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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

Primary Purpose

Fanconi's Anemia

Status
Unknown status
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
filgrastim
Autologous stem cells transduced with FACC retroviral vector
Autologous Stem Cell Transplantation
Sponsored by
University of Minnesota
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi's Anemia focused on measuring Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Fanconi's anemia, complementation group C (FACC) Confirmed by diepoxybutane or mitomycin testing AND DNA analysis indicating FACC mutations Patients at least 25 kg weight No acute leukemia OR Bone marrow aspirate with greater than 10% blasts No patients who elect bone marrow transplantation --Prior/Concurrent Therapy-- At least 14 days since prior therapy for any acute viral, bacterial, or fungal infection --Patient Characteristics-- Performance status: Karnofsky 40-100% Hepatic: SGOT, SGPT, and alkaline phosphatase no greater than 5 times upper limit of normal (ULN) PT/PTT no greater than 1.5 times ULN Serum amylase no greater than 1.5 times ULN Bilirubin no greater than 2.5 mg/dL Triglyceride less than 400 mg/dL Renal: Creatinine clearance greater than 50 mL/min Cardiovascular: Normal cardiac function No ischemic heart disease that may be considered an anesthetic or operative risk Pulmonary: No lung disease that may be considered an anesthetic or operative risk Resting transcutaneous oxygen saturation greater than 90% on room air Other: HIV negative Hepatitis B surface antigen negative No underlying condition that may preclude study therapy (e.g., allergies to study reagents) No acute viral, bacterial, or fungal infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception

Sites / Locations

    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
    NCT00005896
    Brief Title
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2001
    Overall Recruitment Status
    Unknown status
    Study Start Date
    March 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 safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C. II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients. III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.
    Detailed Description
    PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every 2 months for up to 4 courses total. Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for the next year, and then yearly thereafter.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Fanconi's Anemia
    Keywords
    Fanconi's anemia, aplastic anemia, hematologic disorders, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1
    Enrollment
    6 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    filgrastim
    Intervention Type
    Genetic
    Intervention Name(s)
    Autologous stem cells transduced with FACC retroviral vector
    Intervention Type
    Procedure
    Intervention Name(s)
    Autologous Stem Cell Transplantation

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    5 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Fanconi's anemia, complementation group C (FACC) Confirmed by diepoxybutane or mitomycin testing AND DNA analysis indicating FACC mutations Patients at least 25 kg weight No acute leukemia OR Bone marrow aspirate with greater than 10% blasts No patients who elect bone marrow transplantation --Prior/Concurrent Therapy-- At least 14 days since prior therapy for any acute viral, bacterial, or fungal infection --Patient Characteristics-- Performance status: Karnofsky 40-100% Hepatic: SGOT, SGPT, and alkaline phosphatase no greater than 5 times upper limit of normal (ULN) PT/PTT no greater than 1.5 times ULN Serum amylase no greater than 1.5 times ULN Bilirubin no greater than 2.5 mg/dL Triglyceride less than 400 mg/dL Renal: Creatinine clearance greater than 50 mL/min Cardiovascular: Normal cardiac function No ischemic heart disease that may be considered an anesthetic or operative risk Pulmonary: No lung disease that may be considered an anesthetic or operative risk Resting transcutaneous oxygen saturation greater than 90% on room air Other: HIV negative Hepatitis B surface antigen negative No underlying condition that may preclude study therapy (e.g., allergies to study reagents) No acute viral, bacterial, or fungal infection 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

    12. IPD Sharing Statement

    Learn more about this trial

    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

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