A Prospective Active Surveillance Study to Monitor the Real-World Safety of Ritlecitinib Among Adolescents with Alopecia Areata

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  LITFULO
• Name of medicine, other:
  ritlecitinib tosylate
• Study drug International non-proprietary name (INN) or common name:
  RITLECITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AF08) ritlecitinib

  ritlecitinib
• Medical condition to be studied:
  Alopecia areata

Population studied

• Short description of the study population:
  Adolescents 12-17 years old receiving ritlecitinib for Alopecia areata (AA) and adolescents in the comparator cohort, including those exposed to other approved systemic medications for the treatment of AA in adolescents.
• Age groups:
  Adolescents (12 to < 18 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Adolescents
• Estimated number of subjects:
  1000

Study design details

• Study design:
  This will be a prospective observational cohort study of adolescents with severe AA who receive ritlecitinib and those in the comparator cohort, including those exposed to other approved systemic medications for the treatment of AA in adolescents.
• Main study objective:
  Among adolescent participants with AA who are treated with ritlecitinib and, separately, among adolescent participants in the comparator cohort, including those exposed to other approved systemic medications for the treatment of AA in adolescents, to:; • Estimate growth and bone development metrics;; • Estimate maturation and pubertal development metrics;; • Estimate the incidence of bone fractures; and; • Estimate the incidence of neurotoxicity events
• Setting:
  CorEvitas International Adolescent AA Registry is a prospective, multicentre, observational registry initiated to evaluate treatment outcomes in adolescents with severe AA. Sites include dermatological and hair-loss clinics throughout the EU, as well as the UK, the US, and Canada that have a high volume of treated adolescent patients with AA are identified and asked to participate, targeting up to 65 study sites.
• Comparators:
  Participants without previous exposure to ritlecitinib who initiate another systemic medication for the treatment of AA, including non-ritlecitinib JAK inhibitors, at the time of registry enrolment or within 6 months prior to enrolment, will be assigned to the comparator cohort. ; Participants with severe AA in the opinion of the provider at the time of registry enrolment who are unexposed to systemic medications for AA within the 6 months prior to, or at the time of, registry enrolment and who are not prescribed a systemic treatment for AA at registry enrolment, will also be included in the comparator cohort.
• Outcomes:
  Growth outcomes will include the change from baseline to end of follow-up in participant's height standard deviation score (SDS) and weight SDS measures. Other descriptive measurements of growth will also be reported including: standing height, height percentiles, height velocity, height velocity SDS, weight, weight percentiles, body mass index (BMI), BMI percentiles, and BMI SDS. To evaluate bone development, the occurrence of bone fractures will be assessed. Maturation and pubertal development will be measured. Neurotoxicity events will be reported.
• Data analysis plan:
  For the primary analyses, descriptive statistics for each outcome of interest will be computed separately by exposure cohort. For growth and bone development metrics, mean and SD will be presented for continuous metrics related to height, weight, and BMI, including change from baseline to end of follow-up in standing height, height percentile, height SDS, height velocity SDS, weight, weight percentile, weight SDS, BMI, BMI percentile, and BMI SDS. The proportion of participants whose height SDS at the end of follow-up is more than 1 or 2 standard deviations lower than their baseline height SDS will also be reported.; ; Tanner staging, a categorical maturation and pubertal development metric, will be described using counts and percentages by exposure cohort, and by sex within each exposure cohort. The cumulative incidence and IR of bone fractures and neurological adverse events, overall and by type, will be estimated within each exposure cohort.; ; Outcomes of interest will be compared between exposure cohorts as exploratory analyses. Conventional linear regression models will be fit on the PS-matched sample for continuous outcomes, including change in height SDS, change in weight SDS, age at PHV, and age at Tanner stage progression. To compare bone fractures and neurological adverse events, generalised linear regression models will be fit on the PS-matched sample.; ; In order to increase sample size and statistical power, the primary and exploratory analyses are proposed to be performed on a pooled sample of participants from Europe (UK and EU countries) and North America (US and Canada).