Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia

This study has been completed.
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: June 2, 2000
Last updated: June 23, 2005
Last verified: July 2004


I. Determine the effectiveness of moderate dose cyclophosphamide and total lymphoid radiotherapy in terms of improving the survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with Fanconi's aplastic anemia.

Condition Intervention
Fanconi's Anemia
Drug: cyclophosphamide
Procedure: Allogeneic Bone Marrow Transplantation

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

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

Study Start Date: March 2000
Detailed Description:

PROTOCOL OUTLINE: Patients receive cyclophosphamide IV over 2 hours on day -6 through -3 and total lymphoid radiotherapy on day -1. Patients undergo allogeneic bone marrow transplantation on day 0.

Patients are followed for at least 100 days.


Ages Eligible for Study:   up to 54 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • Diagnosis of severe aplastic anemia with the typical phenotype of Fanconi's anemia: Short stature Hypoplastic radii Skin pigmentation Renal anomalies Chromosomal fragility
  • Family history of Fanconi's anemia
  • Histocompatible related donor No evidence of excessive in vitro chromosome fragility typical of Fanconi's anemia Normal CBC and bone marrow
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Please refer to this study by its identifier: NCT00005891

United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
Study Chair: Daniel J. Weisdorf Fairview University Medical Center
  More Information Identifier: NCT00005891     History of Changes
Other Study ID Numbers: 199/15099  UMN-MT-1982-10  UMN-MT-8210 
Study First Received: June 2, 2000
Last Updated: June 23, 2005
Health Authority: Unspecified

Keywords provided by Office of Rare Diseases (ORD):
Fanconi's Anemia
aplastic anemia
hematologic disorders
rare disease

Additional relevant MeSH terms:
Fanconi Anemia
Fanconi Syndrome
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Kidney Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Renal Tubular Transport, Inborn Errors
Urologic Diseases
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Physiological Effects of Drugs processed this record on May 26, 2016