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NCT00001399 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for the Treatment of Fanconi's Anemia Type C
A Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT00001399: Completed Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT00001399 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001399, a Phase 1 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study Summary
Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition, some patients with Fanconi's anemia have physical defects usually involving the skeleton and kidneys. The major problem for most patients is aplastic anemia, the blood counts for red blood cells, white blood cells, and platelets are low because the bone marrow fails to produce these cells. Some patients with Fanconi's anemia can develop leukemia or cancers of other organs. Many laboratory studies have suggested that Fanconi's anemia is caused by an inherited defect in the ability of cells to repair DNA. Recently, the gene for one of the four types of Fanconi's anemia, type C, has been identified. It is known that this gene is defective in patients with Fanconi's anemia type C. Researchers have conducted laboratory studies that suggest Fanconi's anemia type C may be treatable with gene therapy. Gene therapy works by placing a normal gene into the cells of patients with abnormal genes responsible for Fanconi's anemia type C. After the normal gene is in place, new normal cells can develop and grow. Drugs can be given to these patients kill the remaining abnormal cells. The new cells containing normal genes and will not be harmed by these drugs. The purpose of this study is to test whether researchers can safely place the normal Fanconi's anemia type C gene into cells of patients with the disease. The gene will be placed into special cells in the bone marrow called stem cells. These stem cells are responsible for producing new red blood cells, white blood cells, and platelets.
Interventions
- DRUG Transduced CD34+ Cells
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 1993-12-03 |
| Est. Completion | 2009-02-11 |
| Phase | Phase 1 |
What the finished NCT00001399 record still lists
NCT00001399 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which Transduced CD34+ Cells is the first listed.
NCT00001399 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00001399 about?
NCT00001399 is a clinical study titled "Gene Therapy for the Treatment of Fanconi's Anemia Type C". Fanconi's Anemia is an inherited disorder that can produce bone marrow failure. In addition, some patients with Fanconi's anemia have physical defects usually involving the skeleton and kidneys. The major problem for most patients is aplastic anemia, the blood counts for red blood cells, white blood...
What is the current status of trial NCT00001399?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 1993-12-03. Estimated completion is 2009-02-11.
What interventions are being tested in trial NCT00001399?
The interventions under investigation include: Transduced CD34+ Cells (DRUG).
Who is sponsoring clinical trial NCT00001399?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00001399's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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