Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia

NCT00005898 | Completed Phase 1

• 3 other identifiers: 199/15109UMN-MT-1999-05UMN-MT9905
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell transplantation in patients with Fanconi's anemia. II. Determine the incidence of acute and chronic graft-versus-host disease in these patients after undergoing this treatment regimen. III. Determine the one-year survival rate in these patients after undergoing this treatment regimen. IV. Determine the toxicity of this treatment regimen in these patients. V. Determine the incidence of relapse in patients with myelodysplastic syndrome or acute myeloid leukemia after undergoing this treatment regimen.

Conditions

Fanconi's Anemia

Interventions

anti-thymocyte globulin DRUG

cyclophosphamide DRUG

cyclosporine DRUG

filgrastim DRUG

fludarabine DRUG

methylprednisolone DRUG

Eligibility Criteria

• Age: 0 Years - 34 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Patients with a diagnosis of Fanconi's anemia AND Aplastic anemia OR Myelodysplastic syndrome OR Acute leukemia with or without chromosomal anomalies Aplastic anemia as defined by having at least one of the following: Platelet count less than 20,000/mm3 Absolute neutrophil count less than 500/mm3 Hemoglobin less than 8 g/dL * Myelodysplastic syndrome with multilineage dysplasia With or without chromosomal anomalies * Hematologic malignancy (e.g., acute myeloid leukemia, acute lymphoblastic leukemia, or B-cell non-Hodgkin's lymphoma) * No greater than 30% blasts in bone marrow or greater than 5% blasts in peripheral blood * No active CNS leukemia at time of transplantation * Must have an HLA A, B, DRB1 identical or 1 antigen mismatched related (nonsibling) or unrelated bone marrow, peripheral blood, or umbilical cord blood donor --Prior/Concurrent Therapy-- * Radiotherapy: No prior radiotherapy that would preclude total body irradiation * Surgery: Not specified --Patient Characteristics-- * Performance status: Karnofsky 70-100% OR Lansky 50-100% * Hematopoietic: See Disease Characteristics * Hepatic: No hepatic failure (e.g., coagulopathy or ascites) * Renal: Creatinine clearance at least 40 mL/min * Cardiovascular: Ejection fraction at least 45% * Other: No active uncontrolled infection within one week of transplantation No malignant solid tumor (e.g., squamous cell carcinoma of the head, neck, or cervix) within 2 years of transplantation Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• University of Minnesota: lead

Study Sites (1)

University of Minnesota Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

Related Publications (1)

• MacMillan ML, DeFor TE, Young JA, Dusenbery KE, Blazar BR, Slungaard A, Zierhut H, Weisdorf DJ, Wagner JE. Alternative donor hematopoietic cell transplantation for Fanconi anemia. Blood. 2015 Jun 11;125(24):3798-804. doi: 10.1182/blood-2015-02-626002. Epub 2015 Mar 30.

  PMID: 25824692 DERIVED

MeSH Terms

Conditions

Fanconi Anemia

Interventions

Antilymphocyte Serum; Cyclophosphamide; Cyclosporine; Filgrastim; fludarabine; Methylprednisolone

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Biological Factors; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds

Study Officials

• John E. Wagner, Jr.

  Masonic Cancer Center, University of Minnesota

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 2, 2000
• First Posted: June 5, 2000
• Study Start: February 1, 2000
• Last Updated: June 24, 2005

Record last verified: 2004-07

Locations

US (1)