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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 is ongoing, but not recruiting participants.

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

ChemIDplus related topics:   Cyclophosphamide   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized

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

Estimated Enrollment:   224
Study Start Date:   September 1998

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

Investigators
Study Chair:     Philip L. McCarthy, Jr.     Roswell Park Cancer Institute    
  More Information

Study ID Numbers:   199/14004, RPCI-RP-9804, NCI-G98-1491, IBMTR-1
First Received:   October 18, 1999
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00004474
Health Authority:   Unspecified

Keywords provided by Office of Rare Diseases (ORD):
aplastic anemia  
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 September 05, 2008




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