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

This study has been completed.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004474
First received: October 18, 1999
Last updated: October 1, 2008
Last verified: October 2008
History of Changes
  Purpose

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.


Condition Intervention Phase
Aplastic Anemia
 Drug: Anti-thymocyte globulin
Drug: Cyclophosphamide
Procedure: Bone marrow transplantation
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Primary Purpose: Treatment
Official Title: Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Primary Outcome Measures:
  • Graft failure, graft versus host disease, and survival [ Time Frame: Measured at Day 100, Month 6, and Year 1 post-transplant ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 224
Study Start Date: September 1998
Study Completion Date: August 2007
Primary Completion Date: August 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   up to 59 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004474

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

No publications provided

Responsible Party: Richard Champlin, University of Texas - MD Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00004474     History of Changes
Other Study ID Numbers: 199/14004, RPCI-RP-9804, NCI-G98-1491, IBMTR-1
Study First Received: October 18, 1999
Last Updated: October 1, 2008
Health Authority: United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
Hematologic Disorders
Rare Disease

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Antilymphocyte Serum
Cyclophosphamide
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on May 19, 2013