At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- Mostly remote — few or no hospital visits
- Drug or intervention
- Not applicable- observational study (other)
- How long the study runs
- Study runs about 115 months (dates as stated)
- About the drug or intervention
- Not applicable- observational study — other: Not applicable - observational study
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at the safety of a medicine called Elfabrio (pegunigalsidase alfa) for women with Fabry disease who took it during pregnancy or while breastfeeding, and for their babies. Fabry disease is a rare inherited condition. The study is run by Chiesi Farmaceutici S.p.A.
Who can take part
- Women with Fabry disease who had at least one dose of pegunigalsidase alfa during pregnancy (within 30 days before the start of pregnancy or during pregnancy) or while breastfeeding, and their babies.
- The woman, parent, or legal representative must be able to understand and agree to take part by signing an approved consent form.
Who may not be able to
- None stated — the trial lists no exclusion criteria.
What taking part involves
- • Not stated — ask the trial team. This is an observational study, meaning researchers collect information without asking participants to change their treatment.
Time commitment: Not stated — ask the trial team about visits, duration, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- Not specified
- Who
- Female
- Number of participants
- 10
- Started
- 2025-05-23
- Last checked
- 2026-03
Plain English Summary
What is this study?
- • Testing a new treatment for fabry disease
- • Clinical study - 10 participants
- • 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
Who can take part?
- • Adults
- • Diagnosed with fabry disease
- • Female only
Where?
- • London - No physical study sites - Decentralized, web-based registry
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: Female only
What the study is looking for
- ✓o DOC, defined as 20/7 gestational weeks, will be calculated from last menstrual period \[LMP\] or ultrasound
See the full criteria
Where Is This Study? (1 UK site)
No physical study sites - Decentralized, web-based registry
London, United Kingdom
How to Get in Touch
Chiesi Clinical Trial
Sponsor contactCONTACT