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The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS)

This study is currently recruiting participants.
Verified October 2017 by Celgene
Sponsor:
ClinicalTrials.gov Identifier:
NCT01566695
First Posted: March 29, 2012
Last Update Posted: November 2, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Celgene
  Purpose
Evaluation of the Efficacy and Safety of Oral Azacitidine plus Best Supportive care versus Placebo and Best Supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).

Condition Intervention Phase
Myelodysplastic Syndrome Drug: Oral Azacitidine Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Randomized, Double-blind Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo Plus Best Supportive Care in Subjects With Red Blood Cell Transfusion-dependent Anemia and Thrombocytopenia Due to IPSS Lower-risk Myelodysplastic Syndromes.

Resource links provided by NLM:


Further study details as provided by Celgene:

Primary Outcome Measures:
  • Red blood cell (RBC) transfusion independence [ Time Frame: Up to 60 months ]
    Red blood cell (RBC) transfusion independence


Secondary Outcome Measures:
  • Number of patients alive [ Time Frame: Up to 60 months ]
    Number of patients alive

  • Hematological improvement-platelet response (HI-P) [ Time Frame: Up to 60 months ]
    Hematological improvement-platelet response (HI-P)

  • Duration of RBC transfusion independence [ Time Frame: Up to 60 months ]
    Duration of RBC transfusion independence

  • Time to RBC transfusion independence [ Time Frame: Up to 60 months ]
    Time to RBC transfusion independence

  • Progression to acute myeloid leukemia (AML) [ Time Frame: Up to 60 months ]
    Progression to acute myeloid leukemia (AML)

  • Time to AML progression [ Time Frame: Up to 60 months ]
    Time to AML progression

  • Hematological improvement-erythroid response (HI-E) [ Time Frame: Up to 60 months ]
    Hematological improvement-erythroid response (HI-E)

  • Platelet-transfusion independence [ Time Frame: Up to 60 months ]
    Platelet-transfusion independence

  • Duration of platelet transfusion independence [ Time Frame: Up to 60 months ]
    Duration of platelet transfusion independence

  • Time to platelet transfusion independence [ Time Frame: Up to 60 months ]
    Time to platelet transfusion independence

  • Hematologic response [ Time Frame: Up to 60 months ]
    Hematologic response

  • Clinically significant bleeding events [ Time Frame: Up to 60 months ]
    Clinically significant bleeding events

  • Number of subjects with adverse events [ Time Frame: Up to 60 months ]
    Number of subjects with adverse events

  • Health-related quality-of-life [ Time Frame: Up to 60 months ]
    Health-related quality-of-life

  • Healthcare resource utilization [ Time Frame: Up to 60 months ]
    Healthcare resource utilization


Estimated Enrollment: 294
Actual Study Start Date: April 26, 2013
Estimated Study Completion Date: August 31, 2021
Estimated Primary Completion Date: July 31, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Oral Azacitidine
Arm 1: Oral azacitidine 300mg daily + best supportive care (First 21 days of each 28-day cycle)
Drug: Oral Azacitidine
300mg daily, First 21 days of each 28-day cycle
Placebo Comparator: Placebo
Arm 2: Placebo plus best supportive care (First 21 days of each 28-day cycle)
Drug: Placebo
Placebo, First 21 days of each 28-day cycle

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 18 years or older
  • Have a documented diagnosis of MDS
  • Anemia that requires red blood cell transfusions
  • Thrombocytopenia (sustained for at least 21 days) within 14 days prior to randomization
  • Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
  • Must agree to follow pregnancy precautions as required by protocol.
  • Must be willing to consent to two or more bone marrow aspirate procedures to be completed during study.

Exclusion Criteria:

  • Secondary or hypoplastic MDS or other subtype with eligibility for treatment with immunotherapy
  • Prior treatment with azacitidine, decitabine, other hypomethylating agents and lenalidomide ( for lenalidomide : unless the last dose received is >= 8 weeks prior to inclusion into the study).
  • Prior allogeneic or autologous stem cell transplant
  • Eligible for allogenic or autologous stem cell transplant
  • History of inflammatory bowel disease (eg, Crohn's disease, ulcerative colitis), celiac disease (ie, sprue), prior gastrectomy or upper bowel removal, or any other gastrointestinal disorder or defect
  • Thrombocytopenia secondary to other possible causes, including medication(s), congenital disorder(s), immune disorder(s), or microvascular disorder(s)
  • Use of cytotoxic, chemotherapeutic, targeted or investigational agents/therapies, thrombopoiesis-stimulating agents (TSAs), erythropoiesis-stimulating agents (ESAs) and other red blood cell hematopoietic growth factors, and within 28 days prior to randomization
  • Ongoing medically significant adverse events from previous treatment, regardless of the time period
  • Concurrent use of iron-chelating agents, (except for subjects on a stable or decreasing dose for at least 8 weeks (56 days) prior to randomization), corticosteroid (except for subjects on a stable or decreasing dose for ≥ 1 week prior to randomization for medical conditions other than MDS)
  • Prior history of cancer, other than MDS, unless the subject has been free of the disease for ≥ 3 years. (Basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, and incidental histologic finding of prostate cancer) (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system is allowed)
  • Significant active cardiac disease within the previous 6 months
  • Uncontrolled systemic fungal, bacterial, or viral infection
  • Known Human Immunodeficiency Virus (HIV) or Hepatitis C (HCV) infection, or evidence of active Hepatitis B Virus (HBV) infection
  • Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
  • Abnormal coagulation parameters
  • Abnormal liver function test results
  • Abnormal kidney function test results
  • Known or suspected hypersensitivity to azacitidine or mannitol
  • Any significant medical condition, laboratory abnormality, or psychiatric illness
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01566695


Contacts
Contact: Associate Director, Clinical Trial Disclosure 1-888-260-1599 ClinicalTrialDisclosure@celgene.com

  Show 196 Study Locations
Sponsors and Collaborators
Celgene
Investigators
Study Director: Torsten Gerike, MD Celgene Corporation
  More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT01566695     History of Changes
Other Study ID Numbers: AZA-MDS-003
2012-002471-34 ( EudraCT Number )
First Submitted: March 27, 2012
First Posted: March 29, 2012
Last Update Posted: November 2, 2017
Last Verified: October 2017

Additional relevant MeSH terms:
Syndrome
Myelodysplastic Syndromes
Preleukemia
Thrombocytopenia
Anemia
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Blood Platelet Disorders
Azacitidine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors