Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

Brief Title

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

Official Title

Protocol for Prospective Phase II Study of Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) as a First Line Immunosuppressive (IS) Therapy for Severe Aplastic Anemia (sAA)

Brief Summary

      RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may
      improve bone marrow function and increase blood cell counts. PURPOSE: This phase II trial is
      studying how well giving anti-thymocyte globulin together with cyclosporine as first-line
      therapy works in treating patients with severe aplastic anemia.
    

Detailed Description

      PRIMARY OBJECTIVES: To determine the response rate of r-ATG and CsA in the first line
      setting. SECONDARY OBJECTIVES: To determine the level of IS as assessed by Immuknow assay in
      responders and compare it to non-responders. OUTLINE:Patients receive anti-thymocyte globulin
      IV over 4-24 hours daily on days 1-5. Beginning on day 6, patients receive oral cyclosporine
      twice daily for 6 months followed by a taper. Treatment continues in the absence of disease
      progression or unacceptable toxicity.
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Patients Treated With Rabbit Antithymocyte Globulin (r-ATG/Thymoglobulin) and Cyclosporine (CsA) Achieving at Least a Partial Remission (PR) at 6 Months

Secondary Outcome

 Comparison of the Level of IS as Assessed by Immuknow Assay in Responders and Non-responders

Condition

Aplastic Anemia

Intervention

cyclosporine

Study Arms / Comparison Groups

 rATG + Cyclosporine

Description:  Patients receive anti-thymocyte globulin IV daily over 4-24 hours on days 1-5. Beginning on day 6, patients receive oral cyclosporine twice daily for 6 months followed by a taper. Treatment continues in the absence of disease progression or unacceptable toxicity

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

20

Start Date

March 2005

Completion Date

December 2010

Primary Completion Date

December 2010

Eligibility Criteria

        Inclusion Criteria:

          -  All patients with sAA as defined by Camitta who are candidates for IS therapy; these
             criteria include bone marrow cellularity < 25% or 25-50% with < 30% of hematopoietic
             cells; it should also have two of the following three parameters: peripheral blood
             neutrophils < 0.5 x 10^9/L, platelets < 20 x 10^9/L and reticulocytes < 60 x 10^9/L in
             anemic patients

          -  If cytogenetic testing has been done, it should show normal karyotype or be not
             informative

          -  Patients should be either unwilling or otherwise ineligible (age, comorbidities, lack
             of donor) for bone marrow transplantation as a therapeutic modality

          -  Not previously treated with ATG for sAA

          -  Patients must have ECOG performance status of 0, 1, or 2

          -  Vitamin B12 and folic acid deficiency must be ruled out by measurement of serum levels

          -  Patients must have had a bone marrow biopsy examination in the three months prior to
             enrolling in the study

          -  Must be able to provide informed consent

          -  Systemic and other hematologic causes of pancytopenia, based on clinical presentation,
             must have been ruled out

        Exclusion Criteria:

          -  Patients with clinically evident congestive heart failure, serious cardiac
             arrhythmias; symptoms of coronary artery disease must be cleared by cardiology prior
             to therapy

          -  Patients who have had chemotherapy, radiotherapy, or immunotherapy or other
             investigational drug use within 3 weeks prior to study entry

          -  Pregnant women

          -  All females of childbearing potential must have a blood test or urine study within two
             weeks prior to induction registration to rule out pregnancy

          -  Women of childbearing potential are strongly advised to use an accepted and effective
             method of contraception

          -  Patients who have medical, psychological, or social conditions that may interfere with
             the patient's participation in the study or evaluation of the study results
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Jaroslaw Maciejewski, , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT01231841

Organization ID

CCF7922

Secondary IDs

NCI-2010-01365

Responsible Party

Sponsor

Study Sponsor

The Cleveland Clinic

Study Sponsor

Jaroslaw Maciejewski, Principal Investigator, Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

Verification Date

January 2018