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

Primary Purpose

Aplastic Anemia

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Anti-thymocyte globulin
Cyclophosphamide
Bone marrow transplantation
Sponsored by
Roswell Park Cancer Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring Hematologic Disorders, Rare Disease

Eligibility Criteria

undefined - 59 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Roswell Park Cancer Institute
  • University of Texas - MD Anderson Cancer Center
  • Medical College of Wisconsin
  • Midwest Children's Cancer Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

1

2

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Graft failure, graft versus host disease, and survival

Secondary Outcome Measures

Full Information

First Posted
October 18, 1999
Last Updated
March 9, 2018
Sponsor
Roswell Park Cancer Institute
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1. Study Identification

Unique Protocol Identification Number
NCT00004474
Brief Title
Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
Official Title
Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
October 2008
Overall Recruitment Status
Completed
Study Start Date
September 1998 (undefined)
Primary Completion Date
August 2007 (Actual)
Study Completion Date
August 2007 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Roswell Park Cancer Institute

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia
Keywords
Hematologic Disorders, Rare Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Allocation
Randomized
Enrollment
224 (false)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Description
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.
Arm Title
2
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Anti-thymocyte globulin
Intervention Description
Given via IV over 4 hours
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Intervention Description
Given via IV over 60 minutes on Days -5 to -2
Intervention Type
Procedure
Intervention Name(s)
Bone marrow transplantation
Intervention Description
Given on Day 0
Primary Outcome Measure Information:
Title
Graft failure, graft versus host disease, and survival
Time Frame
Measured at Day 100, Month 6, and Year 1 post-transplant

10. Eligibility

Sex
All
Maximum Age & Unit of Time
59 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Roswell Park Cancer Institute
City
Buffalo
State/Province
New York
ZIP/Postal Code
14263-0001
Country
United States
Facility Name
University of Texas - MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030-4009
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Midwest Children's Cancer Center
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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