mRNA treatment for propionic acidemia, tested in a study
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests an mRNA medicine (mRNA-3927) in people with propionic acidemia (PA). It aims to see whether this treatment is safe and can help the body when PA is confirmed by genetic testing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of propionic acidemia from genetic testing (PA gene changes).
- If you’re under 1 year old, you must meet extra rules like newborn screening/suspected PA while testing, and have enough stability and weight at screening.
- If you’re in Part 2, you had at least one “metabolic decompensation event” in the last 12 months (an episode when PA symptoms or labs got significantly worse).
- You must not have serious heart rhythm risk, such as a QTc interval over 480 ms on heart testing.
- You must not have major heart failure (Grade 3–4 in children, or NYHA class III/IV).
- You must not be pregnant or breastfeeding.
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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