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| 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 |
| Study Type: | Interventional |
| Study Design: | Treatment |
| Estimated Enrollment: | 20 |
| Study Start Date: | December 1994 |
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 |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Inherited bone marrow failure syndrome, including:
--Prior/Concurrent Therapy--
--Patient Characteristics--
Contacts and Locations| National Center for Research Resources (NCRR) |
| James Whitcomb Riley Hospital for Children |
| Study Chair: | David A. Williams | James Whitcomb Riley Hospital for Children |
More Information
| 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 |
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