Friedreich Ataxia
Conditions
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.
Interventions
DRUGOmaveloxolone
Administered as specified in the treatment arm.
Sponsors
Biogen
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Major Congenital Malformations (MCMs) | 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | A fetal loss that occurs for any reason at any time during pregnancy will be reported. |
| Number of Spontaneous Abortions | 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. |
| Number of Stillbirths | 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). |
| Number of Elective or Therapeutic Abortion | 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. |
| Number of Live Births | 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. |
| Number of Minor Congenital Malformations | 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. |
| Number of Small for Gestational Age (SGA) | 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. |
| Number of Neonatal Deaths | Within 28 days postdelivery | Neonatal death refers to the death of a live-born infant within the first 28 days of life. |
| Number of Infant Deaths | Up to approximately 1 year | Infant death refers to the death of a live-born infant within the first year of life. |
| Number of Infants with Abnormal Postnatal Growth and Development | 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). |
| Number of Infant Hospitalization due to Serious Illness | 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. |
| Number of Infant Serious or Opportunistic Infections | 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. |
| Number of Preterm Births | Up to 9 months | Preterm birth refers to a live birth at or before 37 weeks of gestation. |
Countries
United States
Contacts
Primary ContactUS Biogen Clinical Trial Center
Backup ContactGlobal Biogen Clinical Trial Center
Outcome results
None listed