Stem cell therapy for severe osteogenesis imperfecta
Part of Bones, joints & muscles, Genetic & congenital, Skin clinical trials.
This trial tests whether mesenchymal stem cells (special cells that can become bone) can help children with severe osteogenesis imperfecta (brittle bone disease) by improving bone strength and growth. It is for children aged 3 to 9 who have a confirmed gene change and have already been treated with pamidronate for at least a year.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child must be between 3 and 9 years old.
- Child must have a confirmed gene change in COL1A1 or COL1A2.
- Child must have severe type 3 osteogenesis imperfecta (brittle bone disease).
- Child must not have started puberty.
- Child must have received intravenous pamidronate therapy for at least one year before the study.
- Child must not have other medical conditions like heart disease, diabetes, or infections.
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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