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

Primary Purpose

Fanconi's Anemia

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
anti-thymocyte globulin
cyclophosphamide
cyclosporine
filgrastim
fludarabine
methylprednisolone
Sponsored by
University of Minnesota
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi's Anemia

Eligibility Criteria

0 Years - 34 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Minnesota Cancer Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
June 2, 2000
Last Updated
June 23, 2005
Sponsor
University of Minnesota
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1. Study Identification

Unique Protocol Identification Number
NCT00005898
Brief Title
Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
July 2004
Overall Recruitment Status
Completed
Study Start Date
February 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
University of Minnesota

4. Oversight

5. Study Description

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.
Detailed Description
PROTOCOL OUTLINE: Donor bone marrow, peripheral blood, or umbilical cord blood is processed to harvest CD34+ cells. Patients receive preparative cytoreductive therapy comprising total body irradiation on day -6; cyclophosphamide IV over 2 hours on days -5 to -2; fludarabine IV over 30 minutes on days -5 to -2; methylprednisone IV on days -5 to 24; anti-thymocyte globulin IV over 4-6 hours on days -5 to -1; cyclosporine IV over 2 hours every 12 hours (every 8 hours for patients less than 40 kg in weight) on days -3 to 180, and then tapering in the absence of graft-versus-host disease; hematopoietic cell transplantation on day 0; and filgrastim (G-CSF) IV starting on day 1 and continuing until blood counts recover. Patients are followed at days 60, 90, and 180, and then annually for 3 years.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi's Anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Enrollment
30 (false)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
anti-thymocyte globulin
Intervention Type
Drug
Intervention Name(s)
cyclophosphamide
Intervention Type
Drug
Intervention Name(s)
cyclosporine
Intervention Type
Drug
Intervention Name(s)
filgrastim
Intervention Type
Drug
Intervention Name(s)
fludarabine
Intervention Type
Drug
Intervention Name(s)
methylprednisolone

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
34 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John E. Wagner, Jr.
Organizational Affiliation
Masonic Cancer Center, University of Minnesota
Official's Role
Study Chair
Facility Information:
Facility Name
University of Minnesota Cancer Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
25824692
Citation
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.
Results Reference
derived

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

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