Gene therapy offers new hope for kids with brittle bone disease
NCT ID NCT07665021
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a new gene therapy for children with a severe inherited bone disease called osteopetrosis. The therapy uses the child's own blood stem cells, modified to fix the genetic defect, and then returned to the body. The goal is to improve blood cell production and survival, potentially avoiding the need for a bone marrow transplant. Eight children aged up to 2 years will be treated and followed for 2 years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- gene-modified stem cells (FT024)
- What this could lead to
- If successful, this could provide a safer treatment option for children with this severe bone disease, reducing the need for risky bone marrow transplants.
- What could go wrong
- This is an early-phase trial with only 8 children, so results are uncertain. The gene therapy may not work for everyone, and there are risks from the conditioning chemotherapy.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for AUTOSOMAL RECESSIVE OSTEOPETROSIS CAUSED BY MUTATIONS IN THE TCIRG1 GENE 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
Locations
-
Ospedale San Raffaele
RECRUITINGMilan, Italy, 20132, Italy
More trials for these conditions
Other studies related to the condition(s) this trial covers.