approximately 20 Weeks) | All participants who received ≥1 dose of study treatment and received \<4 units of RBCs within 8 weeks prior to the first dose of study drug and had data available for the analyses | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.0 g/dL From Baseline During a Rolling 12-week Interval | Any 12-week interval during the study (up to approximately 20 Weeks) | All participants who received ≥1 dose of study treatment and received \<4 units of RBCs within 8 weeks prior to the first dose of study drug and had data available for the analyses | Percentage of participants | NUMBER |
1 to 10 of 58Source ClinicalTrials.gov
See all 58 outcomesResults by group337
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 0.2 mg/kg | 0.0 | 95% Confidence Interval 0.0 to 45.9 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 0.4 mg/kg | 0.0 | 95% Confidence Interval 0.0 to 45.9 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 0.6 mg/kg | 0.0 | 95% Confidence Interval 0.0 to 52.2 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 0.8 mg/kg | 66.7 | 95% Confidence Interval 9.4 to 99.2 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 1.0 mg/kg | 50.0 | 95% Confidence Interval 1.3 to 98.7 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Luspatercept 1.25 mg/kg | 0.0 | 95% Confidence Interval 0.0 to 97.5 | Percentage of participants | |
| Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days | Expansion Cohort | 60.0 | 95% Confidence Interval 26.2 to 87.8 | Percentage of participants | |
| Percentage of Transfusion Dependent (TD) Participants With a ≥20% Reduction in Red Blood Cell (RBC) Transfusion Burden From Baseline During a Rolling 12-week Interval | Luspatercept 0.6 mg/kg | 100 | 95% Confidence Interval 2.5 to 100 | Percentage of participants | |
| Percentage of Transfusion Dependent (TD) Participants With a ≥20% Reduction in Red Blood Cell (RBC) Transfusion Burden From Baseline During a Rolling 12-week Interval | Luspatercept 0.8 mg/kg | 66.7 | 95% Confidence Interval 9.4 to 99.2 | Percentage of participants | |
| Percentage of Transfusion Dependent (TD) Participants With a ≥20% Reduction in Red Blood Cell (RBC) Transfusion Burden From Baseline During a Rolling 12-week Interval | Luspatercept 1.0 mg/kg | 100 | 95% Confidence Interval 39.8 to 100 | Percentage of participants |
1 to 10 of 337Source ClinicalTrials.gov
See all 337 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Luspatercept 0.2 mg/kg | 6 | |
| Overall Study | STARTED | Luspatercept 0.4 mg/kg | 6 | |
| Overall Study | STARTED | Luspatercept 0.6 mg/kg | 6 | |
| Overall Study | STARTED | Luspatercept 0.8 mg/kg | 6 | |
| Overall Study | STARTED | Luspatercept 1.0 mg/kg | 6 | |
| Overall Study | STARTED | Luspatercept 1.25 mg/kg | 5 | |
| Overall Study | STARTED | Expansion Cohort | 29 | |
| Overall Study | COMPLETED | Luspatercept 0.2 mg/kg | 6 | |
| Overall Study | COMPLETED | Luspatercept 0.4 mg/kg | 6 | |
| Overall Study | COMPLETED | Luspatercept 0.6 mg/kg | 6 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events644
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 0.2 mg/kg | 0 | 6 | 0 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 0.4 mg/kg | 2 | 6 | 2 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 0.6 mg/kg | 0 | 6 | 0 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 0.8 mg/kg | 0 | 6 | 0 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 1.0 mg/kg | 0 | 6 | 0 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Luspatercept 1.25 mg/kg | 0 | 5 | 0 |
| other | Bone marrow failure | Blood and lymphatic system disorders | Expansion Cohort | 0 | 29 | 0 |
| other | Erythroblastosis | Blood and lymphatic system disorders | Luspatercept 0.2 mg/kg | 0 | 6 | 0 |
| other | Erythroblastosis | Blood and lymphatic system disorders | Luspatercept 0.4 mg/kg | 0 | 6 | 0 |
| other | Erythroblastosis | Blood and lymphatic system disorders | Luspatercept 0.6 mg/kg | 0 | 6 | 0 |
1 to 10 of 644Source ClinicalTrials.gov
See all 644 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Luspatercept 0.2 mg/kg | 0 | 6 |
| Total, all-cause mortality | Luspatercept 0.4 mg/kg | 0 | 6 |
| Total, all-cause mortality | Luspatercept 0.6 mg/kg | 0 | 6 |
| Total, all-cause mortality | Luspatercept 0.8 mg/kg | 0 | 6 |
| Total, all-cause mortality | Luspatercept 1.0 mg/kg | 0 | 6 |
| Total, all-cause mortality | Luspatercept 1.25 mg/kg | 0 | 5 |
| Total, all-cause mortality | Expansion Cohort | 0 | 29 |
| Total, other adverse events | Luspatercept 0.2 mg/kg | 5 | 6 |
| Total, other adverse events | Luspatercept 0.4 mg/kg | 6 | 6 |
| Total, other adverse events | Luspatercept 0.6 mg/kg | 5 | 6 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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