Post-Authorisation Safety Study (PASS) of CLAIRYG® (Human normal immunoglobulin for intravenous use) in children under 12 years treated for primary immunodeficiency (PID) or immune thrombocytopenic purpura (ITP) (IGNGPASSP)

Study type

• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational study

Study drug and medical condition

• Name of medicine, other:
  CLAIRYG
• Medical condition to be studied:
  Primary immunodeficiency syndrome

  Immune thrombocytopenia

Population studied

• Age groups:
  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)
• Estimated number of subjects:
  59

Study design details

• Main study objective:
  The main objective of the study is to evaluate the safety of CLAIRYG® in common practice as replacement therapy for PIDs in children under 12 years, over a period of 12 months. The children with ITP treated at the same sites as the children with PIDs will also be included in the study in order to collected safety data in children receiving higher doses for immunomodulatory treatment.
• Outcomes:
  This study will collect all serious and non-serious adverse events, whether or not they are related to the administration of CLAIRYG® occurring in the children, with PID or ITP, treated with CLAIRYG® during the period of their follow-up, in order to document the product's safety in real conditions of use. To describe the conditions of use of Clairyg as part of common practice. To collect efficacy data to better document the benefit/risk ratio in the 2 diseases studies (PIDs and ITP) in children Under 12 years.
• Data analysis plan:
  For each group, a descriptive analysis will be performed on all of the data collected. - Quantitative variables will be described by their number, mean, standard deviation, median, limit values and missing data. - Qualitative variables will be described by their number, percentage and missing data. The 95% bilateral confidence intervals will only be given where applicable. Safety analysis : AE will be coded using the MedDRA dictionary classification. The number and frequency of AE will be described and listed by System Organ Class (SOC), Lowest Level Term (LLT) and Preferred Terms (PT). All AE will be described by type, seriousness, intensity/severity, causality, outcome. Exposure to CLAIRYG: mean doses (g and g/kg) administered per infusion (PID) or per treatment session (ITP) will be analysed. Efficacy analysis on each group : - PID: analysis of infections and IgG through serum levels - ITP: analysis of bleedings and platelet counts

Documents

Study results

PASS-CLAIRYG - Results summary_V1.0_17102023