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JOURNAL ONKOLOGIE – STUDIE

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

Rekrutierend

NCT-Nummer:
NCT05017142

Studienbeginn:
April 2020

Letztes Update:
14.03.2023

Wirkstoff:
-

Indikation (Clinical Trials):
Myelitis, Transverse, Anti-N-Methyl-D-Aspartate Receptor Encephalitis, Multiple Sclerosis, Encephalitis, Neuritis, Optic Neuritis, Neuromyelitis Optica, Encephalomyelitis, Myelitis, Vasculitis, Central Nervous System, Encephalomyelitis, Acute Disseminated, Vasculitis, Sarcoidosis, Hashimoto Disease

Geschlecht:
Alle

Altersgruppe:
Alle

Phase:
-

Sponsor:
University of Bern

Collaborator:
Schweizerische Multiple Sklerose Gesellschaft, University Hospital Inselspital, Berne, Roche Pharma (Switzerland) Ltd, Novartis,

Studienleiter

Sandra Bigi, PD MD
Principal Investigator
ISPM, University of Bern, Bern; Luzerner Kantosnspital, Kinderspital, Luzern
Sandra Bigi, PD MD
Study Director
ISPM, University of Bern, Bern; Luzerner Kantonsspital, Kinderspital, Luzern
Claudia E Kuehni, Prof. MD
Study Director
ISPM, University of Bern, Bern

Kontakt

Lorena F Hulliger, MSc
Kontakt:
Phone: 0316845678
Phone (ext.): +41
E-Mail: lorena.hulliger@ispm.unibe.ch» Kontaktdaten anzeigen

Studienlocations
(3 von 12)

Alle anzeigen

Studien-Informationen

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

Ein-/Ausschlusskriterien

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

Studien-Rationale

Primary outcome:

1. Personal data (Time Frame - At registration (Life-long; Up to 80 years)):
Registering patient's personal data

2. Diagnosis (Time Frame - Until reaching of adulthood (0 to 18 years)):
Diagnosis of IBrainD

3. Age at diagnosis (Time Frame - Until reaching of adulthood (0 to 18 years)):
Age at diagnosis (months and years)

4. First symptoms (Time Frame - Until reaching of adulthood (0 to 18 years)):
Symptoms before diagnosis

5. Age at first symptoms (Time Frame - Until reaching of adulthood (0 to 18 years)):
Age at first symptoms

6. Diagnostic delay (Time Frame - Until reaching of adulthood (0 to 18 years)):
Time elapsed between symptom-onset and diagnosis (days)

7. Hospitalization (Time Frame - Until reaching of adulthood (0 to 18 years)):
Length of hospitalization at diagnosis or during a relapse (days)

8. Rehabilitation (Time Frame - Until reaching of adulthood (0 to 18 years)):
Length and type of rehabilitation at diagnosis or during a relapse (days)

9. Death date (Time Frame - Life-long; Up to 80 years):
Date of death

10. Death cause (Time Frame - Life-long; Up to 80 years):
Cause of death

11. Change in EDSS (Time Frame - Until reaching of adulthood (0 to 18 years)):
EDSS change over time

12. Change in Neurostatus (Time Frame - Until reaching of adulthood (0 to 18 years)):
Neurostatus change over time

13. Change in medication (Time Frame - Until reaching of adulthood (0 to 18 years)):
Change of IBrainD medication over time

14. Change in Education (Time Frame - Until reaching of adulthood (0 to 18 years)):
Evolution of education over time

15. Change in MRI data (Time Frame - Until reaching of adulthood (0 to 18 years)):
Change in number of CNS lesions

16. Change in MRI data (Time Frame - Until reaching of adulthood (0 to 18 years)):
Change in activity of CNS lesions

17. Change in laboratory test data (Time Frame - Until reaching of adulthood (0 to 18 years)):
Change in diagnostic markers

18. Electrophysiological testing (Time Frame - Until reaching of adulthood (0 to 18 years)):
Assessment if the patient did undergo electrophysiological testing.

Secondary outcome:

1. Future questionnaires (Time Frame - 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.

Quelle: ClinicalTrials.gov


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