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 (MOS)

March 17, 2026 updated by: Chiesi Farmaceutici S.p.A.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

10

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Berlin, Germany
        • Recruiting
        • No physical study sites - Decentralized, web-based registry
  • Italy
      • Rome, Italy
        • Recruiting
        • No physical study sites - Decentralized, web-based registry
  • Spain
      • Madrid, Spain
        • Recruiting
        • No physical study sites - Decentralized, web-based registry
  • United Kingdom
      • London, United Kingdom
        • Not yet recruiting
        • No physical study sites - Decentralized, web-based registry
  • United States
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20001
        • Recruiting
        • No physical study sites - Decentralized, web-based registry

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Pregnant and/or breastfeeding women with Fabry disease and their infants, after exposure to at least 1 dose of pegunigalsidase alfa during pregnancy and/or during lactation.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation
Other: Not applicable- observational study
Not applicable - observational study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Pregnancy outcome: Number of live births
Time Frame: at the delivery, after an average of 40 weeks of pregnancy
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
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
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

Outcome Measure
Time Frame
Number of neonates/infants with MCMs
Time Frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
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
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
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
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
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
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
up to 12 months of infant age
Number of hospitalisations in infants
Time Frame: up to 12 months of infant age
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
up to 12 months of infant age
Head circumference in infants (cm)
Time Frame: up to 12 months of infant age
up to 12 months of infant age
Weight in infants (kilograms)
Time Frame: up to 12 months of infant age
up to 12 months of infant age
Length in infants (cm)
Time Frame: up to 12 months of infant age
up to 12 months of infant age
Number of infants born as SGA
Time Frame: up to 12 months of infant age
up to 12 months of infant age
Number of infants with postnatal growth deficiency or FTT
Time Frame: up to 12 months of infant age
up to 12 months of infant age
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
up to 12 months of infant age
Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding
Time Frame: up to 12 months of infant age
up to 12 months of infant age

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chiesi Farmaceutici S.p.A.

Collaborators

ICON plc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 23, 2025

Primary Completion (Estimated)

December 1, 2034

Study Completion (Estimated)

December 1, 2034

Study Registration Dates

First Submitted

April 16, 2025

First Submitted That Met QC Criteria

April 22, 2025

First Posted (Actual)

April 23, 2025

Study Record Updates

Last Update Posted (Actual)

March 19, 2026

Last Update Submitted That Met QC Criteria

March 17, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CLI-06657AA1-06

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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