ClinicalTrials.gov record 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
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This global observational registry collects information on pregnancies and infants when women with Fabry disease were exposed to pegunigalsidase alfa within 30 days before conception and/or during pregnancy or breastfeeding. It accepts both retrospective and prospective entries and uses a secure web platform for patient and physician data entry. Pregnancy outcomes are recorded through delivery and infants are followed up to 12 months. The registry is observational and does not change clinical care.
What it is studying. The study aims to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who were exposed to pegunigalsidase alfa, including maternal health, delivery outcomes, congenital malformations, and infant health up to 12 months of age.
What the researchers hope to learn. To assess pregnancy outcomes and maternal and infant safety after exposure to pegunigalsidase alfa, and to evaluate the occurrence of congenital malformations and other neonatal conditions.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Other
- Not applicable- observational study
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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 description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants (women and their infants) will provide medical and pregnancy information via a secure web-based platform; enrollment may occur during pregnancy or after delivery and can include retrospective or prospective data. Collected information includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health and growth up to 12 months. Self-enrolled data will be confirmed by the participant's primary/attending physician where required. This is an observational registry and does not change clinical care or treatment decisions.
How the study is structured. A worldwide, decentralized registry that collects both past (retrospective) and ongoing (prospective) information. Patients and/or their physicians enter data into a secure web-based system. It is a single-group, observational study that does not assign treatments. Pregnancy outcomes are tracked through delivery and infants are followed for up to 12 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Germany, Italy, Spain, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Fabry disease
Important dates
- Study start
- May 23, 2025
- Primary completion
- December 2034 (estimated)
- Study completion
- December 2034 (estimated)
- Target enrollment
- 10 participants (estimated)
- Last updated on registry
- Mar 19, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Collaborators
- ICON plc
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Chiesi Clinical Trial 📞 +3905212791 ✉ clinicaltrials_info@chiesi.com
Participating sites (5)
- No physical study sites - Decentralized, web-based registryRecruiting
Washington D.C., District of Columbia, 20001 · United States
- No physical study sites - Decentralized, web-based registryRecruiting
Berlin · Germany
- No physical study sites - Decentralized, web-based registryRecruiting
Rome · Italy
- No physical study sites - Decentralized, web-based registryRecruiting
Madrid · Spain
- No physical study sites - Decentralized, web-based registryNot Yet Recruiting
London · United Kingdom
Terms used on this page · explanations by MedicalUpdates.ai
- registry
- An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- legally authorized representative
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- Informed Consent Form
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- Enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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