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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004787
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : June 24, 2005
James Whitcomb Riley Hospital for Children
Information provided by:
Office of Rare Diseases (ORD)

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.

Condition or disease Intervention/treatment Phase
Shwachman Syndrome Fanconi's Anemia Dyskeratosis Congenita Thrombocytopenia Drug: filgrastim Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Primary Purpose: Treatment
Study Start Date : December 1994

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004787

Sponsors and Collaborators
National Center for Research Resources (NCRR)
James Whitcomb Riley Hospital for Children
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Study Chair: David A. Williams James Whitcomb Riley Hospital for Children

Layout table for additonal information Identifier: NCT00004787     History of Changes
Other Study ID Numbers: 199/11877
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: December 2001
Keywords provided by Office of Rare Diseases (ORD):
Fanconi's anemia
Shwachman syndrome
aplastic anemia
dermatologic disorders
dyskeratosis congenita
hematologic disorders
rare disease
Additional relevant MeSH terms:
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Fanconi Syndrome
Fanconi Anemia
Dyskeratosis Congenita
Pathologic Processes
Hematologic Diseases
Blood Platelet Disorders
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Skin Abnormalities
Congenital Abnormalities
Genetic Diseases, X-Linked
Skin Diseases, Genetic
Skin Diseases
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs