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 Information
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
Administrative Informations
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