Experimental gene therapy aims to stop bone marrow failure in kids with rare disease
NCT ID NCT04248439
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal is to restore healthy blood cell production and prevent the need for a bone marrow transplant. Only 5 children are enrolled in this early study.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- RP-L102 (gene-corrected stem cells)
- What this could lead to
- If successful, this could provide a one-time treatment to prevent bone marrow failure in children with Fanconi anemia subtype A, reducing the need for bone marrow transplants.
- What could go wrong
- This is a very small, early-phase trial with only 5 participants. It may not work for everyone, and long-term effects are unknown. The procedure also carries risks from stem cell collection and infusion.
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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
-
Stanford University
Stanford, California, 94304, United States
-
University of Minnesota
Minneapolis, Minnesota, 55454, United States
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Other studies related to the condition(s) this trial covers.