New transplant cocktail aims to tame blood cancer without severe side effects

NCT ID NCT07565220

Recruiting now Disease control Sponsor: Sawa Ito, MD Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jul 02, 2026 · Updated 3 times

Summary

This early-stage trial is testing a new combination of chemotherapy drugs (thiotepa, busulfan, and fludarabine) followed by a stem cell transplant for people with certain blood cancers like leukemia and myelodysplastic syndrome. The goal is to see if this approach can reduce the dose of a post-transplant drug (cyclophosphamide) that helps prevent graft-versus-host disease, a serious complication. The study will enroll 48 adults aged 18-70 who are candidates for a stem cell transplant.

What this could mean

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

Active substance: Thiotepa, Fludarabine, Busulfan (chemotherapy drugs) and a stem cell transplant
What this could lead to: If successful, this could lead to a safer and more effective transplant procedure for people with blood cancers, reducing the risk of graft-versus-host disease and relapse.
What could go wrong: This is an early Phase 1 trial with only 48 participants, so the results may not apply to everyone. The treatment involves strong chemotherapy and carries risks like infection, organ damage, and graft-versus-host disease.

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Conditions

The condition(s) this trial relates to.

acute leukemia acute lymphoblastic leukemia acute myeloid leukemia graft versus host disease myelodysplastic syndrome Myelodysplastic Syndromes Myeloproliferative Disorders myeloproliferative neoplasm Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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

ACUTE LEUKEMIA ACUTE LYMPHOCYTIC LEUKEMIA (ALL) ACUTE MYELOID LEUKEMIA (AML) HIGH-RISK MYELOPROLIFERATIVE NEOPLASM (MPN) MYELODYSPLASTIC SYNDROME (MDS) MYELOPROLIFERATIVE NEOPLASM (MPN)

Contacts and locations

Locations

UMPC Hillman Cancer Center
RECRUITING

Pittsburgh, Pennsylvania, 15213, United States

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