Trial · NCT01736683
Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)
NCT01736683, Phase 2, Completed, Merck Sharp & Dohme LLC; results first posted 2019-06-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML). Trials, record NCT01736683. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01736683
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)
- NCT number
- NCT01736683
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 74 participants, actual
- Lead sponsor
- Merck Sharp & Dohme LLC
- Conditions
- Anemia, Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Low to Intermediate-1 MDS, Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML)
- Interventions
- Sotatercept
- First posted
- 2012-11-29
- Last updated
- 2022-10-24
- Other registries' IDs
- eudract:2012-002601-22
- Results first posted
- 2019-06-13
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Day 2 to Day 142 | The responder rate includes non-transfusion dependent efficacy (NTDE) participants and transfusion dependent efficacy (TDE) participants. For non-transfusion dependence efficacy (NTDE) participants who required \< 4 units of RBCs in the 8 weeks prior to start of therapy, HI-E was defined as an increase of \>=1.5 g/dL hemoglobin sustained for 56 days over a period of \>=8 weeks. For transfusion dependence efficacy (TDE) participants who required \>=4 units of RBCs in the 8 weeks prior to start of therapy, HI-E was defined as a decrease of \>= 4 units of RBCs transfused sustained for 56 days over a period of 8 weeks. |
| Secondary | Time to Erythroid Hematological Improvement (HI-E) Response | Day 1 to Day 87 | Time to first response = start date of first response (HI-E) - first dose date + 1 day. For NTDE participants (who required \< 4 units of RBCs in the 8 weeks prior to start of therapy), HI-E was defined as an increase of \>=1.5 g/dL hemoglobin sustained for 56 days over a period of \>=8 weeks. For TDE participants (who required \>=4 units of RBCs in the 8 weeks prior to start of therapy), HI-E was defined as a decrease of \>= 4 units of RBCs transfused sustained for 56 days over a period of 8 weeks. |
| Secondary | Duration of Erythroid Hematological Improvement (HI-E) | Day 1 to 183.7 weeks | The duration of HI-E response for participants who responded was (the last date of the consecutive hemoglobin \[Hgb\] measurements of the first \>=56 day interval) - (the first date of the consecutive Hgb measurements of the first \>=56 day interval) + 1 day. |
| Secondary | Time to Progression to Acute Myeloid Leukemia (AML) for Participants Who Had Progression | Day 1 to 183.7 weeks | Progression to AML used criteria by the International Working Group (IWG) Response Criteria in Myelodysplasia (Cheson, 2006). Progression is considered if any of the following are met: - \>=50% increase in blasts - \>=50% decrement from maximum remission/response levels in granulocytes or platelets - Reduction in Hgb concentration by \>=2 g/dL - Transfusion dependence This outcome was defined as a Kaplan-Meier estimate however few participants progressed so a Kaplan-Meier analysis could not be performed. Disclosed are time to progression values only for participants who did progress to AML. |
| Secondary | Time to Progression to Events of Higher Risk Myelodysplastic Syndromes (MDS) Using the International Prognostic Scoring System (IPSS) For Participants Who Had Progression | Day 1 to 257.3 weeks | Progression to events of higher risk MDS used criteria from the International Prognostic Scoring System for MDS (IPSS) which assigns a prognostic score (0=good and increasing in risk by half-grades with the top score outlined below) for three prognostic variables: - Marrow blasts (score 0-2.0) - Karyotype (score 0-1.0) - Cytopenias: neutrophil, platelets, and Hg counts (score 0-0.5) The three individual scores are summed resulting in a full range of 0- 3.5 and placed into risk categories 0 = low risk 0.5-1.0 = intermediate-1 risk 1.5-2.0 = intermediate-2 risk \>=2.5 = high risk This outcome was defined as a Kaplan-Meier estimate however few participants progressed so a Kaplan-Meier analysis could not be performed. Data reported represent event times (weeks) for participants who did progress to higher risk MDS categories. |
| Secondary | Kaplan-Meier Estimates for Progression-free Survival | Day 1 to 257.3 weeks | Participants who had disease progression were considered to have events. Participants who died without acute myeloid leukemia (AML) were also considered to have events with the event date as the date of death. Those who did not have disease progression and who were lost to follow-up were censored at the last known disease progression assessment date. Participants without disease progression at the last follow-up contact were censored at the date of the last follow-up contact date. Disease Progression to AML used criteria by the International Working Group (IWG) Response Criteria in Myelodysplasia (Cheson, 2006). Progression is considered if any of the following are met: - \>=50% increase in blasts - \>=50% decrement from maximum remission/response levels in granulocytes or platelets - Reduction in hemoglobin (Hgb) concentration by \>=2 g/dL - Transfusion dependence |
| Secondary | Kaplan-Meier Estimates for Overall Survival (OS) | Day 1 to 257.3 weeks | OS was defined as the time between start of treatment and the death/censored date. Participants who died (regardless of the cause of death) were considered to have an event. Participants who were alive at the end of the study, and participants who were lost to follow-up, were censored at the last date when subjects were known to be alive. |
| Secondary | Pharmacokinetic Parameters of Sotatercept: Serum Concentration at Various Study Timepoints | Cycle 1 Day 8 and !5 up to Cycle 2 Day 1 | Maximum observed serum concentration, obtained directly from the observed concentration versus time data. |
| Secondary | Participants With Treatment-Emergent Adverse Events (TEAE) | Day 1 up to 59.2 months | An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen in a subject during the course of a study. Treatment-emergent adverse events (TEAEs) are defined as any AE occurring or worsening on or after the first treatment of the study medication and within 42 days after the last dose. The severity of AEs was graded based on National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), Version 4.0 and the scale: Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Life threatening Grade 5 = Death. Relation to study drug was determined by the investigator. A treatment-related TEAE is defined as TEAE which was considered to be related to the study drug and reported as 'Suspected' on the CRF. AEs with a missing relationship were treated as 'treatment-related' in data summaries. |
| Secondary | Dose Limiting Toxicities (DLTs) | Day 1 to 59.2 months | The following were DLTs if the investigator suspected they were treatment related: 1. Increase to \>= 140 mmHg systolic blood pressure 2. Increase to \>=90 mmHg diastolic blood pressure 3. Increase to \>=140 systolic and increase \> 20 mmHg compared to baseline systolic 4. Increase to \>=90 mmHg diastolic and increase \> 20 mmHg compared to baseline diastolic 5. Introduction of new anti-hypertension medication during treatment 6. Increase in dose of baseline anti-hypertension medication during treatment 7. \>= Grade 2 (moderate severity or worse) hypertension as an adverse event |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
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 Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Day 2 to Day 142 | Efficacy Evaluable Population: all participants who take at least one dose of study medication and have baseline and at least one post-baseline assessment of efficacy without major deviation from protocol. | percentage of participants | NUMBER |
| SECONDARY | Time to Erythroid Hematological Improvement (HI-E) Response | Day 1 to Day 87 | Efficacy Evaluable Population of participants who responded | days | MEDIAN |
| SECONDARY | Duration of Erythroid Hematological Improvement (HI-E) | Day 1 to 183.7 weeks | Efficacy Evaluable Population of participants who responded | days | MEDIAN |
| SECONDARY | Time to Progression to Acute Myeloid Leukemia (AML) for Participants Who Had Progression | Day 1 to 183.7 weeks | Efficacy Evaluable Population of participants who progressed to AML | weeks | NUMBER |
| SECONDARY | Time to Progression to Events of Higher Risk Myelodysplastic Syndromes (MDS) Using the International Prognostic Scoring System (IPSS) For Participants Who Had Progression | Day 1 to 257.3 weeks | Efficacy Evaluable Population of participants who progressed to high risk MDS categories | weeks | NUMBER |
| SECONDARY | Kaplan-Meier Estimates for Progression-free Survival | Day 1 to 257.3 weeks | Efficacy Evaluable Population | weeks | MEDIAN |
| SECONDARY | Kaplan-Meier Estimates for Overall Survival (OS) | Day 1 to 257.3 weeks | Efficacy Evaluable Population | weeks | MEDIAN |
| SECONDARY | Pharmacokinetic Parameters of Sotatercept: Serum Concentration at Various Study Timepoints | Cycle 1 Day 8 and !5 up to Cycle 2 Day 1 | Pharmacokinetic (PK) population includes participants with a sufficient amount of post-dose quantifiable PK sotatercept profile. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Participants With Treatment-Emergent Adverse Events (TEAE) | Day 1 up to 59.2 months | Safety population: all participants who receive at least one dose of study medication. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Dose Limiting Toxicities (DLTs) | Day 1 to 59.2 months | Safety population | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group168
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 Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.1 mg/kg | All participants | 0 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.3 mg/kg | All participants | 66.7 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.5 mg/kg | All participants | 42.9 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 1.0 mg/kg | All participants | 60.0 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 2.0 mg/kg | All participants | 40.0 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.5 mg/kg | NTDE subpopulation | 33.3 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 1.0 mg/kg | NTDE subpopulation | 62.5 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 2.0 mg/kg | NTDE subpopulation | 100 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.1 mg/kg | TDE subpopulation | 0 | percentage of participants | |
| Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate) | Sotatercept 0.3 mg/kg | TDE subpopulation | 66.7 | percentage of participants |
1 to 10 of 168Source ClinicalTrials.gov
See all 168 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sotatercept 0.1 mg/kg | 7 | |
| Overall Study | STARTED | Sotatercept 0.3 mg/kg | 6 | |
| Overall Study | STARTED | Sotatercept 0.5 mg/kg | 21 | |
| Overall Study | STARTED | Sotatercept 1.0 mg/kg | 35 | |
| Overall Study | STARTED | Sotatercept 2.0 mg/kg | 5 | |
| Overall Study | COMPLETED | Sotatercept 0.1 mg/kg | 0 | |
| Overall Study | COMPLETED | Sotatercept 0.3 mg/kg | 0 | |
| Overall Study | COMPLETED | Sotatercept 0.5 mg/kg | 0 | |
| Overall Study | COMPLETED | Sotatercept 1.0 mg/kg | 0 | |
| Overall Study | COMPLETED | Sotatercept 2.0 mg/kg | 0 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events715
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 | Sotatercept 0.1 mg/kg | 0 | 7 | |
| other | Anaemia | Blood and lymphatic system disorders | Sotatercept 0.3 mg/kg | 0 | 6 | |
| other | Anaemia | Blood and lymphatic system disorders | Sotatercept 0.5 mg/kg | 0 | 21 | |
| other | Anaemia | Blood and lymphatic system disorders | Sotatercept 1.0 mg/kg | 2 | 35 | |
| other | Anaemia | Blood and lymphatic system disorders | Sotatercept 2.0 mg/kg | 0 | 5 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Sotatercept 0.1 mg/kg | 0 | 7 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Sotatercept 0.3 mg/kg | 1 | 6 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Sotatercept 0.5 mg/kg | 0 | 21 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Sotatercept 1.0 mg/kg | 0 | 35 | |
| other | Haemolytic anaemia | Blood and lymphatic system disorders | Sotatercept 2.0 mg/kg | 0 | 5 |
1 to 10 of 715Source ClinicalTrials.gov
See all 715 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sotatercept 0.1 mg/kg | 1 | 7 |
| Total, all-cause mortality | Sotatercept 0.3 mg/kg | 1 | 6 |
| Total, all-cause mortality | Sotatercept 0.5 mg/kg | 6 | 21 |
| Total, all-cause mortality | Sotatercept 1.0 mg/kg | 6 | 35 |
| Total, all-cause mortality | Sotatercept 2.0 mg/kg | 1 | 5 |
| Total, other adverse events | Sotatercept 0.1 mg/kg | 6 | 7 |
| Total, other adverse events | Sotatercept 0.3 mg/kg | 4 | 6 |
| Total, other adverse events | Sotatercept 0.5 mg/kg | 18 | 21 |
| Total, other adverse events | Sotatercept 1.0 mg/kg | 32 | 35 |
| Total, other adverse events | Sotatercept 2.0 mg/kg | 5 | 5 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 29331635 | DERIVED |
Source PubMed
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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