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Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders
NCT03980769 · View on ClinicalTrials.gov ↗
Study Summary
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.
Conditions Studied
Interventions
- DRUG Thiotepa
- DRUG Fludarabine Phosphate
- PROCEDURE Allogeneic Hematopoietic Stem Cell Transplantation
- DRUG Treosulfan
- BIOLOGICAL Rabbit Anti-Thymocyte Globulin
Study Locations (1)
Washington
- Fred Hutch/University of Washington Cancer Consortium — Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2021-05-05 |
| Est. Completion | 2028-07-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03980769
The ClinicalTrials.gov registry entry for NCT03980769 describes a study currently listed as recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Fred Hutchinson Cancer Center, which has 319 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder appearing as the primary indexed condition, and to 5 interventions — of which Thiotepa is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03980769 reports 1 study location spanning 1 distinct geographic area — top geographies include Washington. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03980769 about?
NCT03980769 is a clinical study titled "Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders". 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 fo...
What is the current status of trial NCT03980769?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2021-05-05. Estimated completion is 2028-07-01.
What conditions does trial NCT03980769 study?
This clinical trial studies the following conditions: Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03980769?
The interventions under investigation include: Thiotepa (DRUG), Fludarabine Phosphate (DRUG), Allogeneic Hematopoietic Stem Cell Transplantation (PROCEDURE), Treosulfan (DRUG), Rabbit Anti-Thymocyte Globulin (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03980769?
This trial is sponsored by Fred Hutchinson Cancer Center, which has 319 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03980769 being conducted?
This trial has 1 study location across Washington. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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