Global Registry and Natural History Study for Mitochondrial Disorders

LMU Klinikum

Status: Enrolling

Conditions: LHON; Leigh Syndrome; Pearson Syndrome; Barth Syndrome; SCAE; MNGIE; Kearns-Sayre Syndrome; Mitochondrial Diseases; MIRAS; MDS; MELAS Syndrome; Mitochondrial Myopathies; MERRF Syndrome; CPEO; SANDO; NARP Syndrome; MIDD; Coenzyme Q10 Deficiency

Study type: Observational

Funder types: Other

Identifiers

NCT05554835

mitoGLOBAL

Details and patient eligibility

About

The main goal of the project is provision of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.

Full description

The global mitochondrial registry and natural history study is part of the EU-financed GENOMIT project, co-ordinated by Dr. Holger Prokisch, Technische Universität München (TUM).It aims at advancing the understanding of the natural history of mitochondrial disease to inform the design and facilitate the conduction of clinical trials. It also serves as a catalyst for translating basic research results into clinical practice.

The global mitochondrial registry and natural history study provides for all contingencies of national ethics and data protection rules including data access management.

Currently participating networks are:

• German network for mitochondrial diseases - mitoNET, Germany/Austria
• Italian Registry of Mitochondrial Patients - Mitocon, Italy

The inclusion of other networks and countries is possible and explicitly welcome. A major advantage of the global registry is that countries can join in, saving a lot of time, effort and funding.

Enrollment: 6,000 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• suspected or confirmed mitochondrial disease
• willingness to participate

Exclusion criteria

• unwillingness to participate

Trial design

6,000 participants in 1 patient group

Mitochondrial patients

Description:

Patients with a suspected or confirmed mitochondrial disease.