Study drug for mitochondrial depletion disorder due to gene changes
Part of Bones, joints & muscles, Brain & nervous system, Hormones & metabolism clinical trials.
This Phase 2 trial tests a treatment made from deoxynucleosides to help people with mitochondrial depletion disorder caused by certain gene changes. If you match the genetic diagnosis, the study may see whether the treatment improves symptoms or disease markers.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or a parent/legal guardian) can give consent to join the study
- You have a clinical diagnosis of mitochondrial depletion disorder
- Your genetic test shows a disease-causing change in one of these genes: POLG, POLG2, C10orf2, RRM2B, MPV17, SUCLA2, SUCLG1, FBXL4, or DTYMK
- If you can become pregnant, you must have a negative urine pregnancy test and agree to effective birth control during the study
- You cannot have chronic severe diarrhea
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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