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.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for BETA-THALASSEMIA are added.
By submitting, you agree to our Terms of use
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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States