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NCT Number: NCT06941025

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

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.

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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Pregnancy Pregnancy Complications Sphingolipidoses Urogenital Diseases Vascular Diseases

Sex eligibility

Female

Study type

Observational

Primary location

No physical study sites - Decentralized, web-based registry, Berlin, Germany

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About this study

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.

Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

Not applicable- observational study

Other

Not applicable - observational study

Primary outcomes

  1. Pregnancy outcome: Number of live births

    Time frame: at the delivery, after an average of 40 weeks of pregnancy

  2. Pregnancy outcome: Number of preterm birth

    Time frame: at delivery, prior to 37 weeks of gestation

  3. 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 outcomes

  1. Number of neonates/infants with MCMs

    Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age

  2. Number of ectopic or molar pregnancies

    Time frame: through the pregnancy, an average of 40 weeks

  3. Number of women with obstetric and delivery complications

    Time frame: at the delivery, an average of 40 weeks of pregnancy

  4. Number of women with complications of preeclampsia or eclampsia

    Time frame: through the pregnancy, an average of 40 weeks

  5. Number of women with complications of preterm prelabour rupture of membrane

    Time frame: at delivery, prior to 37 weeks of gestation

  6. 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

  7. Number of infants with developmental deficiency

    Time frame: up to 12 months of infant age

  8. Number of hospitalisations in infants

    Time frame: up to 12 months of infant age

  9. Mortality in infants, including neonatal death and infant death

    Time frame: up to 12 months of infant age

  10. Head circumference in infants (cm)

    Time frame: up to 12 months of infant age

  11. Weight in infants (kilograms)

    Time frame: up to 12 months of infant age

  12. Length in infants (cm)

    Time frame: up to 12 months of infant age

  13. Number of infants born as SGA

    Time frame: up to 12 months of infant age

  14. Number of infants with postnatal growth deficiency or FTT

    Time frame: up to 12 months of infant age

  15. Duration of breastfeeding, number of exclusively breastfeeding women and number of breastfeeding women supplemented with formula

    Time frame: up to 12 months of infant age

  16. Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding

    Time frame: up to 12 months of infant age

Study contacts

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

Chiesi Clinical Trial

CONTACT

[email protected]

+3905212791

Sponsors and collaborators

Lead sponsor

Chiesi Farmaceutici S.p.A.

Industry

Collaborators

  • ICON plc

Registry information

Official study 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

Acronym: MOS

Important dates

Study start
2025
Primary completion
2034
Study completion
2034
First posted
Apr 23, 2025
Registry last updated
Mar 19, 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.

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