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NCT01736683 · REGISTRY CONDITION SEARCH

Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase
PHASE2
Status at capture
COMPLETED
Registry last update
2022-10-24

The primary objective of this study is to determine a safe, tolerable and effective dose of sotatercept that results in the greatest frequency of improvement of anemia in patients diagnosed with low- or intermediate-1 risk myelodysplastic syndromes (MDS) or non-proliferative chronic myelomonocytic leukemia (CMML).

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate)

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outcome · POSTED

Time to Erythroid Hematological Improvement (HI-E) Response

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outcome · POSTED

Duration of Erythroid Hematological Improvement (HI-E)

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outcome · POSTED

Time to Progression to Acute Myeloid Leukemia (AML) for Participants Who Had Progression

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outcome · POSTED

Time to Progression to Events of Higher Risk Myelodysplastic Syndromes (MDS) Using the International Prognostic Scoring System (IPSS) For Participants Who Had Progression

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outcome · POSTED

Kaplan-Meier Estimates for Progression-free Survival

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outcome · POSTED

Kaplan-Meier Estimates for Overall Survival (OS)

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outcome · POSTED

Pharmacokinetic Parameters of Sotatercept: Serum Concentration at Various Study Timepoints

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outcome · POSTED

Participants With Treatment-Emergent Adverse Events (TEAE)

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outcome · POSTED

Dose Limiting Toxicities (DLTs)

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outcome · POSTED

Number of Participants Who Achieved Red Blood Cell (RBC)-Transfusion Independence During the Erythroid Hematological Improvement (HI-E) Interval

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
eligibility Criteria
Inclusion Criteria: * Men and women ≥ 18 years of age * Documented diagnosis of myelodysplastic syndromes (MDS) or non-proliferative chronic myelomonocytic leukemia (CMML), white blood cells (WBC) ≤ 13,000 /mm\^3, World Health Organization (WHO) that meets International Prognostic Scoring System (IPSS) criteria for low or intermediate-1 risk disease * Anemia, Hemoglobin (Hgb) ≤ 9.0 g/dL or ≥ 2 units of Red Blood Cells (RBCs) within 84 days * No response or loss of response to Erythropoiesis-Stimulating Agents (ESAs) or erythropoetin (EPO) \> 500 mU/ml * Eastern Cooperative Group (ECOG) score ≤2. * Creatinine \< 1.5 \* Upper Limit of the Normal (ULN) * Total bilirubin ≤3.0 mg/dL * Aspartate aminotransferase (AST)/Serum glutamic oxaloacetic transaminase (SGOT) \& Alanine Aminotransferase (ALT)/Serum Glutamic Pyruvic (SGPT) ≤3.0 \* Upper Limit of Norma (ULN) * Free of metastatic malignancy (other than MDS) for ≥2 years * Highly effective methods of birth control for females and males Exclusion Criteria: * Chromosome 5q deletion * Pregnant or breast feeding women and males who do not agree to use condom during the sexual contact with females of childbearing potential * Major surgery within 30 days * Incomplete recovery or incomplete healing of wounds from previous surgery * Heart failure ≥3 (New York Heart Association (NYHA)) * Thromboembolic or myocardial infarction event within 6 months * Concurrent anti-cancer cytotoxic chemotherapy * History of severe allergic or anaphylactic reaction or hypersensitivity to recombinant protein * Known positive for Human Immunovirus (HIV) or infectious Hepatitis type C or active infectious Hepatitis type B * Clinically significant anemia unrelated to MDS * Thrombocytopenia (\<30,000/uL) * Uncontrolled hypertension * Treatment with another investigational drug or device within 28 days prior to Day 1 * Prior exposure to sotatercept (ACE-011) * Any serious medical condition, lab abnormality or psychiatric illness
healthy Volunteers
false
minimum Age
18 Years
sex
ALL
std Ages
  1. ADULT
  2. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Sotatercept 0.1 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 0.1 mg/kg
    type
    EXPERIMENTAL
  2. description
    Sotatercept 0.3 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 0.3 mg/kg
    type
    EXPERIMENTAL
  3. description
    Sotatercept 0.5 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 0.5 mg/kg
    type
    EXPERIMENTAL
  4. description
    Sotatercept 1.0 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 1.0 mg/kg
    type
    EXPERIMENTAL
  5. description
    Sotatercept 1.5 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 1.5 mg/kg
    type
    EXPERIMENTAL
  6. description
    Sotatercept 2.0 mg/kg
    intervention Names
    1. Drug: Sotatercept
    label
    Sotatercept 2.0 mg/kg
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Sotatercept 0.1 mg/kg
    2. Sotatercept 0.3 mg/kg
    3. Sotatercept 0.5 mg/kg
    4. Sotatercept 1.0 mg/kg
    5. Sotatercept 1.5 mg/kg
    6. Sotatercept 2.0 mg/kg
    description
    Sotatercept is supplied as a lyophilized powder that is reconstituted using Water for Injection (WFI) and administered as a subcutaneous injection (SC) injection by the study staff at the clinical site.
    name
    Sotatercept
    other Names
    1. ACE-011
    2. ActRIIA-IgG1Fc
    type
    DRUG
Study design
allocation
RANDOMIZED
intervention Model
PARALLEL
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
74
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    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.
    measure
    Percentage of Participants With Erythroid Hematological Improvement (HI-E) Starting Before the Completion of Five Cycles of Treatment (Responder Rate)
    time Frame
    Day 2 to Day 142
secondary Outcomes
  1. description
    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.
    measure
    Time to Erythroid Hematological Improvement (HI-E) Response
    time Frame
    Day 1 to Day 87
  2. description
    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.
    measure
    Duration of Erythroid Hematological Improvement (HI-E)
    time Frame
    Day 1 to 183.7 weeks
  3. description
    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.
    measure
    Time to Progression to Acute Myeloid Leukemia (AML) for Participants Who Had Progression
    time Frame
    Day 1 to 183.7 weeks
  4. description
    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.
    measure
    Time to Progression to Events of Higher Risk Myelodysplastic Syndromes (MDS) Using the International Prognostic Scoring System (IPSS) For Participants Who Had Progression
    time Frame
    Day 1 to 257.3 weeks
  5. description
    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
    measure
    Kaplan-Meier Estimates for Progression-free Survival
    time Frame
    Day 1 to 257.3 weeks
  6. description
    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.
    measure
    Kaplan-Meier Estimates for Overall Survival (OS)
    time Frame
    Day 1 to 257.3 weeks
  7. description
    Maximum observed serum concentration, obtained directly from the observed concentration versus time data.
    measure
    Pharmacokinetic Parameters of Sotatercept: Serum Concentration at Various Study Timepoints
    time Frame
    Cycle 1 Day 8 and !5 up to Cycle 2 Day 1
  8. description
    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.
    measure
    Participants With Treatment-Emergent Adverse Events (TEAE)
    time Frame
    Day 1 up to 59.2 months
  9. description
    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
    measure
    Dose Limiting Toxicities (DLTs)
    time Frame
    Day 1 to 59.2 months
  10. description
    Number of participants who achieved RBC-independence was defined as participants who required no RBC-transfusions during a 56-day interval of erythroid hematological improvement (HI-E). NTDE = non-transfusion dependence efficacy participants who required \< 4 units of RBCs in the 8 weeks prior to start of therapy TDE = transfusion dependence efficacy participants who required \>=4 units of RBCs in the 8 weeks prior to start of therapy
    measure
    Number of Participants Who Achieved Red Blood Cell (RBC)-Transfusion Independence During the Erythroid Hematological Improvement (HI-E) Interval
    time Frame
    Day 2 to Day 142
Full study description
brief Summary
The primary objective of this study is to determine a safe, tolerable and effective dose of sotatercept that results in the greatest frequency of improvement of anemia in patients diagnosed with low- or intermediate-1 risk myelodysplastic syndromes (MDS) or non-proliferative chronic myelomonocytic leukemia (CMML).
Source references
references
  1. citation
    Komrokji R, Garcia-Manero G, Ades L, Prebet T, Steensma DP, Jurcic JG, Sekeres MA, Berdeja J, Savona MR, Beyne-Rauzy O, Stamatoullas A, DeZern AE, Delaunay J, Borthakur G, Rifkin R, Boyd TE, Laadem A, Vo B, Zhang J, Puccio-Pick M, Attie KM, Fenaux P, List AF. Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2, dose-ranging trial. Lancet Haematol. 2018 Feb;5(2):e63-e72. doi: 10.1016/S2352-3026(18)30002-4. Epub 2018 Jan 10.
    pmid
    29331635
    type
    DERIVED
Source notices and limitations
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    Preserved source evidence · Not medical advice