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

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ClinicalTrials.gov Identifier: NCT01736683
Recruitment Status : Active, not recruiting
First Posted : November 29, 2012
Last Update Posted : April 26, 2018
Sponsor:
Information provided by (Responsible Party):
Celgene

November 27, 2012
November 29, 2012
April 26, 2018
November 28, 2012
April 30, 2018   (Final data collection date for primary outcome measure)
Erythroid Hematological Improvement (HI-E) [ Time Frame: Up to 24 weeks ]
HI-E (for subjects that require a transfusion of <4 units of RBCs) is an increase ≥1.5 g/dL Hgb sustained over a period ≥8 weeks in the absence of RBC transfusion; or HI-E (for subjects that require a transfusion of ≥4 units of RBCs) is a decrease ≥4 units of RBCs transfused over a period of 8 weeks
Same as current
Complete list of historical versions of study NCT01736683 on ClinicalTrials.gov Archive Site
  • Adverse Event [ Time Frame: Up to 3 years ]
    Number of participants with adverse events
  • Red Blood Cell (RBC) Transfusion Independence [ Time Frame: Up to 24 weeks ]
    The time between randomization (for Part 1)/start of therapy (for Part 2) and the date the start of HI-E
  • Duration to Erythroid Hematological Improvement (HI-E) [ Time Frame: Up to 24 weeks ]
    The length of time between first and last assessment of HI-E
  • Time to progression to Acute Myeloid Leukemia (AML) [ Time Frame: Up to 2 years ]
    Time from baseline until progression to AML
  • Time to progression to events of higher risk MDS [ Time Frame: Up to 2 years ]
    Time from baseline until progression to events of higher risk MDS
  • Progression-free survival (PFS) [ Time Frame: Up to 2 years ]
    Number of participants who survive without progressing.
  • Overall survival (OS) [ Time Frame: Up to 2 years ]
    Number of participants who survive
  • Pharmacokinetics-Cmax [ Time Frame: Up to 24 weeks ]
    Maximum observed concentration in serum
  • Pharmacokinetics-Tmax [ Time Frame: Up to 24 weeks ]
    Time to maximum observed concentration serum
  • Pharmacokinetics- AUC [ Time Frame: Up to 24 weeks ]
    Area under the plasma concentration-time curve
Same as current
Not Provided
Not Provided
 
Study of Sotatercept for the Treatment of Anemia in low-or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) or Non-proliferative Chronic Myelomonocytic Leukemia (CMML)
An Open-label, Randomized, Phase 2, Parallel, Dose-ranging, Multicenter Study of Sotatercept for the Treatment of Patients With Anemia and Low- or Intermediate-1 Risk Myelodysplastic Syndromes or Non-proliferative Chronic Myelomonocytic Leukemia (CMML).
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 MDS or non-proliferative chronic myelomonocytic leukemia (CMML).
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Anemia
  • Myelodysplastic Syndromes
  • Chronic Myelomonocytic Leukemia
  • Low to Intermediate-1 MDS
  • Myelodysplastic Syndromes (MDS)
  • Chronic Myelomonocytic Leukemia (CMML)
  • Drug: Sotatercept
    Sotatercept 0.1 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
  • Drug: Sotatercept
    Sotatercept 0.3 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
    Other Name: ActRIIA-IgG1Fc
  • Drug: Sotatercept
    Sotatercept 0.5 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
  • Drug: Sotatercept
    Sotatercept 1.0 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
  • Drug: Sotatercept
    Sotatercept 1.5 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
    Other Name: ActRIIA-IgG1Fc
  • Drug: Sotatercept
    Sotatercept 2.0 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
  • Drug: Sotatercept 0.1 mg/kg
    Sotatercept 0.1 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
    Other Name: ActRIIA-IgG1Fc
  • Drug: Sotatercept 0.3 mg/kg
    Sotatercept 0.3 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
    Other Name: ActRIIA-IgG1Fc
  • Drug: Sotatercept 0.5 mg/kg
    Sotatercept 0.5 mg/kg subcutaneous (SC) once every 3 weeks (q3W) for 5 cycles
    Other Name: ActRIIA-IgG1c
  • Experimental: Sotatercept 0.1 mg/kg
    Sotatercept 0.1 mg/kg
    Interventions:
    • Drug: Sotatercept
    • Drug: Sotatercept 0.1 mg/kg
  • Experimental: Sotatercept 0.3 mg/kg
    Sotatercept 0.3 mg/kg
    Interventions:
    • Drug: Sotatercept
    • Drug: Sotatercept 0.3 mg/kg
  • Experimental: Sotatercept 0.5 mg/kg
    Sotatercept 0.5 mg/kg
    Interventions:
    • Drug: Sotatercept
    • Drug: Sotatercept 0.5 mg/kg
  • Experimental: Sotatercept 1.0 mg/kg
    Sotatercept 1.0 mg/kg
    Intervention: Drug: Sotatercept
  • Experimental: Sotatercept 1.5 mg/kg
    Sotatercept 1.5 mg/kg
    Intervention: Drug: Sotatercept
  • Experimental: Sotatercept 2.0 mg/kg
    Sotatercept 2.0 mg/kg
    Intervention: Drug: Sotatercept
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
100
Same as current
April 30, 2018
April 30, 2018   (Final data collection date for primary outcome measure)

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 /mm3, 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 X 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 x Upper Limit of Norma (ULN)
  • Free of metastatic malignancy (other than MDS) for ≥2 years
  • Highly effective methods of birth control for females & 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
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
France,   United States
 
 
NCT01736683
ACE-011-MDS-001
No
Not Provided
Not Provided
Celgene
Celgene
Not Provided
Study Director: Abderrahmane Laadem Celgene Corporation
Celgene
April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP