Maternal and Postnatal Outcomes: A worldwide decentralized observational registry to evaluate the safety in women with Fabry disease and their infants after exposure to Elfabrio (pegunigalsidase alfa-iwxj/pegunigalsidase alfa) during pregnancy and/or lactation

Study summary

No information provided.

Study identification

• EU PAS number:
  EUPAS1000000957
• Study ID:
  1000000957
• Official title and acronym:
  Maternal and Postnatal Outcomes: A worldwide decentralized observational registry to evaluate the safety in women with Fabry disease and their infants after exposure to Elfabrio (pegunigalsidase alfa-iwxj/pegunigalsidase alfa) during pregnancy and/or lactation
• DARWIN EU® study:
  No
• Study countries:
  Germany

  Italy

  Spain

  United Kingdom

  United States
• Study description:
  This is a worldwide, decentralized, single arm, prospective and retrospective, observational registry in women with Fabry disease exposed to pegunigalsidase alfa during pregnancy (within 30 days prior to the date of conception [DOC] and/or during pregnancy) and/or lactation, and their infants. ; The objective of this registry is to evaluate pregnancy and clinical outcomes in women with Fabry disease and their infants after exposure to pegunigalsidase alfa at any time during pregnancy and/or lactation, and their infants. ; The registry is planned to enrol over a period of 10 years. ; The registry will allow physicians to enrol eligible patients and will also allow eligible patients to self-enrol, in accordance with local regulations. Patient enrolment and data collection will be coordinated through a centralized web-based platform. The web-based platform will include information about the registry and will allow the patient or the physician to create an online account to participate in the registry and independently and remotely record patient's data through electronic case report forms (eCRFs). ; After enrolment in the registry, pregnancy and clinical outcomes will be collected throughout pregnancy and up to the infant's 12 months of age. Pregnancy information will be collected until deliver or within 42 days of termination of pregnancy. Patients can be enrolled in the registry at any time and depending on when enrolment occurs, patient and infant data will be collected retrospectively and/or prospectively. Information on pregnancy and infant outcomes provided by self-enrolled patients will be confirmed with their primary care or other attending physician. ; Congenital malformations will be described according to established criteria (e.g., Metropolitan Atlanta Congenital Defects Program [MACDP] criteria, European Surveillance of Congenital Anomalies [EUROCAT] criteria). Reported congenital malformations will be adjudicated by an independent Scientific Advisory Committee
• Study status:
  Ongoing

Research institutions and networks

Institutions

Chiesi Farmaceutici

ICON Commercialisation & Outcomes

Germany

Ireland

Study timelines

Date when funding contract was signed

Actual:18/12/2024

Study start date

Actual:23/05/2025

Data analysis start date

Planned:30/03/2035

Date of final study report

Planned:30/10/2035

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Chiesi Farmaceutici

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Non-EU RMP only

Other study registration identification numbers and links

CLI-0667AA1-06

Maternal and Postnatal Outcomes (MOS)

NCT06941025