Milder stem cell transplant could tame aggressive sickle cell disease

NCT ID NCT01499888

First seen Jul 08, 2026 · Last updated Jul 09, 2026 · Updated 1 time

Summary

This clinical trial tests a stem cell transplant using a milder conditioning regimen—without standard chemotherapy—to treat people with aggressive sickle cell disease who cannot take or have failed hydroxyurea therapy. The transplant uses stem cells from a matched sibling donor, along with immune-suppressing drugs and low-dose radiation, aiming to reduce serious complications like strokes and severe pain episodes. The study focuses on whether the donor cells successfully engraft and how safe the procedure is in this high-risk group.

What this could mean

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

Active substance
Allogeneic non-myeloablative stem cell transplant with alemtuzumab, sirolimus, and low-dose total body irradiation
What this could lead to
If successful, this approach could offer a way to control sickle cell disease without the harsh side effects of traditional chemotherapy, potentially reducing complications like strokes and pain crises.
What could go wrong
This is an early-phase trial (Phase 1/2) with a small number of participants. The transplant still carries risks of graft failure, infection, or graft-versus-host disease, and it may not work for everyone.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • University of Illinois at Chicago

    Chicago, Illinois, 60612, United States

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