Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Scope of the study

Safety study (incl. comparative)

Data collection methods

Primary data collection
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

LITFULO

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

RITLECITINIB

Anatomical Therapeutic Chemical (ATC) code

(L04AF08) ritlecitinib
ritlecitinib

Medical condition to be studied

Pregnancy
Alopecia areata
Population studied

Short description of the study population

Two cohorts of pregnant individuals with AA in the US. The study cohorts will include individuals exposed to ritlecitinib during pregnancy and individuals unexposed to ritlecitinib during pregnancy who are enrolled in the registry.

Age groups

  • Paediatric Population (< 18 years)
    • Neonate
      • Preterm newborn infants (0 – 27 days)
      • Term newborn infants (0 – 27 days)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)
    • Adults (18 to < 65 years)
      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)
      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)

Special population of interest

Pregnant women

Estimated number of subjects

400
Study design details

Study design

This study is a prospective, registry-based observational cohort study. This study will be a new, product-based pregnancy registry. Data will be collected from eligible participants in the US.

Main study objective

Primary objective: To estimate the prevalence of MCM births among pregnant individuals with AA who are (1) exposed to ritlecitnib and (2) unexposed to ritlecitnib.

Setting

The study population will be derived from eligible individuals in the US enrolled in the pregnancy registry.

Outcomes

Outcomes of interest
Primary: Major congenital malformation (MCM)
Secondary: Spontaneous abortion (SAB), elective termination, pregnancy complications (pre-eclampsia, eclampsia), stillbirth, preterm birth, SGA, minor congenital malformation, infant postnatal growth deficiency, and infant developmental delay.

Data analysis plan

The analysis population will include valid participants who were prospectively enrolled in the registry, are not exposed to teratogens or investigational medications during pregnancy, and who are not considered lost to follow up. Patient characteristics will be summarized with descriptive statistics for each cohort. The number of observations, median, mean, standard deviation, minimum, and maximum will be reported for each continuous variable. The frequency and percentage per category will be reported for each categorical variable. Prevalence of the outcomes of interest will be calculated according to the conventions described in the protocol. In general, the prevalence of each outcome will be calculated by dividing the number of cases of the outcome by the appropriate denominator for that particular outcome, based on clinical knowledge. For most outcomes, the analysis population (denominator) will be the number of pregnant individuals with pregnancy outcome data, the number of live births, or the number of infants with follow-up data at the timepoint of interest, as appropriate; however, for some outcomes, the analysis population (denominator) will be restricted based on certain relevant factors. Comparative analyses will be conducted for each outcome if sample size permits. Crude (unadjusted) RRs (and corresponding 95% CIs) will be calculated using Exact methods. Adjusted RRs will be calculated using generalized linear models (binomial family) with a log (RR) link and weighted by IPTW. The Clopper-Pearson method will be used to derive 95% CIs. IPTW will be calculated using propensity scores estimated from propensity score models. Each individual’s propensity score will be estimated using a logistic regression model with exposure status as the outcome (dependent variable). Detailed methodology for summary and statistical analysis of the data collected in this study will be documented in a statistical analysis plan