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Swiss Pediatric Inflammatory Brain Disease Registry (Swiss-Ped-IBrainD)

Sponsor
University of Bern (Other)
Overall Status
Recruiting
CT.gov ID
NCT05017142
Collaborator
Schweizerische Multiple Sklerose Gesellschaft (Other), University Hospital Inselspital, Berne (Other), Roche Pharma (Switzerland) Ltd (Other), Novartis (Industry)
500
Enrollment
12
Locations
608.6
Anticipated Duration (Months)
41.7
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The Swiss-Ped-IBrainD is a national patient registry that collects information on diagnosis, symptoms, treatment, and follow-up of pediatric patients with an inflammatory brain disease in Switzerland. It was first implemented in 2020 in the pediatric clinic of the university hospital in Bern. Further centers all over Switzerland were opened for recruitment in 2021; Aarau, Basel, Bellinzona, Chur, Geneva, Lausanne, Lucerne, St. Gallen, and Zurich. The center in Winterthur is expected to be open for recruitment by autumn 2021. The registry provides data for national and international monitoring and research. It supports research on inflammatory brain diseases in Switzerland and the exchange of knowledge between clinicians, researchers, and therapists. The registry aims to improve the treatment of children with inflammatory brain diseases and optimizing their health care and quality of life.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    Background:

    Pediatric onset MS and other inflammatory brain diseases (IBrainDs) are severe diseases affecting children and adolescents in a period of essential brain development. This possibly leads to a variety of focal neurological deficits as well as early cognitive impairment. In turn, the cognitive impairment may impact school performance and vocational achievements.

    Timely diagnosis and treatment initiation as well as individually tailored management are important for a favorable disease course. However, the diagnosis of the different IBrainDs can be challenging, especially in young children, since their first acute inflammation is often accompanied by unspecific symptoms common to all IBrainDs. A systematic assessment of similarities and differences between clinical signs, symptoms, and diagnostic workup of different IBrainDs will enable faster and more reliable diagnosis.

    Furthermore, neither epidemiological data nor information on health care management and disease outcome of pediatric IBrainD patients exist in Switzerland. Therefore, a national registry is being established, which will allow a deeper understanding of pediatric IBrainD epidemiology, clinical presentation, and management. Ultimately, the registry will improve the care of children suffering from an IBrainD in Switzerland.

    The Swiss-Ped-IBrainD Registry (title: "Swiss Pediatric Inflammatory Brain Disease Cohort Study", project number: 2019-00377) has been approved by the ethics committees of Bern, the Ethikkommission Nordwest- und Zentralschweiz (EKNZ), the Ethikkommission Ostschweiz (EKOS), and the ethics committees of Zürich, Lausanne, Geneva, and Bellinzona.

    Objectives:
    The registry pursues the following goals:

    1. Gathering representative, population-based epidemiological data on pediatric IBrainD in Switzerland.

    2. Monitoring treatment, clinical course, education, social aspects, and outcomes of pediatric IBrainD patients.

    3. Providing a platform to facilitate research, national and international collaboration and exchange of knowledge between experts.

    The registry thus addresses the increasing requests for medical trial participation and promotes the exchange with existing adult registries (e.g., Swiss MS Registry).

    Inclusion/exclusion criteria:

    All patients living and/or treated in Switzerland with an IBrainD specified in the following list with a disease onset before the age of 18.

    • Optic neuritis

    • Transverse myelitis

    • Acute disseminated encephalomyelitis

    • Multiple sclerosis

    • Neuromyelitis optica spectrum disorders

    • Anti-NMDA-R associated autoimmune encephalitis

    • Anti-GAD65 associated autoimmune encephalitis

    • Anti-AMPAR-1/2 associated autoimmune encephalitis

    • Anti-Lgi-1 associated autoimmune encephalitis

    • Anti-CASPR-2 associated autoimmune encephalitis

    • Anti-GABAR-1/2 associated autoimmune encephalitis

    • Onconeuronal antibody (Hu, Ri, Yo, Amphiphysin, CRMP-5, Ma-1, Ma-2, SOX-1) associated autoimmune encephalitis

    • Hashimoto encephalopathy

    • CNS vasculitis

    • CNS sarcoidosis

    • CNS Lupus

    • Rasmussen's encephalitis

    Excluded are patients with:

    1. Neurological symptoms due to infectious diseases of the CNS

    2. Genetic/metabolic causes of central demyelinating diseases

    3. Neurological symptoms due to Guillain-Barré-Syndrome

    Registration of Patients and Collection of Medical Data:

    Pediatricians, pediatric neurologists, neurologists, specialists in rehabilitation, and primary care physicians at the participating centers are responsible to identify children with the listed IBrainDs during regular medical consultations. Upon identification, treating physicians inform patients and their parents orally and in writing about the Swiss-Ped-IBrainD. Patients (and their legal representatives if applicable) who want to participate must give their informed consent. Once a patient consents to participate, his/her medical data will be entered in the registry.

    The diagnostic workup and treatment of patients continue as usual and are independent from participation; no examination will be carried out specifically for the Swiss-Ped-IBrainD.

    Medical data is collected through the following sources:

    • Medical records and reports

    • Oral/written information from treating physician

    • Oral/written information from patient/family

    • Routine statistics and other medical registries

    • Questionnaires for patients and families The data collection focuses on diagnostic, follow-up, and relapse variables.

    Routine data and linkages:

    Communities; Federal Statistical Office (e.g. the birth register, cause of death statistics, hospital statistics)

    Current status:

    From 2020-2021, the investigators have included 17 persons diagnosed with an IBrainD.

    Funding:

    • Schweizerische Multiple Sklerose Gesellschaft

    • PedNet Bern

    • Roche Pharma (Switzerland) Ltd

    • Novartis Pharma Schweiz AG

    • SwissPedRegistry, University of Bern

    Study Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    500 participants
    Observational Model:
    Cohort
    Time Perspective:
    Other
    Official Title:
    Swiss Pediatric Inflammatory Bain Disease Cohort Study
    Actual Study Start Date :
    Apr 14, 2020
    Anticipated Primary Completion Date :
    Jan 1, 2071
    Anticipated Study Completion Date :
    Jan 1, 2071

    Arms and Interventions

    Arm Intervention/Treatment
    Patient population

    Children, adolescents and adults diagnosed with an IBrainD before age 18, who are born, treated or living in Switzerland

    Outcome Measures

    Primary Outcome Measures

    1. Personal data [At registration (Life-long; Up to 80 years)]

      Registering patient's personal data

    2. Diagnosis [Until reaching of adulthood (0 to 18 years)]

      Diagnosis of IBrainD

    3. Age at diagnosis [Until reaching of adulthood (0 to 18 years)]

      Age at diagnosis (months and years)

    4. First symptoms [Until reaching of adulthood (0 to 18 years)]

      Symptoms before diagnosis

    5. Age at first symptoms [Until reaching of adulthood (0 to 18 years)]

      Age at first symptoms

    6. Diagnostic delay [Until reaching of adulthood (0 to 18 years)]

      Time elapsed between symptom-onset and diagnosis (days)

    7. Hospitalization [Until reaching of adulthood (0 to 18 years)]

      Length of hospitalization at diagnosis or during a relapse (days)

    8. Rehabilitation [Until reaching of adulthood (0 to 18 years)]

      Length and type of rehabilitation at diagnosis or during a relapse (days)

    9. Death date [Life-long; Up to 80 years]

      Date of death

    10. Death cause [Life-long; Up to 80 years]

      Cause of death

    11. Change in EDSS [Until reaching of adulthood (0 to 18 years)]

      EDSS change over time

    12. Change in Neurostatus [Until reaching of adulthood (0 to 18 years)]

      Neurostatus change over time

    13. Change in medication [Until reaching of adulthood (0 to 18 years)]

      Change of IBrainD medication over time

    14. Change in Education [Until reaching of adulthood (0 to 18 years)]

      Evolution of education over time

    15. Change in MRI data [Until reaching of adulthood (0 to 18 years)]

      Change in number of CNS lesions

    16. Change in MRI data [Until reaching of adulthood (0 to 18 years)]

      Change in activity of CNS lesions

    17. Change in laboratory test data [Until reaching of adulthood (0 to 18 years)]

      Change in diagnostic markers

    18. Electrophysiological testing [Until reaching of adulthood (0 to 18 years)]

      Assessment if the patient did undergo electrophysiological testing.

    Secondary Outcome Measures

    1. Future questionnaires [Life-long; Up to 80 years; Will mainly concern childhood (until reaching of adulthood; 0 to 18 years)]

      Data from validated instrument such as the Pediatric Quality of Life Inventory (PedsQL); Scale from 0-100, where 100 is the best possible outcome and 0 the worst possible outcome.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    All patients living and/or treated in Switzerland with an IBrainD specified in the following list with a disease onset before the age of 18.

    • Written informed consent by patients (and/or legal representative(s), if applicable)

    • Optic Neuritis

    • Transverse Myelitis

    • Acute disseminated encephalomyelitis

    • Multiple Sclerosis

    • Neuromyelitis Optica Spectrum Disorders

    • Anti-NMDA-R Encephalitis

    • Anti-GAD65 Associated Autoimmune Encephalitis

    • Anti-AMPAR-1/2 Associated Autoimmune Encephalitis

    • Anti-Lgi-1 Associated Autoimmune Encephalitis

    • Anti-CASPR-2 Associated Autoimmune Encephalitis

    • Anti-GABAR-1/2 Associated Autoimmune Encephalitis

    • Onconeuronal Antibody (Hu, Ri, Yo, Amphiphysin, CRMP-5, Ma-1, Ma-2, SOX-1) Associated Autoimmune Encephalitis

    • Hashimoto Encephalopathy

    • CNS Vasculitis

    • CNS Sarcoidosis

    • CNS Lupus

    • Rasmussen Encephalitis

    Exclusion Criteria:

    • Neurological symptoms due to infectious diseases of the CNS

    • Genetic/metabolic causes of central demyelinating diseases

    • Neurological symptoms due to Guillain-Barré-Syndrome

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Kantonsspital Aarau Aarau Aargau Switzerland 5001
    2 Kantonsspital Graubünden Chur Graubünden Switzerland 7000
    3 Children's Hospital of Eastern Switzerland St.Gallen Saint Gallen Switzerland 9000
    4 Pediatric Institute of Southern Switzerland, Ospedale San Giovanni Bellinzona Ticino Switzerland 6500
    5 University Children's Hospital Lausanne (CHUV) Lausanne Vaud Switzerland 1011
    6 Kinderspital Winterthur Winterthur Zurich Switzerland 8400
    7 University Children's Hospital Basel, UKBB Basel Switzerland 4031
    8 University Children's Hospital, Inselspital Bern Bern Switzerland 3010
    9 Institute of Social and Preventive Medicine, University of Bern Bern Switzerland 3012
    10 University Hospitals of Geneva (HUG) Geneva Switzerland 1211
    11 Luzerner Kantonsspital Luzern Switzerland 6000
    12 University Children's Hospital Zurich Zurich Switzerland 8032

    Sponsors and Collaborators

    • University of Bern
    • Schweizerische Multiple Sklerose Gesellschaft
    • University Hospital Inselspital, Berne
    • Roche Pharma (Switzerland) Ltd
    • Novartis

    Investigators

    • Principal Investigator: Sandra Bigi, PD MD, ISPM, University of Bern, Bern; University Children's Hospital, University of Bern, Bern
    • Study Director: Sandra Bigi, PD MD, ISPM, University of Bern, Bern; University Children's Hospital, University of Bern, Bern
    • Study Director: Claudia E Kuehni, Prof. MD, ISPM, University of Bern, Bern

    Study Documents (Full-Text)

    None provided.

    More Information

    Additional Information:

    Publications

    None provided.
    Responsible Party:
    University of Bern
    ClinicalTrials.gov Identifier:
    NCT05017142
    Other Study ID Numbers:
    • 2019-00377
    First Posted:
    Aug 23, 2021
    Last Update Posted:
    Aug 23, 2021
    Last Verified:
    Aug 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Encephalomyelitis
    Myelitis
    Myelitis, Transverse
    Anti-N-Methyl-D-Aspartate Receptor Encephalitis
    Multiple Sclerosis
    Encephalitis
    Neuritis
    Neuromyelitis Optica
    Optic Neuritis
    Encephalomyelitis, Acute Disseminated
    Vasculitis, Central Nervous System
    Vasculitis
    Sarcoidosis
    Hashimoto Disease

    Study Results

    No Results Posted as of Aug 23, 2021