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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


Phase 3
18 Years
N/A
Open (Enrolling)
Both
Myelodysplastic Syndrome

Thank you

Trial Information

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


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 adverse events from previous treatment, regardless of the time period

- Concurrent use of iron-chelating agents, (except for subjects on a stable 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

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Red blood cell (RBC) transfusion independence

Outcome Description:

Red blood cell (RBC) transfusion independence

Outcome Time Frame:

Up to 60 months

Safety Issue:

No

Principal Investigator

Barry Skikne, MD

Investigator Role:

Study Director

Investigator Affiliation:

Celgene Corporation

Authority:

United States: Food and Drug Administration

Study ID:

AZA-MDS-003

NCT ID:

NCT01566695

Start Date:

December 2012

Completion Date:

December 2016

Related Keywords:

  • Myelodysplastic Syndrome
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thrombocytopenia

Name

Location

Mayo ClinicRochester, Minnesota  55905
University of Nebraska Medical CenterOmaha, Nebraska  68198-3330
Jackson Oncology Associates, PLLCJackson, Mississippi  39202
The University of Texas, MD Anderson Cancer CenterHouston, Texas  77030
Kaiser Permanente NorthwestPortland, Oregon  97227
University of Kansas Cancer CenterKansas City, Kansas  66160
Saint Luke's Cancer InstituteKansas City, Missouri  64111
Marin Specialty CareGreenbrae, California  94904
California Cancer Associates for Research & Excellence (cCARE)Fresno, California  93720
East Jefferson General Hospital C/O Cancer Care of LouisianaMetairie, Louisiana  70006
Weill Cornell Medical College - New York-Presbyterian HospitalNew York, New York  10021
University Hospitals, Case Medical CenterCleveland, Ohio  44106
Western Pennsylvania Cancer Institute, c/o Western Pennsylvania HospitalPittsburgh, Pennsylvania  15224