Global registry for mitochondrial disorders
Global Mitochondrial Registry to Define Natural History and Outcome Measures to Achieve Definite Trial Readiness for Mitochondrial Disorders
This study is creating a global database for mitochondrial disorders to help understand these diseases better and improve future treatments for patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 6000 (estimated) |
| Sex | All |
| Sponsor | LMU Klinikum Academic / other |
| Locations | 18 sites (Innsbruck and 17 other locations) |
| Trial ID | NCT05554835 on ClinicalTrials.gov |
What this trial studies
This project aims to create a comprehensive global registry for mitochondrial disorders, harmonizing existing national registries to enable worldwide participation. It focuses on understanding the natural history of mitochondrial diseases, which will inform the design of clinical trials and the definition of outcome measures. The study is part of the EU-financed GENOMIT project, coordinated by Dr. Holger Prokisch at Technische Universität München. It also ensures compliance with national ethics and data protection regulations while encouraging the inclusion of additional networks and countries.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with suspected or confirmed mitochondrial diseases who are willing to participate.
Not a fit: Patients who are unwilling to participate in the registry will not benefit from this study.
Why it matters
Potential benefit: If successful, this registry could significantly enhance the understanding and treatment of mitochondrial disorders, leading to improved patient outcomes.
How similar studies have performed: Other studies have successfully utilized registry approaches to advance understanding and treatment of rare diseases, making this a promising initiative.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * suspected or confirmed mitochondrial disease * willingness to participate Exclusion Criteria: * unwillingness to participate
Where this trial is running
Innsbruck and 17 other locations
- Medical University Innsbruck, Department of Pediatrics — Innsbruck, Austria (Recruiting)
- Salzburger Landeskliniken, SALK, Paracelsus Medizinische Privatuniversität — Salzburg, Austria (Recruiting)
- Department of neurology, Klinikum rechts der Isar, Technical University Munich — Munich, Bavaria, Germany (Recruiting)
- Charité Virchow Klinikum, Klinik für Pädiatrie m. S. Neurologie — Berlin, Germany (Recruiting)
- Universität Bonn, Klinik und Poliklinik für Neurologie — Bonn, Germany (Recruiting)
- Universitätsklinikum Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin — Cologne, Germany (Recruiting)
- Universitätsklinikum Düsseldorf, Klinik für allgemeine Pädiatrie, Neonatologie und Kinderkardiologie — Düsseldorf, Germany (Recruiting)
- Universitätsklinikum Frankfurt, Klinik für Kinder- und Jugendmedizin, Schwerpunkt Neurologie, Neurometabolik und Prävention — Frankfurt am Main, Germany (Recruiting)
- University Medical Center Freiburg, Center for children and youth medicine — Freiburg im Breisgau, Germany (Recruiting)
- Martin-Luther-Universität Halle-Wittenberg, Neurologische Klinik und Poliklinik — Halle, Germany (Recruiting)
- Universitätsklinikum Hamburg Eppendorf Institut für Humangenetik — Hamburg, Germany (Recruiting)
- Universitätsklinikum Hamburg Eppendorf, Klinik für Kinder-und Jugendmedizin — Hamburg, Germany (Recruiting)
- Universitätsklinikum Hamburg Eppendorf, Klinik für Neurologie — Hamburg, Germany (Recruiting)
- Universitätsklinikum Heidelberg, Zentrum für Kinder- und Jugendmedizin, Sektion für Neuropädiatrie und Stoffwechselmedizin — Heidelberg, Germany (Recruiting)
- LMU Klinikum, Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik — München, Germany (Recruiting)
- Klinikum am Steinenberg, Kreiskliniken Reutlingen, Klinik für Kinder-und Jugendmedizin, Perinatal- u. Stoffwechselzentrum — Reutlingen, Germany (Recruiting)
- Universitätsklinikum Tübingen, Neurologische Klinik und Hertie Institut für Klinische Hirnforschung — Tübingen, Germany (Recruiting)
- Department of Clinical and Experimental Medicine, Neurological Institute, University of Pisa & AOUP — Pisa, Italy (Recruiting)
Study contacts
- Principal investigator: Thomas Klopstock, Prof. Dr. — LMU Klinikum, Munich
- Study coordinator: Boriana Büchner, Dr.
- Email: boriana.buechner@med.uni-muenchen.de
- Phone: +49 89 4400
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.