Trial · NCT03682536
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
NCT03682536, Phase 3, Active not recruiting, Celgene; results first posted 2024-06-26
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve. Trials, record NCT03682536. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03682536
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve
- NCT number
- NCT03682536
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 363 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Myelodysplastic Syndromes
- Interventions
- Luspatercept, Epoetin alfa
- First posted
- 2018-09-24
- Last updated
- 2024-11-20
- Results first posted
- 2024-06-26
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL | Week 1 through Week 24 | Percentage of participants who are RBC transfusion-free for any 12-week period associated with a concurrent mean hemoglobin (Hgb) increase ≥ 1.5 g/dL compared to baseline. After applying below 14/3-day rule, the baseline Hgb value is defined as the lowest Hgb value from the central, local laboratory, or pre transfusion Hgb from transfusion records that is within 56 days on or prior to the first dose of treatment, or randomization date if participants were not treated. 4/3-day rule: only Hgb values that are at least 14 days after a transfusion may be used unless there is another transfusion within 3 days after the Hgb assessment. If this occurs, that Hgb value will be used despite being \< 14 days after the previous transfusion. |
| Secondary | Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 24 Weeks | Week 1 through Week 24 | Red blood cell transfusion independence (RBC-TI) for 24 weeks is defined as the percentage of participants who did not receive RBC transfusions from Week 1 through Week 24. |
| Secondary | Mean Hemoglobin Change Over 24 Weeks | Week 1 through Week 24 | Mean hemoglobin (Hgb) change over the 24-week period of Week 1 through Week 24 compared to baseline. After applying below 14/3-day rule, the baseline Hgb value is defined as the lowest Hgb value from the central, local laboratory, or pre transfusion Hgb from transfusion records that is within 56 days on or prior to the first dose of treatment, or randomization date if participants were not treated. 4/3-day rule: only Hgb values that are at least 14 days after a transfusion may be used unless there is another transfusion within 3 days after the Hgb assessment. If this occurs, that Hgb value will be used despite being \< 14 days after the previous transfusion. |
| Secondary | Percentage of Participants Achieving Hematologic Improvement - Erythroid Response (HI-E) Per IWG | Week 1 through Week 24 | The percentage of participants meeting the modified HI-E criteria per the International Working Group (IWG) sustained over any consecutive 56-day period in Week 1-24: For participants with baseline red blood cell (RBC) transfusion burden of \>= 4 units/8 weeks, a reduction of at least 4 units RBC transfusion; for participants with baseline RBC transfusion burden of \< 4 units/8 weeks, mean increase of hemoglobin of at least 1.5 g/dL in the absence of transfusions. |
| Secondary | Time to Hematologic Improvement - Erythroid Response (HI-E) | Week 1 through Week 24 | Time from first dose to first onset of achieving modified HI-E. The modified HI-E criteria per the International Working Group (IWG) sustained over any consecutive 56-day period in Week 1-24: For participants with baseline red blood cell (RBC) transfusion burden of \>= 4 units/8 weeks, a reduction of at least 4 units RBC transfusion; for participants with baseline RBC transfusion burden of \< 4 units/8 weeks, mean increase of hemoglobin of at least 1.5 g/dL in the absence of transfusions. |
| Secondary | Percentage of Participants Achieving Red Blood Cell Transfusion Independence (RBC-TI) for ≥ 12 Weeks (84 Days) | Week 1 through Week 24 | Percentage of participants who are RBC transfusion-free over a consecutive 84-day period. |
| Secondary | Duration of Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks (84 Days) | Week 1 through End of Treatment (Up to approximately an average of 66 weeks and a maximum of 202 weeks) | Maximum duration of RBC transfusion independence for participants who achieve RBC-TI ≥ 84 days. |
| Secondary | Time to Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks (84 Days) | Week 1 through Week 24 | Time from first dose to first onset of transfusion independence ≥ 84 days. |
| Secondary | Time to First Red Blood Cell (RBC) Transfusion | Week 1 through End of Treatment (Up to approximately an average of 66 weeks and a maximum of 202 weeks) | Time to first RBC transfusion is defined as time from Week 1 to first RBC transfusion on treatment. Participants who maintain RBC-TI through the end of the Treatment Period or time of analysis will be censored at EOT visit date, subsequent MDS therapy start date, study discontinuation date, analysis cutoff date or death, whichever occurs first. Median is from un-stratified Kaplan-Meier method. |
| Secondary | The Number of Red Blood Cell (RBC) Units Transfused Within the First 24 Weeks of Treatment | Week 1 through Week 24 | RBC transfusion burden on treatment is defined as total number of packed red blood cell (pRBC) units transfused within the first 24 weeks of treatment since Week 1. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes21
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL | Week 1 through Week 24 | ITT Population - all randomized participants regardless of whether or not the participant received treatment. | Percent of participants | NUMBER |
| SECONDARY | Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 24 Weeks | Week 1 through Week 24 | ITT Population - all randomized participants regardless of whether or not the participant received treatment. | Percentage of participants | NUMBER |
| SECONDARY | Mean Hemoglobin Change Over 24 Weeks | Week 1 through Week 24 | ITT Population (all randomized participants regardless of whether or not the participant received treatment) with baseline measurements. | g/dL | MEAN |
| SECONDARY | Percentage of Participants Achieving Hematologic Improvement - Erythroid Response (HI-E) Per IWG | Week 1 through Week 24 | ITT Population - all randomized participants regardless of whether or not the participant received treatment. | Percentage of participants | NUMBER |
| SECONDARY | Time to Hematologic Improvement - Erythroid Response (HI-E) | Week 1 through Week 24 | ITT Population (all randomized participants regardless of whether or not the participant received treatment) who achieve HI-E response (Week 1-24) | Days | MEDIAN |
| SECONDARY | Percentage of Participants Achieving Red Blood Cell Transfusion Independence (RBC-TI) for ≥ 12 Weeks (84 Days) | Week 1 through Week 24 | ITT Population - all randomized participants regardless of whether or not the participant received treatment. | Percent of participants | NUMBER |
| SECONDARY | Duration of Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks (84 Days) | Week 1 through End of Treatment (Up to approximately an average of 66 weeks and a maximum of 202 weeks) | ITT Population (all randomized participants regardless of whether or not the participant received treatment) who were RBC-TI \>= 12 Weeks (Week 1-24) responders | Weeks | MEDIAN |
| SECONDARY | Time to Red Blood Cell Transfusion Independence (RBC-TI) ≥ 12 Weeks (84 Days) | Week 1 through Week 24 | ITT Population (all randomized participants regardless of whether or not the participant received treatment) who were RBC-TI \>= 12 Weeks (Week 1-24) responders | Days | MEDIAN |
| SECONDARY | Time to First Red Blood Cell (RBC) Transfusion | Week 1 through End of Treatment (Up to approximately an average of 66 weeks and a maximum of 202 weeks) | ITT Population - all randomized participants regardless of whether or not the participant received treatment. | Days | MEDIAN |
| SECONDARY | The Number of Red Blood Cell (RBC) Units Transfused Within the First 24 Weeks of Treatment | Week 1 through Week 24 | All participants who were randomized and received at least one dose. | RBC units | MEAN |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 outcomesResults by group92
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL | Luspatercept | 60.4 | 95% Confidence Interval 52.9 to 67.6 | Percent of participants | |
| Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 12 Weeks (84 Days) With a Mean Hemoglobin Increase ≥ 1.5 g/dL | Epoetin Alfa | 34.8 | 95% Confidence Interval 27.9 to 42.2 | Percent of participants | |
| Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 24 Weeks | Luspatercept | 47.8 | 95% Confidence Interval 40.4 to 55.3 | Percentage of participants | |
| Percentage of Participants With Red Blood Cell Transfusion Independence (RBC-TI) for 24 Weeks | Epoetin Alfa | 30.9 | 95% Confidence Interval 24.3 to 38.2 | Percentage of participants | |
| Mean Hemoglobin Change Over 24 Weeks | Luspatercept | 2.0 | Standard Deviation 1.14 | g/dL | |
| Mean Hemoglobin Change Over 24 Weeks | Epoetin Alfa | 1.5 | Standard Deviation 1.12 | g/dL | |
| Percentage of Participants Achieving Hematologic Improvement - Erythroid Response (HI-E) Per IWG | Luspatercept | 74.2 | 95% Confidence Interval 67.2 to 80.4 | Percentage of participants | |
| Percentage of Participants Achieving Hematologic Improvement - Erythroid Response (HI-E) Per IWG | Epoetin Alfa | 53.0 | 95% Confidence Interval 45.5 to 60.5 | Percentage of participants | |
| Time to Hematologic Improvement - Erythroid Response (HI-E) | Luspatercept | 1.0 | Full Range 1.0 to 113.0 | Days | |
| Time to Hematologic Improvement - Erythroid Response (HI-E) | Epoetin Alfa | 6.0 | Full Range 1.0 to 108.0 | Days |
1 to 10 of 92Source ClinicalTrials.gov
See all 92 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Randomization | STARTED | Luspatercept | 182 | |
| Randomization | STARTED | Epoetin Alfa | 181 | |
| Randomization | COMPLETED | Luspatercept | 182 | |
| Randomization | COMPLETED | Epoetin Alfa | 179 | |
| Randomization | NOT COMPLETED | Luspatercept | 0 | |
| Randomization | NOT COMPLETED | Epoetin Alfa | 2 | |
| Treatment | STARTED | Luspatercept | 182 | |
| Treatment | STARTED | Epoetin Alfa | 179 | |
| Treatment | COMPLETED | Luspatercept | 78 | |
| Treatment | COMPLETED | Epoetin Alfa | 53 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events402
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 | Anaemia | Blood and lymphatic system disorders | Luspatercept | 20 | 182 | |
| other | Anaemia | Blood and lymphatic system disorders | Epoetin Alfa | 17 | 179 | |
| other | Neutropenia | Blood and lymphatic system disorders | Luspatercept | 10 | 182 | |
| other | Neutropenia | Blood and lymphatic system disorders | Epoetin Alfa | 14 | 179 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Luspatercept | 12 | 182 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Epoetin Alfa | 5 | 179 | |
| other | Constipation | Gastrointestinal disorders | Luspatercept | 11 | 182 | |
| other | Constipation | Gastrointestinal disorders | Epoetin Alfa | 12 | 179 | |
| other | Diarrhoea | Gastrointestinal disorders | Luspatercept | 32 | 182 | |
| other | Diarrhoea | Gastrointestinal disorders | Epoetin Alfa | 25 | 179 |
1 to 10 of 402Source ClinicalTrials.gov
See all 402 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Luspatercept | 39 | 182 |
| Total, all-cause mortality | Epoetin Alfa | 38 | 181 |
| Total, other adverse events | Luspatercept | 153 | 182 |
| Total, other adverse events | Epoetin Alfa | 134 | 179 |
| Total, serious adverse events | Luspatercept | 82 | 182 |
| Total, serious adverse events | Epoetin Alfa | 71 | 179 |
Source ClinicalTrials.gov
Papers4
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 comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Luspatercept vs Epoetin Alfa: The Number of Participants With Acute Myeloid Leukemia (AML) Progression | Trial comparisons |
| Luspatercept vs Epoetin Alfa: deaths from any cause | Trial comparisons |
| Luspatercept vs Epoetin Alfa: 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/NCT03682536
GET https://conductscience.org/api/trials/v1/NCT03682536/registered?page=2