Skip to main content
OpenTrials
Recruiting

NCT Number: 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

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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

16 year and older

Sex eligibility

Female

Study type

Observational

Primary location

Evidera

Morrisville, North Carolina, 27560, United States

Location status: Recruiting

Location contact

Ronna Chan

PRINCIPAL_INVESTIGATOR

CONTACT

919-270-6678

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Omaveloxolone

Drug

Administered as specified in the treatment arm.

Other names: SKYCLARYS, BIIB141, RTA 408

Primary outcomes

  1. Number of Major Congenital Malformations (MCMs)

    Time frame: Up to 10 years

    A MCM is defined as an abnormality of body structure or function that is present at birth; is of prenatal origin (i.e., birth defect); has significant medical, social, or cosmetic consequences for the affected participant; and typically requires medical intervention.

Secondary outcomes

  1. Number of Minor Congenital Malformations

    Time frame: Up to 10 years

    Minor congenital malformations include an anomaly or abnormality of body structure that is present at birth, is of prenatal origin (i.e., birth defect), poses no significant health problem in the neonatal period, and tends to have limited social or cosmetic consequences for the affected participant.

  2. Number of Participants With Gestational Diabetes

    Time frame: Up to 9 months

  3. Number of Participants With Pre-eclampsia

    Time frame: Up to 9 months

  4. Number of Fetal Loss

    Time frame: Up to 9 months

    A fetal loss that occurs for any reason at any time during pregnancy will be reported.

  5. Number of Spontaneous Abortions

    Time frame: Up to 9 months

    Spontaneous abortion refers to an involuntary fetal loss or the expulsion of the products of conception occurring prior to 20 gestational weeks.

  6. Number of Stillbirths

    Time frame: Up to 9 months

    Still birth refers to an involuntary fetal loss occurring at or after 20 gestational weeks or, if gestational age is unknown, a fetus weighing ≥ 350 grams (g).

  7. Number of Elective or Therapeutic Abortion

    Time frame: Up to 9 months

    Elective or therapeutic abortion refers to a voluntary fetal loss or interruption of pregnancy that occurs for any reason, including but not limited to for the preservation of maternal health or due to fetal abnormalities.

  8. Number of Live Births

    Time frame: Up to 9 months

    Live birth refers to the birth of a living fetus after 20 weeks of gestation or, if gestational age is unknown, weighing ≥ 350 g.

  9. Number of Preterm Births

    Time frame: Up to 9 months

    Preterm birth refers to a live birth at or before 37 weeks of gestation.

  10. Number of Small for Gestational Age (SGA)

    Time frame: Up to 9 months

    SGA is defined as birth weight less than 10th percentile for sex and gestational age using standard growth charts for full and preterm live-born infants.

  11. Number of Neonatal Deaths

    Time frame: Within 28 days postdelivery

    Neonatal death refers to the death of a live-born infant within the first 28 days of life.

  12. Number of Infant Deaths

    Time frame: Up to approximately 1 year

    Infant death refers to the death of a live-born infant within the first year of life.

  13. Number of Infants with Abnormal Postnatal Growth and Development

    Time frame: Up to approximately 1 year

    Infant growth measurements will be used to estimate gender-specific weight-for-length, head circumference-for-age, length-for-age, and weight-for-age percentiles. Developmental milestones (i.e., social/emotional, language/communication, neurocognitive, movement/physical development) will be evaluated to determine results of infant status (i.e., infants who are failing to achieve at least one milestone in any category will be considered developmentally delayed in that category).

  14. Number of Infant Hospitalization due to Serious Illness

    Time frame: Up to 1 year

    Infant hospital visit due to a serious (that is, results in significant disability, incapacity, or death; is life-threatening; requires inpatient or prolonged hospitalization; or is considered medically important) illness will be reported.

  15. Number of Infant Serious or Opportunistic Infections

    Time frame: Up to 1 year

    Infections that occur within an infant's first year of life and is either opportunistic (i.e., occurs more often or is more severe in people with weakened immune systems than in people with healthy immune systems) or serious (that is, results in significant disability, incapacity, or death; is life-threatening; requires inpatient or prolonged hospitalization; or is considered medically important) will be reported.

Study contacts

Contact information is provided by the study sponsor or research team.

Global Biogen Clinical Trial Center

CONTACT

[email protected]

US Biogen Clinical Trial Center

CONTACT

[email protected]

866-633-4636

Sponsors and collaborators

Lead sponsor

Biogen

Industry

Registry information

Official study 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

Important dates

Study start
2026
Primary completion
2035
Study completion
2035
First posted
Oct 8, 2024
Registry last updated
Jul 10, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.