A Post-marketing, Observational, Descriptive Study to Assess the Risk Associated With Pregnancy, the Maternal Complications and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Omaveloxolone During Pregnancy and/or Lactation

Study type

• Study topic:
  Disease /health condition

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine, other:
  Omaveloxolone (SKYCLARYS)
• Medical condition to be studied:
  Friedreich's ataxia

Population studied

• Short description of the study population:
  The study population will include participants with FA who were exposed to omaveloxolone at any time during pregnancy and/or lactation. Participants will be enrolled prospectively and retrospectively. Women who enroll prior to pregnancy outcome, and/or prior to start of breastfeeding will be enrolled as prospective participants. Women who enroll after the pregnancy has occurred and/or following the start of breastfeeding will be enrolled as retrospective participants. Retrospective participants will not be included in the analysis population. For prospective and retrospective participants exposed during lactation only, data will be reported separately from pregnancy exposure cases.
• 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:
  Nursing women

  Pregnant women
• Estimated number of subjects:
  20

Study design details

• Study design:
  A post-marketing, prospective and retrospective study
• Main study objective:
  In this study, researchers will learn more about the safety of BIIB141, also known as omaveloxolone or SKYCLARYS. This is a drug available for doctors to prescribe for people with Friedrich’s Ataxia, also known as FA. This is known as an “observational” study, which collects health information about study participants without changing their medical care. Participants for this study will have taken BIIB141 at any time during pregnancy and/or while breastfeeding or pumping up through the first year after delivery. Participants can join this study on their own or they may be enrolled by their regular doctors. This study is also known as the “SKYCLARYS (Omaveloxolone) Pregnancy and Lactation Surveillance Program.”; ; The main objective of this study is to learn more about how BIIB141 may affect pregnancy, as well as any effects on the health of the mother and of the baby during its first year of life.; ; The main question researchers want to answer in this study is:; ; · Does taking BIIB141 during pregnancy or breastfeeding lead to any major birth defects?; ; Researchers will also learn more about:; ; · Does taking BIIB141 during pregnancy or breastfeeding lead to any minor birth defects?; ; · Does taking BIIB141 during pregnancy or breastfeeding affect the following:; ; · Gestational diabetes, a disease that can happen during pregnancy that affects how your body uses sugar; ; · Pre-eclampsia, a pregnancy-related high blood pressure disease; ; · Unborn baby being small for its expected age (usually in weeks); ; · Loss of an unborn baby; ; · Live birth; ; · Premature birth; ; · Loss of a newborn; ; · Growth or developmental delays in the baby; ; · Serious illness in the baby resulting in hospitalization; ; · Serious infections in the baby, or ones in babies with a weakened immune system; ; This study will be done as follows:; ; · Participants will join the study after signing an informed consent form, also known as an ICF.; ; · During the study, health information from the participants’ regular visits to their doctor will be collected based on whether participant joined the study while pregnant or after the baby is born.; ; · Each participant will be in the study for up to 1 year after the birth of their child, unless they decide to leave early. Overall, this study is expected to last at least 10 years.
• Outcomes:
  Number of Major Congenital Malformations, Number of Minor Congenital Malformations, Number of Participants With Gestational Diabetes and Number of Participants With Preeclampsia, Number of Fetal Losses due to Stillbirth, Spontaneous Abortions, Elective or Therapeutic Abortions, Number of Live Births, and Number of Preterm Births, Number of Small Gestational Age, Number of Neonatal Deaths and Number of Infant Deaths, Number of Infants With Abnormal Postnatal Growth and Development, Number of Infant Hospitalization due to Serious Illness, Number of Infant Serious or Opportunistic Infections
• Data analysis plan:
  Epidemiologic methods will be employed for data collection and analyses. For each continuous variable, the number of observations, median, mean, standard deviation, interquartile range, minimum, and maximum will be reported. For each categorical variable, the frequency and percentage in each category will be reported.