Milder chemo may make stem cell transplants safer for Non-Cancer patients

NCT ID NCT03980769

Recruiting now Disease control Sponsor: Fred Hutchinson Cancer Center Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditioning regimen can still allow the donor cells to take hold (engraft) while causing fewer toxic side effects than standard high-dose chemo. The study is currently recruiting.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: treosulfan, fludarabine, thiotepa, and rabbit anti-thymocyte globulin (rATG)
What this could lead to: If successful, this could offer a safer way to cure non-cancerous blood disorders using donor stem cells, with fewer severe side effects than standard high-dose chemotherapy.
What could go wrong: This is an early phase 2 trial with only 40 participants, so results may not apply to everyone. There are still risks of graft failure, infection, or other serious complications from the transplant.

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Conditions

The condition(s) this trial relates to.

bone marrow failure syndrome hematologic disorder hemoglobinopathy hemophagocytic syndrome inborn error of immunity premalignant hematological system disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NON-NEOPLASTIC HEMATOPOIETIC AND LYMPHOID CELL DISORDER

Contacts and locations

Locations

Fred Hutch/University of Washington Cancer Consortium
RECRUITING

Seattle, Washington, 98109, United States

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