Ignite Creation Date:
2024-05-05 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 9:05 AM
Study NCT ID:
NCT00005783
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
2000-06-03
Brief Title:
A Phase III Trial of Recombinant-Methionyl Human Stem Cell Factor SCF in Adult Patients With Sickling Disorders
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Phase III Trial of Recombinant-Methionyl Human Stem Cell Factor SCF in Adult Patients With Sickling Disorders
Status:
COMPLETED
Status Verified Date:
2000-02
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:
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which in the homozygous state produces an abnormal hemoglobin that is prone to polymer formation when deoxygenated The polymerized hemoglobin leads to impaired deformability and sickling of red blood cells which subsequently lodge in end-arterioles producing the classic and most prominent feature of the disorder repeated vasoocclusive crises Despite knowledge of the precise genetic defect for decades only recently has there been therapeutic impact based upon this knowledge when a clear benefit from treatment with hydroxyurea a cell cycle-specific agent administered to induce production of fetal hemoglobin HbF by stimulating gamma-globin synthesis was reported in patients with sickle cell disease SCD The reduction in the frequency and severity of vasoocclusive crises seen has been attributed to the increase in HbF levels in responsive patients While the majority of patients demonstrate a rise in HbF not all such patients benefit from treatment Given these results alternative agents that also stimulate the production of HbF warrant investigation in the treatment of SCD Recombinant-methionyl human stem cell factor SCF is a hematopoietic growth factor with activity on immature hematopoietic progenitor cells SCF stimulates the production of HbF in vitro and in vivo and this effect is attainable without the myelosuppression associated with hydroxyurea In this phase III trial we will administer SCF in a dose escalating fashion to patients with sickling disorders Parameters to be measured are HbF levels F cell levels peripheral blood CD34 levels frequency duration and severity of vasoocclusive crises and toxicity
Detailed Description:
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which in the homozygous state produces an abnormal hemoglobin that is prone to polymer formation when deoxygenated The polymerized hemoglobin leads to impaired deformability and sickling of red blood cells which subsequently lodge in end-arterioles producing the classic and most prominent feature of the disorder repeated vasoocclusive crises Despite knowledge of the precise genetic defect for decades only recently has there been therapeutic impact based upon this knowledge when a clear benefit from treatment with hydroxyurea a cell cycle-specific agent administered to induce production of fetal hemoglobin HbF by stimulating gamma-globin synthesis was reported in patients with sickle cell disease SCD The reduction in the frequency and severity of vasoocclusive crises seen has been attributed to the increase in HbF levels in responsive patients While the majority of patients demonstrate a rise in HbF not all such patients benefit from treatment Given these results alternative agents that also stimulate the production of HbF warrant investigation in the treatment of SCD Recombinant-methionyl human stem cell factor SCF is a hematopoietic growth factor with activity on immature hematopoietic progenitor cells SCF stimulates the production of HbF in vitro and in vivo and this effect is attainable without the myelosuppression associated with hydroxyurea In this phase III trial we will administer SCF in a dose escalating fashion to patients with sickling disorders Parameters to be measured are HbF levels F cell levels peripheral blood CD34 levels frequency duration and severity of vasoocclusive crises and toxicity
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 00-DK-0087 | None | None | None |