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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. (see Contacts and Locations)
Verified July 2014 by Celgene Corporation
Sponsor:
Information provided by (Responsible Party):
Celgene Corporation
ClinicalTrials.gov Identifier:
NCT01566695
First received: March 27, 2012
Last updated: July 8, 2014
Last verified: July 2014

March 27, 2012
July 8, 2014
May 2013
June 2019   (final data collection date for primary outcome measure)
Red blood cell (RBC) transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
Red blood cell (RBC) transfusion independence
Same as current
Complete list of historical versions of study NCT01566695 on ClinicalTrials.gov Archive Site
  • Number of patients alive [ Time Frame: Up to 60 months ] [ Designated as safety issue: Yes ]
    Number of patients alive
  • Hematological improvement-platelet response (HI-P) [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Hematological improvement-platelet response (HI-P)
  • Duration of RBC transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Duration of RBC transfusion independence
  • Time to RBC transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Time to RBC transfusion independence
  • Progression to acute myeloid leukemia (AML) [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Progression to acute myeloid leukemia (AML)
  • Time to AML progression [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Time to AML progression
  • Hematological improvement-erythroid response (HI-E) [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Hematological improvement-erythroid response (HI-E)
  • Platelet-transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Platelet-transfusion independence
  • Duration of platelet transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Duration of platelet transfusion independence
  • Time to platelet transfusion independence [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Time to platelet transfusion independence
  • Hematologic response [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Hematologic response
  • Clinically significant bleeding events [ Time Frame: Up to 60 months ] [ Designated as safety issue: Yes ]
    Clinically significant bleeding events
  • Number of subjects with adverse events [ Time Frame: Up to 60 months ] [ Designated as safety issue: Yes ]
    Number of subjects with adverse events
  • Health-related quality-of-life [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Health-related quality-of-life
  • Healthcare resource utilization [ Time Frame: Up to 60 months ] [ Designated as safety issue: No ]
    Healthcare resource utilization
Same as current
Not Provided
Not Provided
 
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)
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.

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).

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Myelodysplastic Syndrome
  • Drug: Oral Azacitidine
    300mg daily, First 21 days of each 28-day cycle
  • Drug: Placebo
    Placebo, First 21 days of each 28-day cycle
  • Experimental: Oral Azacitidine
    Arm 1: Oral azacitidine 300mg daily + best supportive care (First 21 days of each 28-day cycle)
    Intervention: Drug: Oral Azacitidine
  • Placebo Comparator: Placebo
    Arm 2: Placebo plus best supportive care (First 21 days of each 28-day cycle)
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
386
December 2019
June 2019   (final data collection date for primary outcome measure)

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
  • 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
Both
18 Years and older
No
Contact: Associate Director, Clinical Trial Disclosure 1-888-260-1599 ClinicalTrialDisclosure@celgene.com
United States,   Australia,   Belgium,   Brazil,   Canada,   Czech Republic,   Denmark,   Finland,   France,   Germany,   Israel,   Italy,   Mexico,   Netherlands,   Norway,   Poland,   Portugal,   Spain,   Sweden,   United Kingdom
 
NCT01566695
AZA-MDS-003, 2012-002471-34
No
Celgene Corporation
Celgene Corporation
Not Provided
Study Director: Barry Skikne, MD Celgene Corporation
Celgene Corporation
July 2014

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