Recruiting

Pegunigalsidase Alfa

Sponsor:

Chiesi Farmaceutici S.p.A.

Code:

NCT06941025

Conditions

Fabry Disease

Pregnancy

Pregnancy Complications

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Interventions

Not applicable- observational study

Study Details

Brief summary:

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.

Conditions

Fabry Disease

Pregnancy

Pregnancy Complications

Study ID

NCT06941025

Start date

May 23, 2025

Status verified date

Mar, 2026

Completion date

Dec, 2034

Anticipated

Primary completion date

Dec, 2034

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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 Design

Enrollment

10 participants

Anticipated

Interventions and Outcome Measures

Arms

Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation

Interventions

Not applicable- observational study

Not applicable - observational study

Primary outcome measure

  • Pregnancy outcome: Number of live births [ Time Frame: 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 ]
  • 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 ]

Central Contacts and Locations

Central contacts

Chiesi Clinical Trial

+3905212791clinicaltrials_info@chiesi.com

Locations

No physical study sites - Decentralized, web-based registry

Recruiting

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

More Information

Sponsor

Chiesi Farmaceutici S.p.A.

Last update posted

Mar 19, 2026

Last verified

Mar, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Chiesi Farmaceutici S.p.A. on 2026-03-19.