Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Not applicable- observational study.
- Who it may be relevant to
- Registry conditions: Fabry Disease, Pregnancy, Pregnancy Complications. Basic parameters: No limits · Female.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Germany, Italy, Spain, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj/Pegunigalsidase Alfa) During Pregnancy and/or Lactation
Overview
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The main objectives are to: * Assess pregnancy outcomes, including maternal and infant health. * Evaluate the occurrence of congenital malformations and other neonatal outcomes. This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs). Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.
Detailed description
This is a global, decentralized, single-arm, prospective and retrospective observational registry designed to evaluate pregnancy and infant outcomes in women with Fabry disease who have been exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The registry aims to assess maternal and infant safety, pregnancy outcomes, and the occurrence of congenital malformations and other neonatal conditions.
The registry will enroll patients over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data collection will be facilitated through a secure, centralized web-based platform, where patients and physicians can enter information using electronic case report forms (eCRFs).
Enrollment \& Data Collection:
Patients can be enrolled at any time, either during pregnancy or after delivery. Depending on the timing of enrollment, data will be collected retrospectively and/or prospectively.
Pregnancy and clinical outcomes will be monitored from enrollment until the infant reaches 12 months of age.
Collected data includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health parameters.
Reported congenital malformations will be classified according to established criteria (e.g., MACDP, EUROCAT) and adjudicated by an independent Scientific Advisory Committee.
The registry is observational and does not alter clinical care, physician treatment decisions, or patient management.
Interventions
- Other Not applicable- observational study
Not applicable - observational study
Primary outcome measures
- Pregnancy outcome: Number of live births [Time frame: at the delivery, after an average of 40 weeks of pregnancy]
- Pregnancy outcome: Number of preterm birth [Time frame: at delivery, prior to 37 weeks of gestation]
- Pregnancy outcome: Number of pregnancy losses (number of spontaneous abortions , number of pregnancy terminations, number of foetal deaths or stillbirths) [Time frame: spontaneous abortions: up to 20 weeks of pregnancy; pregnancy terminations: through the pregnancy; number of foetal deaths or stillbirths: greater than 20 weeks of pregnancy and through the pregnancy, average of 40 weeks]
Secondary outcome measures (12)
- Number of neonates/infants with MCMs [Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
- Number of ectopic or molar pregnancies [Time frame: through the pregnancy, an average of 40 weeks]
- Number of women with obstetric and delivery complications [Time frame: at the delivery, an average of 40 weeks of pregnancy]
- Number of women with complications of preeclampsia or eclampsia [Time frame: through the pregnancy, an average of 40 weeks]
- Number of women with complications of preterm prelabour rupture of membrane [Time frame: at delivery, prior to 37 weeks of gestation]
- Number of neonates/infants with minor congenital malformations [Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
- Number of infants with developmental deficiency [Time frame: up to 12 months of infant age]
- Number of hospitalisations in infants [Time frame: up to 12 months of infant age]
- Mortality in infants, including neonatal death and infant death [Time frame: up to 12 months of infant age]
- Head circumference in infants (cm) [Time frame: up to 12 months of infant age]
- Weight in infants (kilograms) [Time frame: up to 12 months of infant age]
- Length in infants (cm) [Time frame: up to 12 months of infant age]
Eligibility criteria
Inclusion criteria
- Female patients with Fabry disease who have been exposed to at least 1 dose of pegunigalsidase alfa at any time during pregnancy (defined as having received pegunigalsidase alfa within 30 days prior to the DOC and/or during pregnancy) and/or during lactation, and their infants.
o DOC, defined as 20/7 gestational weeks, will be calculated from last menstrual period \[LMP\] or ultrasound
- Patient or parent/legally authorized representative must be able to understand and provide consent through an Institutional Review Board / Independent Ethics Committee (IRB/IEC) approved Informed Consent Form.
Exclusion criteria
- None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- No physical study sites - Decentralized, web-based registry — Washington D.C.
Germany · 1 center
- No physical study sites - Decentralized, web-based registry — Berlin
Italy · 1 center
- No physical study sites - Decentralized, web-based registry — Rome
Spain · 1 center
- No physical study sites - Decentralized, web-based registry — Madrid
United Kingdom · 1 center
- No physical study sites - Decentralized, web-based registry — London
Identifiers
NCT: NCT06941025 · CLI-06657AA1-06