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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)

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ClinicalTrials.gov Identifier: NCT01566695
Recruitment Status : Active, not recruiting
First Posted : March 29, 2012
Last Update Posted : May 24, 2018
Sponsor:
Information provided by (Responsible Party):
Celgene

March 27, 2012
March 29, 2012
May 24, 2018
April 26, 2013
July 31, 2021   (Final data collection date for primary outcome measure)
Red blood cell (RBC) transfusion independence [ Time Frame: Up to 122 months ]
Red blood cell (RBC) transfusion independence
Red blood cell (RBC) transfusion independence [ Time Frame: Up to 60 months ]
Red blood cell (RBC) transfusion independence
Complete list of historical versions of study NCT01566695 on ClinicalTrials.gov Archive Site
  • Number of patients alive [ Time Frame: Up to 122 months ]
    Number of patients alive
  • Hematological improvement-platelet response (HI-P) [ Time Frame: Up to 122 months ]
    Hematological improvement-platelet response (HI-P)
  • Duration of RBC transfusion independence [ Time Frame: Up to 122 months ]
    Duration of RBC transfusion independence
  • Time to RBC transfusion independence [ Time Frame: Up to 122 months ]
    Time to RBC transfusion independence
  • Progression to acute myeloid leukemia (AML) [ Time Frame: Up to 122 months ]
    Progression to acute myeloid leukemia (AML)
  • Time to AML progression [ Time Frame: Up to 122 months ]
    Time to AML progression
  • Hematological improvement-erythroid response (HI-E) [ Time Frame: Up to 122 months ]
    Hematological improvement-erythroid response (HI-E)
  • Platelet-transfusion independence [ Time Frame: Up to 122 months ]
    Platelet-transfusion independence
  • Duration of platelet transfusion independence [ Time Frame: Up to 122 months ]
    Duration of platelet transfusion independence
  • Time to platelet transfusion independence [ Time Frame: Up to 122 months ]
    Time to platelet transfusion independence
  • Hematologic response [ Time Frame: Up to 122 months ]
    Hematologic response
  • Clinically significant bleeding events [ Time Frame: Up to 122 months ]
    Clinically significant bleeding events
  • Number of subjects with adverse events [ Time Frame: Up to 122 months ]
    Type, frequency, severity of AEs and relationship of AEs to oral azacytidine/placebo; monitoring for progression to AML and secondary primary malignancy
  • Health-related quality-of-life [ Time Frame: Up to 122 months ]
    Health-related quality-of-life
  • Healthcare resource utilization [ Time Frame: Up to 122 months ]
    Healthcare resource utilization
  • 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
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
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
Garcia-Manero G, Almeida A, Giagounidis A, Platzbecker U, Garcia R, Voso MT, Larsen SR, Valcarcel D, Silverman LR, Skikne B, Santini V. Design and rationale of the QUAZAR Lower-Risk MDS (AZA-MDS-003) trial: a randomized phase 3 study of CC-486 (oral azacitidine) plus best supportive care vs placebo plus best supportive care in patients with IPSS lower-risk myelodysplastic syndromes and poor prognosis due to red blood cell transfusion-dependent anemia and thrombocytopenia. BMC Hematol. 2016 May 3;16:12. doi: 10.1186/s12878-016-0049-5. eCollection 2016.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
217
386
August 31, 2021
July 31, 2021   (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 ( 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
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Belgium,   Brazil,   Canada,   Czechia,   Denmark,   Finland,   France,   Germany,   Greece,   Israel,   Italy,   Korea, Republic of,   Mexico,   Netherlands,   Norway,   Poland,   Portugal,   Spain,   Sweden,   Turkey,   United Kingdom,   United States
Czech Republic
 
NCT01566695
AZA-MDS-003
2012-002471-34 ( EudraCT Number )
No
Not Provided
Not Provided
Celgene
Celgene
Not Provided
Study Director: Ignazia Ignazia La Torre, MD Celgene Corporation
Celgene
May 2018

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