Hydroxocobalamin treatment for methylmalonic acidemia in children
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study tests whether a new form of hydroxocobalamin (a vitamin B12 treatment) can safely control methylmalonic acidemia (MMA) in children and teens. It aims to see if this treatment can help prevent metabolic crises and keep the condition stable.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age 6 months to under 18 years
- Diagnosed with cobalamin C (cblC) methylmalonic acidemia that responds to vitamin B12
- Clinically stable — no hospital stays for metabolic crisis in the past 6 months
- On injectable hydroxocobalamin (vitamin B12) for at least 3 months before starting
- Meet standard lab values (blood counts and kidney function) — will be checked by the study team
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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