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

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 III

MedlinePlus related topics:

Anemia Bone Marrow Transplantation

Drug Information available for:

Cyclophosphamide

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Parallel Assignment, Safety/Efficacy Study

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

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
1: Experimental 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
2: Experimental 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

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

Keywords provided by Office of Rare Diseases (ORD):

Hematologic Disorders

Rare Disease

Study placed in the following topic categories:

Antilymphocyte Serum

Hematologic Diseases

Rare Diseases

Anemia, Aplastic

Anemia

Cyclophosphamide

Aplastic anemia

Bone Marrow Diseases

Additional relevant MeSH terms:

Molecular Mechanisms of Pharmacological Action

Immunologic Factors

Antineoplastic Agents

Therapeutic Uses

Physiological Effects of Drugs

Myeloablative Agonists

Antineoplastic Agents, Alkylating

Antirheumatic Agents

Alkylating Agents

Immunosuppressive Agents

Pharmacologic Actions

ClinicalTrials.gov processed this record on November 20, 2008

Sponsored by:	Roswell Park Cancer Institute
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004474