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New registry aims to fill gap on fabry drug safety in pregnancy

NCT ID NCT06941025

Knowledge-focused Sponsor: Chiesi Farmaceutici S.p.A. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This 10-year observational registry will follow up to 10 women with Fabry disease who received pegunigalsidase alfa (Elfabrio) around the time of pregnancy or while breastfeeding, along with their infants. Researchers will track pregnancy outcomes, birth defects, and infant health up to 12 months after birth. The study does not change any treatment decisions—it simply collects real-world safety information.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pegunigalsidase alfa (Elfabrio)
What this could lead to
If successful, this study could provide crucial safety data on using pegunigalsidase alfa during pregnancy and breastfeeding for women with Fabry disease.
What could go wrong
This is a small observational registry (10 participants) with no control group, so results may not apply broadly. It only collects data, not testing a new treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2025

Expected to finish

Dec 2034

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Female participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Fabry disease pregnancy disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease Pregnancy Pregnancy complications

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    5 sites in 5 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • No physical study sites - Decentralized, web-based registry

    RECRUITING

    Washington D.C., District of Columbia, 20001, United States

  • No physical study sites - Decentralized, web-based registry

    RECRUITING

    Berlin, Germany

  • No physical study sites - Decentralized, web-based registry

    RECRUITING

    Rome, Italy

  • No physical study sites - Decentralized, web-based registry

    RECRUITING

    Madrid, Spain

  • No physical study sites - Decentralized, web-based registry

    NOT_YET_RECRUITING

    London, United Kingdom

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