Trial · NCT02604433
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
NCT02604433, Phase 3, Completed, Celgene; results first posted 2020-01-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. 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. Trials, record NCT02604433. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02604433
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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
- NCT number
- NCT02604433
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 336 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Erythrocyte Transfusion, Beta-Thalassemia
- Interventions
- Luspatercept, Placebo
- First posted
- 2015-11-13
- Last updated
- 2023-04-18
- Results first posted
- 2020-01-27
Source ClinicalTrials.gov
Registered outcomes28
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Erythroid Response was defined as red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Week 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 33% with a reduction of at least 2 units during Weeks 37 - 48 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Weeks 13 - 24 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | Percentage of participants who achieved a red blood cell (RBC) transfusion burden reduction from baseline ≥ 50% with a reduction of at least 2 units during Week 37 to Week 48 compared to the 12-week interval on or prior to Dose 1 Day 1. |
| Secondary | Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | Baseline was defined as the total number of Red Blood Cells (RBC) units transfused during the 12-week interval on or prior to Dose 1 Day 1. This is compared to the total number of RBC units transfused during the 12-week interval from treatment weeks 13-24. |
| Secondary | Mean Change From Baseline In Liver Iron Concentration (LIC) At Week 48 | Baseline: Week -12 to Day -1; Treatment: Week 48 | Baseline was defined as the last value on or before the first dose of study drug was administered; if multiple values were present for the same date, the average of these values was used. If a participant had 1 postbaseline assessment, it was used as the Week 48 value. If a participant had multiple postbaseline assessments, the last one was used as the Week 48 value. The value of LIC was collected by magnetic resonance imaging. Participants with a LIC value \> 43 mg/g were not included in the analysis. |
| Secondary | Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | Three different types of Iron Chelation Therapy (ICT) were analyzed: 1. Deferasirox 2. Deferiprone 3. Deferoxamine Mesilate/Deferoxamine The baseline mean daily dose was calculated using the ICT dosage during the 12 weeks prior to first study drug administration and the postbaseline mean daily dose was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or the last 12 weeks of the study treatment for early discontinued participants. |
| Secondary | Mean Change From Baseline In Mean Serum Ferritin At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | For each participant, the baseline mean serum ferritin level was calculated during the 12 weeks prior to first study drug administration. The postbaseline mean serum ferritin level was calculated during the last 12 weeks of the 48-week double-blind Treatment Period or last 12 weeks of study treatment, if discontinued early. The change was calculated as the difference of post baseline mean serum ferritin level and baseline mean serum ferritin level. |
| Secondary | Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48 | Baseline: Day 1; Treatment: Week 48 | For BMD, the lumbar spine and total hip were measured at baseline and 48 weeks by dual energy x-ray absorptiometry (DXA). Baseline was defined as the last value on or before the first dose of study drug is administered; if multiple values are present for the same date, the average of these values was used. If during the 48 week double-blinded treatment period, a participant has only one assessment, it is counted as 'Week 48' visit; if a participant has multiple assessments, the last one is used as 'Week 48' visit. The analysis was done on the population that had at least 2 measurements. |
| Secondary | Mean Change From Baseline In Myocardial Iron At Week 48 | Baseline: Day 1; Treatment: Week 48 | Myocardial Iron levels were measured by Magnetic Resonance Imaging (MRI), using MRI parameter T2\* (Unit: ms). T2\* values correlates with heart failure (HF) risk (e.g. T2\*\<6ms: high HF risk). |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 registered outcomesOutcomes28
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | All randomized participants | Percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Weeks 37 to Week 48 | All randomized participants | Percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | All randomized participants | Percentage of participants | NUMBER |
| SECONDARY | Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | All randomized participants | Percentage of participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24 | Baseline: Day -83 to Day 1; Treatment: Weeks 13 to Week 24 | All randomized participants with available measurements at the specified timepoints | RBC units | MEAN |
| SECONDARY | Mean Change From Baseline In Liver Iron Concentration (LIC) At Week 48 | Baseline: Week -12 to Day -1; Treatment: Week 48 | All randomized participants with available measurements at the specified timepoints | mg/g dry weight | MEAN |
| SECONDARY | Mean Change From Baseline In Mean Daily Dose Of Iron Chelation Therapies (ICT) At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | All randomized participants with available measurements at the specified timepoints | mg | MEAN |
| SECONDARY | Mean Change From Baseline In Mean Serum Ferritin At Week 48 | Baseline: Day -83 to Day 1; Treatment: Week 37 to Week 48 | All randomized participants with available measurements at the specified timepoints | μg/L | MEAN |
| SECONDARY | Mean Change From Baseline In Total Hip And Lumbar Spine Bone Mineral Density (BMD) At Week 48 | Baseline: Day 1; Treatment: Week 48 | All randomized participants with available measurements at the specified timepoints | gm/cm^2 | MEAN |
| SECONDARY | Mean Change From Baseline In Myocardial Iron At Week 48 | Baseline: Day 1; Treatment: Week 48 | All randomized participants with available measurements at the specified timepoints | ms | MEAN |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 outcomesResults by group103
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 | Luspatercept + BSC | 21.0 | Percentage of participants | ||
| Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 | Placebo + BSC | 4.5 | Percentage of participants | ||
| Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Luspatercept + BSC | 19.6 | Percentage of participants | ||
| Percentage Of Participants Who Achieved ≥ 33% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Placebo + BSC | 3.6 | Percentage of participants | ||
| Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24 | Luspatercept + BSC | 7.1 | Percentage of participants | ||
| Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 13 to Week 24 | Placebo + BSC | 1.8 | Percentage of participants | ||
| Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Luspatercept + BSC | 10.3 | Percentage of participants | ||
| Percentage Of Participants Who Achieve ≥ 50% Reduction From Baseline in Transfusion Burden - Week 37 to Week 48 | Placebo + BSC | 0.9 | Percentage of participants | ||
| Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24 | Luspatercept + BSC | -0.67 | Standard Deviation 1.792 | RBC units | |
| Mean Change From Baseline in Transfusion Burden - Week 13 to Week 24 | Placebo + BSC | 0.66 | Standard Deviation 1.774 | RBC units |
1 to 10 of 103Source ClinicalTrials.gov
See all 103 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Luspatercept + BSC | 224 | |
| Overall Study | STARTED | Placebo + BSC | 112 | |
| Overall Study | Participants Treated | Luspatercept + BSC | 223 | |
| Overall Study | Participants Treated | Placebo + BSC | 109 | |
| Overall Study | Completed 24 Weeks of Treatment | Luspatercept + BSC | 211 | |
| Overall Study | Completed 24 Weeks of Treatment | Placebo + BSC | 102 | |
| Overall Study | Completed 48 Weeks of Treatment | Luspatercept + BSC | 202 | |
| Overall Study | Completed 48 Weeks of Treatment | Placebo + BSC | 96 | |
| Overall Study | Completed 96 Weeks of Treatment | Luspatercept + BSC | 155 | |
| Overall Study | Completed 96 Weeks of Treatment | Placebo + BSC | 3 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events252
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 | Abdominal pain | Gastrointestinal disorders | Luspatercept + BSC | 30 | 223 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo + BSC | 7 | 109 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Luspatercept + BSC | 25 | 223 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo + BSC | 8 | 109 | |
| other | Constipation | Gastrointestinal disorders | Luspatercept + BSC | 12 | 223 | |
| other | Constipation | Gastrointestinal disorders | Placebo + BSC | 4 | 109 | |
| other | Diarrhoea | Gastrointestinal disorders | Luspatercept + BSC | 41 | 223 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo + BSC | 14 | 109 | |
| other | Dyspepsia | Gastrointestinal disorders | Luspatercept + BSC | 19 | 223 | |
| other | Dyspepsia | Gastrointestinal disorders | Placebo + BSC | 1 | 109 |
1 to 10 of 252Source ClinicalTrials.gov
See all 252 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Luspatercept + BSC | 4 | 223 |
| Total, all-cause mortality | Placebo + BSC | 1 | 109 |
| Total, other adverse events | Luspatercept + BSC | 211 | 223 |
| Total, other adverse events | Placebo + BSC | 95 | 109 |
| Total, serious adverse events | Luspatercept + BSC | 53 | 223 |
| Total, serious adverse events | Placebo + BSC | 8 | 109 |
Source ClinicalTrials.gov
Papers7
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Luspatercept + BSC vs Placebo + BSC: deaths from any cause | Trial comparisons |
| Luspatercept + BSC vs Placebo + BSC: serious adverse events | Trial comparisons |
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02604433
GET https://conductscience.org/api/trials/v1/NCT02604433/registered?page=2