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NCT00005783 · ClinicalTrials.gov registry record · Phase 1
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
A Phase 1 study, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
- Completed
- Registry status
- Phase 1
- Development phase
- 50
- Enrollment target
NCT00005783 is a Phase 1 study that has completed, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The registered enrollment target is 50 participants.
The verdict
NCT00005783, a Phase 1 study, has completed, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 50 participants
- Enrollment target
Study 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 I/II 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.
Interventions
- DRUG Recombinant-methionyl human stem cell factor
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2000-03 |
| Est. Completion | 2000-10 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00005783
The ClinicalTrials.gov registry entry for NCT00005783 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 50 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which has 490 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Recombinant-methionyl human stem cell factor is the first listed.
NCT00005783 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00005783 about?
NCT00005783 is a clinical study titled "A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders". 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 deformabil...
What is the current status of trial NCT00005783?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 50 participants. The study started on 2000-03. Estimated completion is 2000-10.
What interventions are being tested in trial NCT00005783?
The interventions under investigation include: Recombinant-methionyl human stem cell factor (DRUG).
Who is sponsoring clinical trial NCT00005783?
This trial is sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which has 490 total clinical trials registered on ClinicalTrials.gov.
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