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NCT00604201 · ClinicalTrials.gov registry record · Phase 2
Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 31
- Enrollment target
NCT00604201: Completed Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT00604201 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 31 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00604201, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 31 participants
- Enrollment target
Study Summary
This study will evaluate the safety and effectiveness of treating patients with severe aplastic anemia (SAA) or myelodysplastic syndrome (MDS) with both peripheral blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor. Patients with SAA or MDS for whom other treatments have failed or are not available may be eligible for this study. Candidates may not have a tissue-matched sibling or matched unrelated donor and must have a family member who is a partial tissue type match. Participants undergo the following tests and procedures: * Insertion of a central intravenous (IV) line (plastic tube) into a large vein. The tube is used for giving the donated stem cells and antibiotics and other medicines, for transfusions of red blood cells and platelets, and for collecting blood samples. * Preparatory chemotherapy (fludarabine, cyclophosphamide and anti-thymocyte globulin) and total body irradiation to suppress immunity and prevent rejection of the donated cells. * Infusion of the donated stem cells and umbilical cord cells. * Immune suppression with the drugs tacrolimus, mycophenolate mofetil and prednisone to prevent rejection of the donated cells and to prevent graft-versus-host disease (GVHD), a complication of stem cell transplants in which the donors immune cells destroy the patients healthy tissues. The average hospital stay after stem cell transplantation is 3 to 4 weeks. Patients return for frequent follow-up visits for the first 2 to 4 months after transplantation. Once the patient returns home, his or her referring physician is asked to send results of any laboratory testing to the NIH researchers at least every 3 months for the first 3 years and annually thereafter. Patient follow-up visits are scheduled at NIH at 1, 2, 3, 4 and 5 years after transplantation to monitor for signs of disease or post-transplantation complications, such as infection or GVHD. After 5 years, participants are offered the opportunity to enrol
Primary Outcome
Participants who reached engraftment by day 42 (±3 days) defined as an absolute neutrophil count (ANC) of U\> U500 cells/µl
Interventions
- BIOLOGICAL Umbilical Cord Blood
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 31 participants |
| Start Date | 2008-05-21 |
| Est. Completion | 2021-09-14 |
| Phase | Phase 2 |
What the finished NCT00604201 record still lists
NCT00604201 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 31 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 1 intervention - of which Umbilical Cord Blood is the first listed.
NCT00604201 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00604201 about?
NCT00604201 is a clinical study titled "Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome". This study will evaluate the safety and effectiveness of treating patients with severe aplastic anemia (SAA) or myelodysplastic syndrome (MDS) with both peripheral blood stem cells from a family member and umbilical cord blood stem cells from an unrelated donor. Patients with SAA or MDS for whom ot...
What is the current status of trial NCT00604201?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 31 participants. The study started on 2008-05-21. Estimated completion is 2021-09-14.
What interventions are being tested in trial NCT00604201?
The interventions under investigation include: Umbilical Cord Blood (BIOLOGICAL).
Who is sponsoring clinical trial NCT00604201?
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 NCT00604201's enrollment target sits among peer trials
31 1578th of 2000 higher than 419 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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