Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia

Study Overview
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Brief Summary
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OBJECTIVES: I. Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin as a conditioning regimen.

Detailed Description
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PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to receive cyclophosphamide IV over 60 minutes on days -5 to -2 with or without antithymocyte globulin IV over 4 hours. All patients then receive bone marrow over 60-120 minutes on day 0, 36 hours after the last dose of cyclophosphamide. Patients are followed at day 100, at 6 months, and at 1 year posttransplant.

Official Title
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Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia

Conditions
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Aplastic Anemia

Intervention / Treatment
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* Drug: Anti-thymocyte globulin
* Drug: Cyclophosphamide
* Procedure: Bone marrow transplantation


Participation Criteria
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Eligibility Criteria
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PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Severe aplastic anemia with the following criteria: Hypocellular bone marrow with cellularity less than 20% At least 2 of the following hematologic abnormalities: Neutrophil count no greater than 500/mm3 Platelet count no greater than 20,000/mm3 Reticulocyte count no greater than 50,000/mm3 HLA-identical sibling donor available No clonal cytogenetic abnormalities, paroxysmal nocturnal hemoglobinuria, or myelodysplastic syndrome within 3 months of diagnosis of aplastic anemia No congenital or constitutional aplastic anemia or Fanconi anemia --Patient Characteristics-- Hepatic: Bilirubin less than 3 times upper limit of normal (ULN) Renal: Creatinine less than 2 times ULN Cardiovascular: Normal cardiac function Other: No uncontrolled infection No severe concurrent disease HIV negative Fertile patients must use effective contraception

Ages Eligible for Study
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Maximum Age: 59 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2 with antithymocyte globulin IV over 4 hours; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. | Drug: Anti-thymocyte globulin<br>* Given via IV over 4 hours<br>Drug: Cyclophosphamide<br>* Given via IV over 60 minutes on Days -5 to -2<br>Procedure: Bone marrow transplantation<br>* Given on Day 0<br>|
| Experimental: 2<br>Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0. | Drug: Cyclophosphamide<br>* Given via IV over 60 minutes on Days -5 to -2<br>Procedure: Bone marrow transplantation<br>* Given on Day 0<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Graft failure, graft versus host disease, and survival |  | Measured at Day 100, Month 6, and Year 1 post-transplant | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hematologic Disorders, Rare Disease