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

Information source: The Cleveland Clinic
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Aplastic Anemia

Intervention: cyclosporine (Drug); flow cytometry (Other); anti-thymocyte globulin (Biological)

Phase: Phase 2

Status: Completed

Sponsored by: The Cleveland Clinic

Official(s) and/or principal investigator(s):
Jaroslaw Maciejewski, Principal Investigator, Affiliation: Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center


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.

Clinical Details

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)

Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

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

Reduction of VB Repertoire Associated With r-ATG/CsA Combination

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.


Minimum age: 12 Years. Maximum age: N/A. Gender(s): Both.


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


- 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


- 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

Locations and Contacts

Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center, Cleveland, Ohio 44195, United States
Additional Information

Starting date: March 2005
Last updated: March 25, 2013

Page last updated: August 23, 2015

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