Prospective, Registry-Based Observational Cohort Study of Zavegepant Safety in Pregnancy

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, other:
  Zavzpret
• Study drug International non-proprietary name (INN) or common name:
  MIGLUSTAT
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AX06) miglustat

  miglustat

Population studied

• Short description of the study population:
  Pregnant individuals 15 to 50 years of age in the US, including individuals with migraine exposed to zavegepant during pregnancy and individuals with migraine unexposed to zavegepant during pregnancy.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
• Special population of interest:
  Pregnant women
• Estimated number of subjects:
  728

Study design details

• Study design:
  A registry-based, prospective observational cohort study
• Main study objective:
  1. To estimate the prevalence of MCM births (primary outcome) among pregnant individuals with migraine who are (1) exposed to zavegepant (internal exposed cohort), and (2) unexposed to zavegepant (internal comparator cohort).; ; 2. To estimate the RR of MCM births in the exposed internal cohort versus the unexposed internal cohort.
• Setting:
  The 2 internal study cohorts will be derived from eligible individuals enrolled in the pregnancy registry. The virtual registry coordinating center (VRCC) will coordinate enrollment and data collection for the internal study cohorts (details provided in Protocol Section 9.4). The external nonmigraine cohort will include individuals identified within the claims database. All 3 cohorts of pregnant individuals will be identified in the US during the study period.
• Comparators:
  Internal comparator cohort; All remaining eligible individuals who are not exposed to zavegepant during pregnancy will be included in the comparator cohort. This cohort will include individuals who are and are not exposed to migraine treatments. ; ; External nonmigraine cohort; An external cohort of pregnant individuals without migraine will provide context to the main study results by estimating background rates of the study outcomes among pregnant individuals. This cohort will be identified within the ORD, a US-based health insurance claims database. See Protocol Section 9.4.5 for details about the ORD. Methods for identifying and characterizing the external nonmigraine cohort are described in detail in Annex 1, the draft protocol “Observational Cohort Study of Zavegepant Safety in Pregnancy Within a US Claims Database.”;
• Outcomes:
  MCM is the primary outcome of interest and all other outcomes are secondary. All outcome data are collected via HCPs. Protocol Table 1 presents the definitions of the outcomes of interest.
• Data analysis plan:
  Detailed methodology for summary and statistical analyses of data collected in this study will be documented in a statistical analysis plan (SAP), which will be dated, filed and maintained by the Sponsor. The SAP may modify the plans outlined in the protocol; any major modifications of primary endpoint definitions or their analyses would be reflected in a protocol amendment.