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NCT00987480 · ClinicalTrials.gov registry record · Phase 2
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
A Phase 2 study, sponsored by Memorial Sloan Kettering Cancer Center.
- Completed
- Registry status
- Phase 2
- Development phase
- 45
- Enrollment target
NCT00987480: Completed Phase 2 study, sponsored by Memorial Sloan Kettering Cancer Center.
NCT00987480 is a Phase 2 study that has completed, run by Memorial Sloan Kettering Cancer Center. The registered enrollment target is 45 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (66% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00987480, a Phase 2 study, has completed, sponsored by Memorial Sloan Kettering Cancer Center.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 45 participants
- Enrollment target
Study Summary
This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic syndrome (MDS) or leukemia (acute myelogenous leukemia or AML). Without treatment these complications of Fanconia anemia (FA) are fatal. The only treatment that can cure these complications is an allogeneic transplant of stem cells, meaning, giving the patient bone marrow cells from a healthy donor that can produce normal blood cells that will replace the bone marrow that is sick. What has been given for the treatment of FA in the past is to use a combination of low doses of radiation to the whole body (total body irradiation) and low doses of the chemotherapy drugs (cyclophosphamide and fludarabine) before the transplant. However, the use of radiation can, later on, increase the chances of getting a second cancer of the skin, head or the neck. These chances of a second cancer are higher than normal in patients with FA. The purpose of this study is to find out if the doctors can do the same thing with the same chemotherapy drugs used in the past. However physicians will use another chemotherapy drug called busulfan instead of the radiation. The goal of this study is to get rid of the short term and long term risks of the radiation. The first new part of this treatment will be to replace drugs for radiation with chemotherapy drugs.
Interventions
- DRUG Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.
- DEVICE CliniMACS device
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2009-09-25 |
| Est. Completion | 2017-07-10 |
| Phase | Phase 2 |
What the finished NCT00987480 record still lists
NCT00987480 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 45 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (66% lower).
The record links to 0 conditions, and to 2 interventions - of which Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine. is the first listed.
NCT00987480 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00987480 about?
NCT00987480 is a clinical study titled "Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine". This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic syndrome (MDS) or leukemia (acute myelogenous leukemia or AML). Withou...
What is the current status of trial NCT00987480?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 45 participants. The study started on 2009-09-25. Estimated completion is 2017-07-10.
What interventions are being tested in trial NCT00987480?
The interventions under investigation include: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine. (DRUG), CliniMACS device (DEVICE).
Who is sponsoring clinical trial NCT00987480?
This trial is sponsored by Memorial Sloan Kettering Cancer Center, which has 1,884 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00987480's enrollment target sits among peer trials
45 1296th of 2000 higher than 679 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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