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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00005898
Other study ID # 199/15109
Secondary ID UMN-MT-1999-05UM
Status Completed
Phase Phase 1/Phase 2
First received June 2, 2000
Last updated June 23, 2005
Start date February 2000

Study information

Verified date July 2004
Source Office of Rare Diseases (ORD)
Contact n/a
Is FDA regulated No
Health authority Unspecified
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 30
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A to 34 Years
Eligibility 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

Study Design

Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
anti-thymocyte globulin

cyclophosphamide

cyclosporine

filgrastim

fludarabine

methylprednisolone


Locations

Country Name City State
United States University of Minnesota Cancer Center Minneapolis Minnesota

Sponsors (1)

Lead Sponsor Collaborator
University of Minnesota - Clinical and Translational Science Institute

Country where clinical trial is conducted

United States, 

See also
  Status Clinical Trial Phase
Completed NCT03609827 - Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Active, not recruiting NCT00005896 - Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia Phase 1
Completed NCT00005892 - Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia N/A
Completed NCT00005891 - Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Fanconi's Anemia N/A
Completed NCT00001749 - Medical Treatment for Diamond Blackfan Anemia Phase 2
Completed NCT03609814 - Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Completed NCT00004378 - Stem Cell Transplantation (SCT) for Genetic Diseases N/A
Completed NCT00001399 - Gene Therapy for the Treatment of Fanconi's Anemia Type C Phase 1
Completed NCT00004787 - Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes Phase 2
Active, not recruiting NCT00006127 - Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia Phase 1