Study of how MNGIE changes over time
Part of Brain & nervous system, Digestive system, Genetic & congenital clinical trials.
This study follows people with MNGIE (a rare mitochondrial disease) to better understand what happens over time and how lab markers relate to health. It does not test a new drug as part of the study.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have confirmed MNGIE related to a TYMP gene change or very low TP enzyme activity
- Your blood has higher-than-normal thymidine levels (Thd greater than 3 micromole/L)
- Your blood has higher-than-normal deoxyuridine levels (dUrd greater than 7.5 micromole/L)
- You are at least 5 years old
- You have not joined another medicine or experimental-intervention study in the last month
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 natural history study follows children and adults with suspected or confirmed mitochondrial myopathy to better understand symptoms and how the condition changes. It may also include healthy participants to compare test results and improve future treatments.
This study collects tissue samples to help researchers learn more about mitochondrial disorders—conditions related to how the body makes energy. It may help by improving genetic understanding for families who suspect an inherited cause.
This trial tests whether a supplement called NMN is safe and well-tolerated in children and adults with a rare genetic condition called DHDDS-CDG. NMN may help support cellular energy and protein production, which are affected by this condition.
This study looks at brain chemicals in people with primary mitochondrial disease using an MRI scan. It aims to understand how the disease affects the brain and may help guide future treatments.
This Phase 1 study tests a special MRI approach (using a contrast agent) to better measure changes in muscles in people with mitochondrial diseases. It also includes some healthy volunteers and other critically ill patients to compare results and check safety.
This study collects information from patients with MNGIE (a rare genetic condition affecting how cells break down certain molecules) to better understand how the disease develops and progresses over time. By learning more about your experience, researchers hope to develop better treatments.
Hear when a new Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.