Global registry for neurodegeneration with brain iron accumulation disorders
TIRCON International NBIA (Neurodegeneration Associated With Brain Iron Accumulation) Patient Registry and Natural History Study
This study is creating a global registry to help people with neurodegeneration disorders linked to brain iron buildup by tracking their conditions and improving future treatments.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2000 (estimated) |
| Sex | All |
| Sponsor | LMU Klinikum Academic / other |
| Locations | 9 sites (Ottawa and 8 other locations) |
| Trial ID | NCT05522374 on ClinicalTrials.gov |
What this trial studies
The TIRCON International NBIA Registry aims to create a comprehensive global registry and natural history study for various neurodegeneration disorders associated with brain iron accumulation. It seeks to harmonize existing national and single-site registries, allowing participation from countries and sites without prior access to an NBIA registry. The study focuses on defining the natural history of these disorders and identifying appropriate outcome measures to inform future clinical trials. Data is collected annually or biannually for rapidly progressing cases, ensuring a robust understanding of disease progression.
Who should consider this trial
Good fit: Ideal candidates for this registry are individuals with suspected or confirmed NBIA disorders who are willing to participate.
Not a fit: Patients who are unwilling to participate in the registry will not benefit from this initiative.
Why it matters
Potential benefit: If successful, this registry could significantly enhance the understanding and management of NBIA disorders, leading to improved patient outcomes.
How similar studies have performed: Other similar registries have shown success in enhancing understanding and treatment approaches for rare neurodegenerative disorders, indicating a promising potential for this initiative.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * suspected or confirmed NBIA * willingness to participate Exclusion Criteria: * unwillingness to participate
Where this trial is running
Ottawa and 8 other locations
- Children's Hospital of Eastern Ontario, Division of Neurology, Department of Pediatrics — Ottawa, Canada (Recruiting)
- Charles University, Department of neurology — Prague, Czechia (Recruiting)
- LMU Klinikum, Friedrich-Baur-Institute — Munich, Bavaria, Germany (Recruiting)
- The Foundation of the Carlo Besta Neurological Institute, IRCCS — Milan, Italy (Recruiting)
- University medical Center Groningen (UMCG) Department of Neurology AB 51 — Groningen, Netherlands (Recruiting)
- The Childrens Memorial Health Institute — Warsaw, Poland (Recruiting)
- University of Belgrade, Department of Movement Disorders and Degenerative Brain Diseases — Belgrade, Serbia (Recruiting)
- Hospital Vall d'Hebron - Institut de Recerca (VHIR), Pediatric Neurology, Movement Disorders — Barcelona, Spain (Active_not_recruiting)
- Hospital Sant Joan de Déu, Universitat de Barcelona, Servei de Neurología — Barcelona, Spain (Active_not_recruiting)
Study contacts
- Principal investigator: Thomas Klopstock, Prof. Dr. — LMU Klinikum, Friedrich-Baur-Institute
- 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.