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

Phase 3

Completed

• Conditions: Aplastic Anemia
• Interventions: Drug: Anti-thymocyte globulin; Drug: Cyclophosphamide; Procedure: Bone marrow transplantation

• Registration Number: NCT00004474
• Lead Sponsor: Roswell Park Cancer Institute

Brief Summary

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

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.

Study Timeline

• Study Start: 1998-09
• Study First Submitted: 1999-10-18
• Study First Submitted QC: 1999-10-18
• Study First Posted: 1999-10-19
• Primary Completion: 2007-08
• Study Completion: 2007-08
• Status Verified: 2008-10
• Last Update Submitted: 2018-03-09
• Last Update Posted: 2018-03-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 224

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	Anti-thymocyte globulin	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.
1	Bone marrow transplantation	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.
2	Bone marrow transplantation	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.
1	Cyclophosphamide	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.
2	Cyclophosphamide	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.

Primary Outcome Measures

Name	Time	Method
Graft failure, graft versus host disease, and survival	Measured at Day 100, Month 6, and Year 1 post-transplant

Trial Locations

Locations (4)

Midwest Children's Cancer Center; 🇺🇸 Milwaukee, Wisconsin, United States

Roswell Park Cancer Institute; 🇺🇸 Buffalo, New York, United States

University of Texas - MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Medical College of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States