DCA treatment for people with PDCD
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial provides access to an investigational drug called DCA (dichloroacetate) for people with pyruvate dehydrogenase complex deficiency, a rare genetic condition that affects energy production in the body. The goal is to offer a possible treatment option for those who have few other choices.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You may be any age, from newborn to adult.
- You must have a confirmed diagnosis of pyruvate dehydrogenase complex deficiency (PDCD), based on clinical signs or metabolic features.
- A genetic test must show a disease-causing mutation in one of the specific genes associated with PDCD (PDHA1, PDHB, DLAT, PDHX, or DLD).
- If you are a female of childbearing age, you must agree to use effective contraception during the study.
- You cannot have other mitochondrial diseases, certain metabolic disorders, severe kidney problems, or other medical conditions that would be unsafe, and you cannot 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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