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MNGIE Natural History Study

Recruitingn/aAges any - anyAllUniversity of Cambridge
NCT07627217 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

The MNGIE Retrospective Natural History Study is a collaborative study between the University of Cambridge and the University of Bologna. The aim of this study is to better understand the natural history and progression of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE). New treatment strategies for MNGIE, including gene therapies, enzyme replacement therapy, and other advanced treatments, are currently being developed and may soon be tested in clinical trials. A comprehensive and up-to-date natural history study of MNGIE is therefore very important to help inform the design of these clinical trials and to identify appropriate clinical and biochemical outcome measure. This international natural history study aims to include as many patients with MNGIE (living or deceased) as possible, worldwide. This study will collect anonymised clinical information through a secure onli

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📋 Eligibility rules (as published)
Inclusion Criteria: All patients with a laboratory-confirmed TP deficiency: * any age or stage of disease; living or deceased * both previously published and unpublished patients * symptomatic and asymptomatic patients * TP deficiency defined by a and/or b and/or c: 1. Homozygous or compound heterozygous pathogenic or likely pathogenic mutations in the TYMP gene; and/or 2. Decreased TP enzyme activity \<20% of normal; and/or 3. Increased plasma dThd\> 1 µmol/L, or increased plasma dUrd \> 5 µmol/L. Exclusion Criteria: * There are no formal exclusion criteria for this retrospective observational study.
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What it tests

  • Not listed
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Where

1 site
Department of Clinical Neurosciences
Cambridge, United Kingdom
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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