New antibody could boost stem cell transplant success for blood disorders
NCT ID NCT05357482
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether adding an antibody called briquilimab (JSP191) to a standard stem cell transplant can help more donor cells take root in people with sickle cell disease or beta-thalassemia. About 40 patients aged 13 and older will receive the antibody along with low-dose radiation and immune-suppressing drugs before getting donor stem cells. The goal is to see if this combination leads to high levels of donor cells in the blood one year after transplant, which could mean a safer and more effective treatment.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- briquilimab (JSP191), a monoclonal antibody that targets CD117 on stem cells
- What this could lead to
- If successful, this approach could make stem cell transplants safer and more effective for people with sickle cell disease or beta-thalassemia, potentially offering a functional cure without the need for lifelong medication.
- What could go wrong
- This is an early-phase trial with only 40 participants, so results may not apply to everyone. There are risks from the transplant process itself, including graft failure, infection, and side effects from immune-suppressing drugs.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New sickle cell drug candidate enters first human tests
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- New stem cell transplant aims to ease severe blood disorders