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Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Primary Purpose

Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita

Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
filgrastim
Sponsored by
National Center for Research Resources (NCRR)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Shwachman Syndrome focused on measuring Fanconi's anemia, Shwachman syndrome, aplastic anemia, dermatologic disorders, dyskeratosis congenita, hematologic disorders, rare disease, thrombocytopenia

Eligibility Criteria

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

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Inherited bone marrow failure syndrome, including: Fanconi's anemia Dyskeratosis congenita Shwachman syndrome Amegakaryocytic thrombocytopenia Decreased megakaryocytes in infancy No thrombocytopenia with absent radius syndrome (TAR) No trisomy 13 or 18 No clonal bone marrow karyotype --Prior/Concurrent Therapy-- At least 4 weeks since growth factors Concurrent therapy allowed if not altered for 30 days prior to entry through week 8 No concurrent investigational drugs --Patient Characteristics-- Hematopoietic: ANC <1000 No leukemia Other: No medical or psychiatric contraindication to protocol participation No pregnant or nursing women

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    June 23, 2005
    Sponsor
    National Center for Research Resources (NCRR)
    Collaborators
    James Whitcomb Riley Hospital for Children
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004787
    Brief Title
    Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2001
    Overall Recruitment Status
    Completed
    Study Start Date
    December 1994 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    National Center for Research Resources (NCRR)
    Collaborators
    James Whitcomb Riley Hospital for Children

    4. Oversight

    5. Study Description

    Brief Summary
    OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes. II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF. III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.
    Detailed Description
    PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF) subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted to maintain an absolute neutrophil count above 1500. Patients are removed from study for failure to achieve a complete response by week 16, unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or the onset of leukemia.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, Thrombocytopenia
    Keywords
    Fanconi's anemia, Shwachman syndrome, aplastic anemia, dermatologic disorders, dyskeratosis congenita, hematologic disorders, rare disease, thrombocytopenia

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Enrollment
    20 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    filgrastim

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    0 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Inherited bone marrow failure syndrome, including: Fanconi's anemia Dyskeratosis congenita Shwachman syndrome Amegakaryocytic thrombocytopenia Decreased megakaryocytes in infancy No thrombocytopenia with absent radius syndrome (TAR) No trisomy 13 or 18 No clonal bone marrow karyotype --Prior/Concurrent Therapy-- At least 4 weeks since growth factors Concurrent therapy allowed if not altered for 30 days prior to entry through week 8 No concurrent investigational drugs --Patient Characteristics-- Hematopoietic: ANC <1000 No leukemia Other: No medical or psychiatric contraindication to protocol participation No pregnant or nursing women
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    David A. Williams
    Organizational Affiliation
    James Whitcomb Riley Hospital for Children
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    8781414
    Citation
    Rackoff WR, Orazi A, Robinson CA, Cooper RJ, Alter BP, Freedman MH, Harris RE, Williams DA. Prolonged administration of granulocyte colony-stimulating factor (filgrastim) to patients with Fanconi anemia: a pilot study. Blood. 1996 Sep 1;88(5):1588-93.
    Results Reference
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    Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

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