NCT00004787

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (planned)
• Sponsor: National Center for Research Resources (NCRR) · NIH
• Sex: All
• Age: 0 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Shwachman Syndrome
• Fanconi's Anemia
• Dyskeratosis Congenita
• Thrombocytopenia

Interventions

Type	Name	Description
DRUG	filgrastim

Timeline

• Start date: 1994-12-01
• First posted: 2000-02-25
• Last updated: 2005-06-24