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

NCT06628687 | Recruiting FDA Drug

• 2 other identifiers: 296FA402EUPAS1000000327
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (1)

• Number of Major Congenital Malformations (MCMs)

  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.

  Up to 10 years

Secondary Outcomes (15)

• Number of Minor Congenital Malformations

  Up to 10 years

• Number of Participants With Gestational Diabetes

  Up to 9 months

• Number of Participants With Pre-eclampsia

  Up to 9 months

• Number of Fetal Loss

  Up to 9 months

• Number of Spontaneous Abortions

  Up to 9 months

Study Arms (1)

Omaveloxolone

Participants with FA who were exposed to omaveloxolone at any time during pregnancy (from 12 days prior to conception through the end of pregnancy outcome), and/or at any time during lactation (up to 1 year of infant age or weaning, whichever comes first).

Drug: Omaveloxolone

Interventions

Omaveloxolone DRUG

Administered as specified in the treatment arm.

Also known as: SKYCLARYS, BIIB141, RTA 408

Omaveloxolone

Eligibility Criteria

• Age: 16 Years+
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• \- 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).

You may not qualify if:

• \- Not having exposure to omaveloxolone for FA.

Sponsors & Collaborators

• Biogen: lead

Study Sites (1)

Evidera

Morrisville, North Carolina, 27560, United States

RECRUITING

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

omaveloxolone

Condition Hierarchy (Ancestors)

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

Study Officials

• Medical Director

  Biogen

  STUDY DIRECTOR

Central Study Contacts

US Biogen Clinical Trial Center

CONTACT

866-633-4636; clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

CONTACT

clinicaltrials@biogen.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 30, 2024
• First Posted: October 8, 2024
• Study Start (Estimated): October 26, 2026
• Primary Completion (Estimated): April 30, 2035
• Study Completion (Estimated): April 30, 2035
• Last Updated: June 26, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will share

Locations

US (1)