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| Name | Class |
|---|---|
| ICON plc | INDUSTRY |
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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:
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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Not applicable- observational study | Other | Not applicable - observational study |
|
| Measure | Description | Time Frame |
|---|---|---|
| Pregnancy outcome: Number of live births | at the delivery, after an average of 40 weeks of pregnancy | |
| Pregnancy outcome: Number of preterm birth | 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) | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of neonates/infants with MCMs | through the pregnancy, an average of 40 weeks and up to 12 months of infant age | |
| Number of ectopic or molar pregnancies | through the pregnancy, an average of 40 weeks |
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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:
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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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Chiesi Clinical Trial | Contact | +3905212791 | clinicaltrials_info@chiesi.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| No physical study sites - Decentralized, web-based registry | Recruiting | Washington D.C. | District of Columbia | 20001 | United States |
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| Label | URL |
|---|---|
| Official registry website with information on eligibility, participation, and data collection for the observational study on pregnancy and infant outcomes in women with Fabry disease. | View source |
| Official registry website with information on eligibility, participation, and data collection for the observational study on pregnancy and infant outcomes in women with Fabry disease. | View source |
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| Number of women with obstetric and delivery complications | at the delivery, an average of 40 weeks of pregnancy |
| Number of women with complications of preeclampsia or eclampsia | through the pregnancy, an average of 40 weeks |
| Number of women with complications of preterm prelabour rupture of membrane | at delivery, prior to 37 weeks of gestation |
| Number of neonates/infants with minor congenital malformations | through the pregnancy, an average of 40 weeks and up to 12 months of infant age |
| Number of infants with developmental deficiency | up to 12 months of infant age |
| Number of hospitalisations in infants | up to 12 months of infant age |
| Mortality in infants, including neonatal death and infant death | up to 12 months of infant age |
| Head circumference in infants (cm) | up to 12 months of infant age |
| Weight in infants (kilograms) | up to 12 months of infant age |
| Length in infants (cm) | up to 12 months of infant age |
| Number of infants born as SGA | up to 12 months of infant age |
| Number of infants with postnatal growth deficiency or FTT | up to 12 months of infant age |
| Duration of breastfeeding, number of exclusively breastfeeding women and number of breastfeeding women supplemented with formula | up to 12 months of infant age |
| Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding | up to 12 months of infant age |
| 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 |
| ID | Term |
|---|---|
| D000795 | Fabry Disease |
| D011248 | Pregnancy Complications |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D059345 | Cerebral Small Vessel Diseases |
| D002561 | Cerebrovascular Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
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