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

This study has been completed.

Sponsors and Collaborators: National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children
Information provided by: Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier: NCT00004787
  Purpose

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.


Condition Intervention Phase
Shwachman Syndrome
Fanconi's Anemia
Dyskeratosis Congenita
Thrombocytopenia
Drug: filgrastim
Phase II

Genetics Home Reference related topics:   L1 syndrome    Shwachman-Diamond syndrome   

MedlinePlus related topics:   Anemia   

ChemIDplus related topics:   Filgrastim    Sargramostim    Granulocyte-macrophage colony-stimulating factor    Granulocyte colony-stimulating factor   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:   20
Study Start Date:   December 1994

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.

  Eligibility
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004787

Sponsors and Collaborators
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children

Investigators
Study Chair:     David A. Williams     James Whitcomb Riley Hospital for Children    
  More Information

Publications:

Study ID Numbers:   199/11877, UTMB-416
First Received:   February 24, 2000
Last Updated:   June 23, 2005
ClinicalTrials.gov Identifier:   NCT00004787
Health Authority:   United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
Fanconi's anemia  
Shwachman syndrome  
aplastic anemia  
dermatologic disorders  
dyskeratosis congenita
hematologic disorders
rare disease
thrombocytopenia

Study placed in the following topic categories:
Shwachman-Diamond syndrome
Bone Marrow failure syndromes
Thrombocytopenia
Dyskeratosis congenita
Fanconi's anemia
Dyskeratosis Congenita
Anemia, Aplastic
Genetic Diseases, X-Linked
Congenital Abnormalities
Skin Diseases, Genetic
Metabolic Diseases
Skin Diseases
Hematologic Diseases
Blood Platelet Disorders
Fanconi Anemia
Rare Diseases
Anemia
Skin Abnormalities
Pancytopenia
Shwachman syndrome
Thrombocytopathy
Genetic Diseases, Inborn
Bone Marrow Diseases
Aplastic anemia
Metabolic disorder

Additional relevant MeSH terms:
Pathologic Processes
Disease
Anemia, Hypoplastic, Congenital
Syndrome
DNA Repair-Deficiency Disorders

ClinicalTrials.gov processed this record on September 05, 2008




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