Stem cell trial for severe brittle bone in young children
Part of Bones, joints & muscles, Genetic & congenital, Skin clinical trials.
This trial tests a cell-based treatment to improve bone strength in children with the most severe type of osteogenesis imperfecta (brittle bone disease). If you qualify, it may help slow bone weakening and support growth.
Summary written for real people, not researchers, by Clin2.
Who can take part
- A parent or legal guardian is willing to sign consent forms
- Your child is older than 3 years and younger than 10 years at enrollment
- Genetic testing shows an COL1A1 or COL1A2 gene mutation linked to severe type 3 OI
- Your child has not started puberty yet (or results suggest they are still pre-puberty)
- Your child has been on IV pamidronate treatment for at least 1 year before joining
- Blood and health tests must be safe (no vitamin D deficiency despite treatment, and no major blood-count problems or liver test elevations)
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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