Long-term Follow-up of Nivolumab and Ipilimumab- (as Monotherapy and as Combination Therapy)-treated Pediatric Patients Enrolled in the Dutch Melanoma Treatment Registry (DMTR)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  IPILIMUMAB

  NIVOLUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01FX04) ipilimumab

  ipilimumab

  (L01FF01) nivolumab

  nivolumab
• Medical condition to be studied:
  Malignant melanoma

Population studied

• 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)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The objective of this study is to evaluate long-term outcomes in pediatric patients with melanoma receiving nivolumab monotherapy, ipilimumab monotherapy, or nivolumab in combination with ipilimumab.
• Outcomes:
  Frequency of Grade 3- and 4 treatment-related AEs and their management and outcomes. Treatment patterns, and long-term outcomes (overall survival, time to progression, progression free survival, and growth and sexual maturation) in pediatric patients with melanoma treated with Nivolumab and Ipilimumab- (as Monotherapy or as Combination Therapy).
• Data analysis plan:
  Analyses will be descriptive and will include mean, standard deviation (SD) median, minimum, and maximum for continuous variables, and count and percentage for categorical variables. Data will be presented stratified by cohort, based on age at time of first dose of treatment (Nivolumab and Ipilimumab- as Monotherapy or as Combination Therapy). Cohort 1: patients 12 to under 18 years of age and Cohort 2: patients under 12 years of age.