Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
2000 participants
OBSERVATIONAL
2012-06-14
2040-12-31
Brief Summary
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* 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
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Detailed Description
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Harmonization of existing data has been performed by establishing and applying matching and transformation rules. The web-based registry is now fully functional for a critically needed natural history study of all NBIA subtypes. A focus has been set on scores that are most appropriate to reflect stage and progression of disease, e.g. the Barry Albright Dystonia scale, the Patient´s Global Impression of Improvement (PGII), the Unified Parkinson Disease Rating Scale (UPDRS; parts I-III and VI) and quality-of-life scores. The natural history data are collected yearly, or in rapidly progressing cases every six months, if applicable. Patients who present to one of our centers are eligible after informed consent to participate.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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NBIA Patients
Patients with suspected or confirmed NBIA
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* willingness to participate
Exclusion Criteria
ALL
No
Sponsors
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Seventh Framework Programme
OTHER
NBIA Alliance
UNKNOWN
LMU Klinikum
OTHER
Responsible Party
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Prof. Thomas Klopstock
Prof. Dr. med.
Principal Investigators
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Thomas Klopstock, Prof. Dr.
Role: PRINCIPAL_INVESTIGATOR
LMU Klinikum, Friedrich-Baur-Institute
Locations
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Children's Hospital of Eastern Ontario, Division of Neurology, Department of Pediatrics
Ottawa, , Canada
Charles University, Department of neurology
Prague, , Czechia
LMU Klinikum, Friedrich-Baur-Institute
Munich, Bavaria, Germany
The Foundation of the Carlo Besta Neurological Institute, IRCCS
Milan, , Italy
University medical Center Groningen (UMCG) Department of Neurology AB 51
Groningen, , Netherlands
The Childrens Memorial Health Institute
Warsaw, , Poland
University of Belgrade, Department of Movement Disorders and Degenerative Brain Diseases
Belgrade, , Serbia
Hospital Vall d'Hebron - Institut de Recerca (VHIR), Pediatric Neurology, Movement Disorders
Barcelona, , Spain
Hospital Sant Joan de Déu, Universitat de Barcelona, Servei de Neurología
Barcelona, , Spain
Countries
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Central Contacts
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Facility Contacts
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Iva Stankovic, Dr.
Role: backup
References
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Kalman B, Lautenschlaeger R, Kohlmayer F, Buchner B, Kmiec T, Klopstock T, Kuhn KA. An international registry for neurodegeneration with brain iron accumulation. Orphanet J Rare Dis. 2012 Sep 17;7:66. doi: 10.1186/1750-1172-7-66.
Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele HE, Horvath R, Chinnery PF, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E. Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. Lancet Neurol. 2019 Jul;18(7):631-642. doi: 10.1016/S1474-4422(19)30142-5.
Karin I, Buchner B, Gauzy F, Klucken A, Klopstock T. Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON)-An International Network on Care and Research for Patients With Neurodegeneration With Brain Iron Accumulation (NBIA). Front Neurol. 2021 Feb 22;12:642228. doi: 10.3389/fneur.2021.642228. eCollection 2021.
Iankova V, Karin I, Klopstock T, Schneider SA. Emerging Disease-Modifying Therapies in Neurodegeneration With Brain Iron Accumulation (NBIA) Disorders. Front Neurol. 2021 Apr 15;12:629414. doi: 10.3389/fneur.2021.629414. eCollection 2021.
Related Links
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Project website
Other Identifiers
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TIRCON-reg
Identifier Type: -
Identifier Source: org_study_id
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