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NCT00011505 · ClinicalTrials.gov registry record · Phase 2
Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 15
- Enrollment target
NCT00011505 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 15 participants.
The verdict
NCT00011505, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 15 participants
- Enrollment target
Study Summary
This study will provide information needed to develop more effective treatments for patients with Diamond-Blackfan anemia (DBA). Current treatments include steroids, such as prednisone, and blood transfusions. These treatments have potential long-term risk and side effects, including osteoporosis and impaired growth from steroids or iron overload from transfusions. In addition, as patients reach adulthood, they can develop acute leukemia or bone marrow failure. The only cure for DBA is bone marrow transplant, a procedure that itself carries serious risks and is an option for only about 25 percent of patients. DBA is caused by a mutation (error) in a gene that codes for producing red blood cells from stem cells (blood-forming cells produced by the bone marrow). In 5 to 10 years, gene transfer therapy may prove to be an effective treatment for DBA. Before this treatment can be considered, however, more information is needed about DBA patients and how their stem cells function. This study will examine: 1) whether stem cells of patients with DBA respond to G-CSF the same way those of healthy people do. (G-CSF is a drug that causes stem cells to move from the bone marrow to the blood stream, where they can be collected more easily and in larger numbers by a procedure called leukapheresis, described below. If G-CSF does not work well in DBA patients, other collection strategies will have to be explored); and 2) whether the genetic error in DBA can be corrected by gene transfer into patients' stem cells. Patients with Diamond-Blackfan anemia 4 years of age and older who weigh at least 27 pounds and who are dependent on red blood cell transfusions may be eligible for this study. Candidates will have a medical history taken and a physical examination and will be seen by the Clinical Center's Department of Medicine Transfusion for leukapheresis evaluation. They will have a bone marrow aspiration and biopsy to confirm the diagnosis of DBA. For these tests, the hip area is an
Interventions
- DRUG G-CSF
- PROCEDURE Leukapheresis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2001-02 |
| Est. Completion | 2006-04 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00011505
The ClinicalTrials.gov registry entry for NCT00011505 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 501 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which G-CSF is the first listed.
NCT00011505 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00011505 about?
NCT00011505 is a clinical study titled "Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia". This study will provide information needed to develop more effective treatments for patients with Diamond-Blackfan anemia (DBA). Current treatments include steroids, such as prednisone, and blood transfusions. These treatments have potential long-term risk and side effects, including osteoporosis an...
What is the current status of trial NCT00011505?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 15 participants. The study started on 2001-02. Estimated completion is 2006-04.
What interventions are being tested in trial NCT00011505?
The interventions under investigation include: G-CSF (DRUG), Leukapheresis (PROCEDURE).
Who is sponsoring clinical trial NCT00011505?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
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