A Multicenter Observational Study to Evaluate Pediatric Multiple Sclerosis in Brazil

Multiple Sclerosis Clinical Trial

— EMOCEMP  

Official title:

A Multicenter Observational Study to Evaluate Pediatric Multiple Sclerosis in Brazil

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03087136
• Other study ID #: 61080516.4.1001.5336
• Status: Active, not recruiting
• Start date: March 1, 2017
• Est. completion date: March 31, 2025

Study information

• Verified date: January 2024
• Source: Pontificia Universidade Católica do Rio Grande do Sul
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Pediatric Multiple sclerosis (MS) is a chronic inflammatory disease defined by multiple episodes of demyelination of the central nervous system (CNS) separated by time and space as specified in adults not explained by acute disseminated encephalomyelitis (ADEM). Several studies have indicated that at least 5% of MS patients are in the pediatric population, but no prospective study was performed in Brazil. There are particular characteristics of Pediatric MS that differs from the adult population and have been focus of interest in the last years. However, we still lack high evidence data, specially concerning treatment, of this age group.

This is an observational non-interventional multicenter study in pediatric MS patients in which participating subjects will be characterized by their clinical, MRI and immunological features. In this observational study, we will select 8 MS centers in Brazil to recruit at least 100 pediatric patients currently followed on each center with idiopathic inflammatory CNS disorders over a 2-year study period. We will collect retrospective and prospective clinical and MRI data to determine the proportion of patients who fulfill the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for MS,7 especially on those below 10 years where the use of McDonald 2010 criteria is not recommended routinely. Following the inclusion on the study, subjects will be followed for at least 2 years.

All subjects enrolled in this study will have serum collected to test autoantibodies including anti-AQP4 and anti-MOG using cell-based assays with transfected cells.8 Pediatric patients with positive testing for these autoantibodies will be analyzed separately.

Exploratory MRI sub-study In 10 patients recruited at Hospital São Lucas PUCRS, we will perform an exploratory substudy with advanced MRI using q-space diffusion protocol on a 3-Tesla MRI (GE Signa HDx 3.0T, General Electric, Milwaukee, WI, USA) and a 8-channel head coil to visualize remyelinating brain MS lesions. Normalized leptokurtic diffusion (NLD) data will be acquired using diffusion-weighted echo planar imaging. All MRI scans from this exploratory study will be performed at the Brain Institute of Rio Grande do Sul (BraIns). Only patients with previous brain demyelinating lesions will be included in this exploratory sub-study.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 100
• Est. completion date: March 31, 2025
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 18 Years

Eligibility

Inclusion Criteria:

1. Parents / patient have an understanding, ability and willingness to fully comply with study procedures

2. Parents / patient have the ability to provide voluntary written, signed and dated informed consent to participate in the study

3. Be 6 months - 18 years of age at screening

4. Have clinical evidence of at least 1 attack suggestive of idiopathic inflammatory CNS disorder

5. Have at least one brain MRI with available images (for review)

• For the exploratory MRI study, parents / patients have to agree to perform 2 MRI scans (at baseline and at the end of the study). Considering the age of the study subjects, sedation may be required during the acquisition of the MRI.

Exclusion Criteria:

1. Current evidence or known history of clinically significant infection including:

• Chronic or ongoing active infectious disease requiring long-term systemic treatment such as active hepatitis B or C, HIV or tuberculosis

2. Current malignancy or history of malignancy in the past 5 years

3. Significant concurrent, uncontrolled medical condition that could affect subject's safety or impair the subject's participation in the study.

4. Current participation in any interventional trial.

5. Pregnant or breastfeeding

Related Conditions & MeSH terms

• Multiple Sclerosis
• Sclerosis

Intervention

Diagnostic Test:
• anti-aquaporin-4 antibody
Serum anti-aquaporin-4 antibody
• anti-myelin oligodendrocyte glycoprotein
Serum anti-myelin oligodendrocyte glycoprotein

Locations

Country	Name	City	State
Brazil	Instituto de Neurologia de Curitiba	Curitiba	PR
Brazil	Pontifical Catholic University of Rio Grande do Sul	Porto Alegre	RS
Brazil	Santa Casa de Misericordia de Porto Alegre	Porto Alegre	RS
Brazil	Hospital da Restauração de Recife	Recife	PE
Brazil	Hospital das Clinicas da Faculdade de Medicina de Ribeirão Preto / Universidade de São Paulo	Ribeirao Preto	SP
Brazil	Universidade Federal do Estado do Rio de Janeiro	Rio de Janeiro	RJ
Brazil	Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo	Sao Paulo	SP
Brazil	Universidade Federal de Uberlândia	Uberlandia	MG

Sponsors (2)

Lead Sponsor	Collaborator
Pontificia Universidade Católica do Rio Grande do Sul	Teva Pharmaceuticals USA

Country where clinical trial is conducted

Brazil, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Convertion rate to MS	To evaluate the proportion of patients (below and above 10 years-old) with idiopathic inflammatory CNS disorders converting to pediatric MS in Brazilian MS referral centers.	2 years
Secondary	Clinical features	• To compare the clinical and MRI features between pediatric MS patients with disease onset < 10 and > 10 years	2 years
Secondary	epidemiology	• To analyze the influence of latitude, ethnicity and past viral infections on the development of pediatric MS	2 years
Secondary	autoantibodies	• To determine the proportion of patients with pediatric MS and other inflammatory demyelinating CNS disorders with autoantibodies (anti-AQP4 and anti-MOG)	2 years
Secondary	treatments	• To identify the disease-modifying treatments used in pediatric MS cases by the treating physician and the occurrence of adverse events	2 years