Hydroxocobalamin for children with a specific form of MMA
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests whether a vitamin B12 treatment (hydroxocobalamin) can help children with a specific type of methylmalonic acidemia (cblC type). It aims to see if the treatment is effective and safe for those who have already shown benefit from vitamin B12 therapy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 6 months and less than 18 years old.
- You have been diagnosed with cblC-type methylmalonic acidemia and have confirmed vitamin B12 responsiveness.
- You have been stable, with no hospital stays for metabolic crises in the last 6 months and normal blood MMA levels.
- You have been getting hydroxocobalamin injections for at least 3 months.
- You are willing to use effective birth control if applicable.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study is for children up to 16 years old with a severe form of methylmalonic acidemia (MMA) caused by a specific gene change (MMUT). It aims to better understand the disease and may lead to future treatments. Both children who have had a liver transplant and those who have not can join, as long as certain criteria are met.
This research study follows patients with methylmalonic acidemia (a rare metabolic disorder affecting how the body breaks down certain proteins) or cobalamin (vitamin B12) disorders to better understand how these conditions develop and progress over time. Researchers will collect medical information, blood samples, and eye exams to help improve future treatments.
This study tests whether early infant nutrition using specific micronutrients (small amounts of key vitamins and nutrients) can support healthy growth and development. It includes babies whose moms plan to breastfeed for long enough, and it excludes infants with serious health problems.
This observational study looks at how Carbaglu® is used and how well it works for people with methylmalonic acidemia (MMA) or propionic acidemia (PA). It may help doctors understand real-world care for adults and children living with these conditions.
This trial tests a new medicine, sonlicromanol, for people with a specific type of mitochondrial disease (caused by the m.3243A>G mutation) who also have chronic fatigue and muscle weakness. The goal is to see if it helps improve symptoms and daily function.
This trial tests a gene therapy given by a one-time injection into the spinal fluid for children with Mucolipidosis type IV. It aims to see if the therapy is safe and can help slow or stop the disease.
Hear when a new Methylmalonic Acidemia (MMA) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.