Gene therapy offers new hope for kids with brittle bone disease

NCT ID NCT07665021

Recruiting now Disease control Sponsor: Fondazione Telethon Source: ClinicalTrials.gov ↗

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.

Disclaimer Read more

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.

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