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NCT00919503 · ClinicalTrials.gov registry record · Phase 2
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
A Phase 2 study, sponsored by Fred Hutchinson Cancer Center.
- Completed
- Registry status
- Phase 2
- Development phase
- 98
- Enrollment target
NCT00919503: Completed Phase 2 study, sponsored by Fred Hutchinson Cancer Center.
NCT00919503 is a Phase 2 study that has completed, run by Fred Hutchinson Cancer Center. The registered enrollment target is 98 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (26% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00919503, a Phase 2 study, has completed, sponsored by Fred Hutchinson Cancer Center.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 98 participants
- Enrollment target
Study Summary
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (noncancerous) diseases such as primary immunodeficiency disorders, bone marrow failure syndromes, hemoglobinopathies, and inborn errors of metabolism (metabolic disorders). Powerful chemotherapy drugs and/or radiation are often used to condition the patient before infusion of the new healthy donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal bone marrow which doesn't work properly in order to make way for the new healthy donor cells which functions normally. Although effective in curing the patient's disease, many hematopoietic cell transplantation regimens use intensive chemotherapy and/or radiation which can be quite toxic, have significant side effects, and can potentially be life-threatening. Investigators are investigating whether a new conditioning regimen that uses less intensive drugs (treosulfan and fludarabine phosphate) with or without low dose radiation results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (noncancerous) diseases.
Primary Outcome
Number of patients engrafted (\>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant
Interventions
- OTHER Laboratory Biomarker Analysis
- BIOLOGICAL Anti-Thymocyte Globulin
- DRUG Fludarabine Phosphate
- DRUG Cyclosporine
- PROCEDURE Allogeneic Bone Marrow Transplantation
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 98 participants |
| Start Date | 2009-07-31 |
| Est. Completion | 2020-06-10 |
| Phase | Phase 2 |
What the finished NCT00919503 record still lists
NCT00919503 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 98 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (26% lower).
The record links to 0 conditions, and to 5 interventions - of which Laboratory Biomarker Analysis is the first listed.
NCT00919503 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00919503 about?
NCT00919503 is a clinical study titled "Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders". This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many...
What is the current status of trial NCT00919503?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 98 participants. The study started on 2009-07-31. Estimated completion is 2020-06-10.
What interventions are being tested in trial NCT00919503?
The interventions under investigation include: Laboratory Biomarker Analysis (OTHER), Anti-Thymocyte Globulin (BIOLOGICAL), Fludarabine Phosphate (DRUG), Cyclosporine (DRUG), Allogeneic Bone Marrow Transplantation (PROCEDURE).
Who is sponsoring clinical trial NCT00919503?
This trial is sponsored by Fred Hutchinson Cancer Center, which has 483 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00919503's enrollment target sits among peer trials
98 743rd of 2000 higher than 1,255 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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