Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders
Public ClinicalTrials.gov record NCT03980769. 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 Non-Malignant Disorders Using Treosulfan, Fludarabine, and Thiotepa
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This phase II clinical trial studies how well treosulfan, thiotepa, fludarabine, and rabbit anti-thymocyte globulin (rATG) before donor stem cell transplantation works in treating patients with nonmalignant (non-cancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (non-cancerous) diseases such as primary immunodeficiency disorders, immune dysregulatory disorders, hemophagocytic lymphohistiocytosis, bone marrow failure syndromes, and hemoglobinopathies. Powerful chemotherapy drugs 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 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, thiotepa, and fludarabine phosphate) results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (non-cancerous) diseases.
Study identification
- NCT ID
- NCT03980769
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 40 participants
Conditions and interventions
Interventions
- Thiotepa Drug
- Treosulfan Drug
- Fludarabine Phosphate Drug
- Rabbit Anti-Thymocyte Globulin Biological
- Allogeneic Hematopoietic Stem Cell Transplantation Procedure
- Bone Marrow Biopsy Procedure
- Bone Marrow Aspiration Procedure
- Magnetic Resonance Imaging Procedure
- Biospecimen Collection Procedure
Drug · Biological · Procedure
Eligibility (public fields only)
- Age range
- Up to 50 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
- May 5, 2021 (Actual)
- Primary completion
- Jul 1, 2028 (Estimated)
- Completion
- Jul 1, 2028 (Estimated)
- Last update posted
- Sep 16, 2026
2021 – 2028
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | Recruiting |
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 NCT03980769, 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 Sep 16, 2026 · Synced Oct 16, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03980769 live on ClinicalTrials.gov.