TyPed - Natalizumab for the treatment of pediatric-onset multiple sclerosis in Portugal

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  NATALIZUMAB
• Medical condition to be studied:
  Multiple sclerosis

Population studied

• Short description of the study population:
  Patients diagnosed with Pediatric-onset multiple sclerosis (POMS) who have been treated for > 12 months with natalizumab in Portugal.; ; To be eligible to participate in this study, candidates must meet the following eligibility criteria:; 1. Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local privacy regulations or, if at inclusion date patient is less than 18 years old, signed informed consent obtained from patient’s legal representative (parent or guardian).; 2. Diagnosis of MS as defined by the International Pediatric Multiple Sclerosis Study Group and by the McDonald criteria 2010.; 3. Age under 18 years old at the beginning of treatment with natalizumab.; 4. At least 1 infusion of natalizumab in the context of POMS
• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Multiple sclerosis patients
• Estimated number of subjects:
  21

Study design details

• Main study objective:
  The primary objective of the study is to characterize the efficacy of natalizumab in patients diagnosed with POMS who have been treated for > 12 months with natalizumab in Portugal.
• Outcomes:
  •Annual relapse rate (ARR)•Proportion of subjects with EDSS progression•Proportion of subjects relapsed•Association of demographics and disease characteristics with ARR and time to relapse•Proportion of patients with no gadolinium-enhancing lesions•Proportion of patients with no new or enlarging T2 hyperintense lesions•Proportion of patients with No Evidence of Disease Activity, •Incidence adverse events(AE) and serious adverse events, discontinuation due to an AE •Discontinuation due to Anti-John Cunningham virus + serostatus/ progressive multifocal leukoencephalopathy risk concern•Discontinuation due to anti-natalizumab positive antibodies detected•Number of subjects hospitalized/with ER visits/requiring IV steroid treatment/who visit specialists due to MS relapse
• Data analysis plan:
  A descriptive analysis of all collected data and endpoints will be performed at the end of the study. All categorical data will be described by their absolute and relative frequencies. Continuous variables will be described by their minimum, maximum, median, mean and standard deviations. In the analysis of effects of baseline demographics and clinical characteristics on ARR at 12 months and on time to relapse will be performed with a 95% significance level. Because the sample size was chosen based solely on availability and was not to power for any endpoint, all analyses are exploratory in nature.

Documents

Study results

TyPed Study Report_v3_20190125__Redacted