Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
Public ClinicalTrials.gov record NCT00919503. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT00919503
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 98 participants
Conditions and interventions
Interventions
- Allogeneic Bone Marrow Transplantation Procedure
- Anti-Thymocyte Globulin Biological
- Cyclosporine Drug
- Fludarabine Phosphate Drug
- Laboratory Biomarker Analysis Other
- Methotrexate Drug
- Mycophenolate Mofetil Drug
- Peripheral Blood Stem Cell Transplantation Procedure
- Tacrolimus Drug
- Total-Body Irradiation Radiation
- Treosulfan Drug
- Umbilical Cord Blood Transplantation Procedure
Procedure · Biological · Drug + 2 more
Eligibility (public fields only)
- Age range
- Up to 49 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 30, 2009
- Primary completion
- Jun 9, 2020
- Completion
- Jun 9, 2020
- Last update posted
- Aug 12, 2021
2009 – 2020
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Oregon Health and Science University | Portland | Oregon | 97239 | — |
| Vanderbilt University/Ingram Cancer Center | Nashville | Tennessee | 37232 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | — |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00919503, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 12, 2021 · Synced Sep 4, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00919503 live on ClinicalTrials.gov.