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

March 9, 2018 updated by: Roswell Park Cancer Institute

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment

224

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New York
      • Buffalo, New York, United States, 14263-0001
        • Roswell Park Cancer Institute
    • Texas
      • Houston, Texas, United States, 77030-4009
        • University of Texas - MD Anderson Cancer Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Midwest Children's Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 59 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 1
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
Given via IV over 4 hours
Drug: Cyclophosphamide
Given via IV over 60 minutes on Days -5 to -2
Procedure: Bone marrow transplantation
Given on Day 0
Experimental: 2
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
Given via IV over 60 minutes on Days -5 to -2
Procedure: Bone marrow transplantation
Given on Day 0

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Roswell Park Cancer Institute

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 1998

Primary Completion (Actual)

August 1, 2007

Study Completion (Actual)

August 1, 2007

Study Registration Dates

First Submitted

October 18, 1999

First Submitted That Met QC Criteria

October 18, 1999

First Posted (Estimate)

October 19, 1999

Study Record Updates

Last Update Posted (Actual)

March 12, 2018

Last Update Submitted That Met QC Criteria

March 9, 2018

Last Verified

October 1, 2008

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 199/14004
  • RPCI-RP-9804
  • NCI-G98-1491
  • IBMTR-1

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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