RecruitingNot Applicable

TIRCON International NBIA Registry

Canada, Czechia, Germany2,000 participantsStarted 2012-06-14

Plain-language summary

TIRCON-reg aims to * continue the provision of a global registry and natural history study for NBIA disorders * harmonize and cover existing national and single site registries * enable participation of countries and single sites that so far have no access to an NBIA registry * join forces in order to recruit sufficient numbers of patients * define the natural history of NBIA disorders * define the most appropriate outcome measures * inform the design and facilitate the conduction of clinical trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * suspected or confirmed NBIA * willingness to participate Exclusion Criteria: * unwillingness to participate

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Score on the Barry-Albright Dystonia (BAD) Scale

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Change in Score on Unified Parkinson's Disease Rating (UPDRS) Scale, Part I-III, VI

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Change in Score on Pediatric Quality of Life (PedsQL)

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Disease progression

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Trial details

NCT IDNCT05522374

SponsorLMU Klinikum

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2040-12

Contact for this trial

Boriana Büchner, Dr.

+49 89 4400 boriana.buechner@med.uni-muenchen.de

View on ClinicalTrials.gov More Neurodegeneration With Brain Iron Accumulation (NBIA) trials

Plain-language summary

What they're measuring

Change in Score on the Barry-Albright Dystonia (BAD) Scale

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Change in Score on Unified Parkinson's Disease Rating (UPDRS) Scale, Part I-III, VI

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Change in Score on Pediatric Quality of Life (PedsQL)

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.

Disease progression

Timeframe: The individual participants are followed with annual assessments over a long time period (up to 30 years) or until discontinuation or death.