Clinical trial NCT06628687: A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
Official title: 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: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Friedreich Ataxia
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2024-10-08
Enrollment: 20
Sex: FEMALE
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief summary: 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.

Detailed description: The objective is to conduct a worldwide descriptive study to collect prospective and retrospective data in women exposed to omaveloxolone during pregnancy and/or lactation to assess risk associated with pregnancy, the maternal complications, and adverse effects on the developing fetus, neonate, and infant (through at least the first year of life) in the post-marketing setting.

Eligibility criteria: Key Inclusion Criteria:

- Exposure to omaveloxolone for FA at any time during pregnancy (from 12 days prior to conception to pregnancy outcome) and/or at any time during lactation (up to 1 year of infant age or weaning, whichever comes first).

Key Exclusion Criteria:

- Not having exposure to omaveloxolone for FA.

Note: Other protocol-defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Number of Major Congenital Malformations (MCMs)
timeFrame: Up to 10 years
Secondary outcomes:
measure: Number of Minor Congenital Malformations
timeFrame: Up to 10 years
measure: Number of Participants With Gestational Diabetes
timeFrame: Up to 9 months
measure: Number of Participants With Pre-eclampsia
timeFrame: Up to 9 months
measure: Number of Fetal Loss
timeFrame: Up to 9 months
measure: Number of Spontaneous Abortions
timeFrame: Up to 9 months
measure: Number of Stillbirths
timeFrame: Up to 9 months
measure: Number of Elective or Therapeutic Abortion
timeFrame: Up to 9 months
measure: Number of Live Births
timeFrame: Up to 9 months
measure: Number of Preterm Births
timeFrame: Up to 9 months
measure: Number of Small for Gestational Age (SGA)
timeFrame: Up to 9 months
measure: Number of Neonatal Deaths
timeFrame: Within 28 days postdelivery
measure: Number of Infant Deaths
timeFrame: Up to approximately 1 year
measure: Number of Infants with Abnormal Postnatal Growth and Development
timeFrame: Up to approximately 1 year
measure: Number of Infant Hospitalization due to Serious Illness
timeFrame: Up to 1 year
measure: Number of Infant Serious or Opportunistic Infections
timeFrame: Up to 1 year
Other outcomes:
Locations:
city: Morrisville
country: United States
facility: Evidera
state: North Carolina
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: