Ignite Creation Date:
2024-05-05 @ 11:19 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004787
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Brief Title:
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
Sponsor:
National Center for Research Resources NCRR
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
2001-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
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
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
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UTMB-416 | None | None | None |