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 ID: NCT01736683

Last Updated: 2022-10-24

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

74 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-28

Study Completion Date

2018-04-30

Brief Summary

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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).

Detailed Description

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Conditions

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Anemia Myelodysplastic Syndromes Chronic Myelomonocytic Leukemia Low to Intermediate-1 MDS Myelodysplastic Syndromes (MDS) Chronic Myelomonocytic Leukemia (CMML)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Sotatercept 0.1 mg/kg

Sotatercept 0.1 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Sotatercept 0.3 mg/kg

Sotatercept 0.3 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Sotatercept 0.5 mg/kg

Sotatercept 0.5 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Sotatercept 1.0 mg/kg

Sotatercept 1.0 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Sotatercept 1.5 mg/kg

Sotatercept 1.5 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Sotatercept 2.0 mg/kg

Sotatercept 2.0 mg/kg

Group Type EXPERIMENTAL

Sotatercept

Intervention Type DRUG

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.

Interventions

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Sotatercept

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.

Intervention Type DRUG

Other Intervention Names

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ACE-011 ActRIIA-IgG1Fc

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Merck Sharp & Dohme LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Rodrigo Ito, MD

Role: STUDY_DIRECTOR

Celgene Corporation

Locations

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Rocky Mountain Cancer Center-Midtown

Denver, Colorado, United States

Site Status

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, United States

Site Status

Johns Hopkins

Baltimore, Maryland, United States

Site Status

Dana-Farber / Harvard Cancer Institute

Boston, Massachusetts, United States

Site Status

Monter Cancer Center, North Shore LIJ Health Systems

Lake Success, New York, United States

Site Status

Columbia University Medical Center/New York-Presbyterian Hospital

New York, New York, United States

Site Status

The Cleveland Clinic Foundation Hematology and Medical Oncology Rm 35

Cleveland, Ohio, United States

Site Status

Sarah Cannon Research Inst

Nashville, Tennessee, United States

Site Status

Texas Oncology Round Rock Cancer Center - Round Rock

Round Rock, Texas, United States

Site Status

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status

Texas Oncology, P.A. - Tyler

Tyler, Texas, United States

Site Status

Virginia Oncology Associates

Norfolk, Virginia, United States

Site Status

Yakima Valley Memorial Hospital/ North Star Lodge

Yakima, Washington, United States

Site Status

Centre Hospitalier Universitaire d'Avicennes

Bobigny, , France

Site Status

Institute Paoli-Calmettes Service Haematology

BP 156,, , France

Site Status

CHRU de Lille-Hopital Claude Huriez Service des Maladies du Sang

Lille, , France

Site Status

CHRU Nantes

Nantes, , France

Site Status

Hopital Cochin Hematologie

Paris, , France

Site Status

Centre Henri Becquerel

Rouen, , France

Site Status

CHU Purpan

Toulouse, , France

Site Status

Countries

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United States France

References

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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.

Reference Type DERIVED
PMID: 29331635 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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ACE-011-MDS-001

Identifier Type: OTHER

Identifier Source: secondary_id

2012-002601-22

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

7962-017

Identifier Type: -

Identifier Source: org_study_id

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