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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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Fred Hutch/University of Washington Cancer Consortium

    RECRUITING

    Seattle, Washington, 98109, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact