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

Multiple Sclerosis Clinical Trial

Official title:

Swiss Pediatric Inflammatory Bain Disease Cohort Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05017142
• Other study ID #: 2019-00377
• Status: Recruiting
• Start date: April 14, 2020
• Est. completion date: January 1, 2071

Study information

• Verified date: March 2023
• Source: University of Bern
• Contact: Lorena F Hulliger, MSc
• Phone: 0316845678
• Email: lorena.hulliger[@]ispm.unibe.ch
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: January 1, 2071
• Est. primary completion date: January 1, 2071
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Acute Disseminated Encephalomyelitis
• Anti-N-Methyl-D-Aspartate Receptor Encephalitis
• Anti-NMDAR Encephalitis
• CNS Sarcoidosis
• CNS Vasculitis
• Encephalitis
• Encephalomyelitis
• Hashimoto Disease
• Multiple Sclerosis
• Myelitis
• Myelitis, Transverse
• Neuritis
• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder
• Optic Neuritis
• Rasmussen Encephalitis
• Sarcoidosis
• Transverse Myelitis
• Vasculitis
• Vasculitis, Central Nervous System

Locations

Country	Name	City	State
Switzerland	Kantonsspital Aarau	Aarau	Aargau
Switzerland	University Children's Hospital Basel, UKBB	Basel
Switzerland	Pediatric Institute of Southern Switzerland, Ospedale San Giovanni	Bellinzona	Ticino
Switzerland	Institute of Social and Preventive Medicine, University of Bern	Bern
Switzerland	University Children's Hospital, Inselspital Bern	Bern
Switzerland	Kantonsspital Graubünden	Chur	Graubünden
Switzerland	University Hospitals of Geneva (HUG)	Geneva
Switzerland	University Children's Hospital Lausanne (CHUV)	Lausanne	Vaud
Switzerland	Luzerner Kantonsspital	Luzern
Switzerland	Children's Hospital of Eastern Switzerland	St.Gallen	Saint Gallen
Switzerland	Kinderspital Winterthur	Winterthur	Zurich
Switzerland	University Children's Hospital Zurich	Zurich

Sponsors (5)

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

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Personal data	Registering patient's personal data	At registration (Life-long; Up to 80 years)
Primary	Diagnosis	Diagnosis of IBrainD	Until reaching of adulthood (0 to 18 years)
Primary	Age at diagnosis	Age at diagnosis (months and years)	Until reaching of adulthood (0 to 18 years)
Primary	First symptoms	Symptoms before diagnosis	Until reaching of adulthood (0 to 18 years)
Primary	Age at first symptoms	Age at first symptoms	Until reaching of adulthood (0 to 18 years)
Primary	Diagnostic delay	Time elapsed between symptom-onset and diagnosis (days)	Until reaching of adulthood (0 to 18 years)
Primary	Hospitalization	Length of hospitalization at diagnosis or during a relapse (days)	Until reaching of adulthood (0 to 18 years)
Primary	Rehabilitation	Length and type of rehabilitation at diagnosis or during a relapse (days)	Until reaching of adulthood (0 to 18 years)
Primary	Death date	Date of death	Life-long; Up to 80 years
Primary	Death cause	Cause of death	Life-long; Up to 80 years
Primary	Change in EDSS	EDSS change over time	Until reaching of adulthood (0 to 18 years)
Primary	Change in Neurostatus	Neurostatus change over time	Until reaching of adulthood (0 to 18 years)
Primary	Change in medication	Change of IBrainD medication over time	Until reaching of adulthood (0 to 18 years)
Primary	Change in Education	Evolution of education over time	Until reaching of adulthood (0 to 18 years)
Primary	Change in MRI data	Change in number of CNS lesions	Until reaching of adulthood (0 to 18 years)
Primary	Change in MRI data	Change in activity of CNS lesions	Until reaching of adulthood (0 to 18 years)
Primary	Change in laboratory test data	Change in diagnostic markers	Until reaching of adulthood (0 to 18 years)
Primary	Electrophysiological testing	Assessment if the patient did undergo electrophysiological testing.	Until reaching of adulthood (0 to 18 years)
Secondary	Future questionnaires	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.	Life-long; Up to 80 years; Will mainly concern childhood (until reaching of adulthood; 0 to 18 years)

External Links

•   Website of the registry