Study identification

EU PAS number

EUPAS38668

Study ID

38669

Official title and acronym

A Global Prospective Observational Study Of Women With Fabry Disease And Their Infants During Pregnancy And Breastfeeding (AT1001-037)

DARWIN EU® study

No

Study countries

United Kingdom
United States

Study description

This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat. All pregnant women with Fabry disease are eligible to enroll, an unexposed cohort potentially can be used for comparisons. Cases will be reported voluntarily to the Pregnancy Coordinating Center (PCC) from any country by Healthcare Providers (HCPs), by patients and secondary contacts, as well as, from any Fabry Amicus Registry, and from any ongoing Amicus clinical trials or expanded access programs. Patient enrollment and follow-up is facilitated remotely by the PCC and PCC Investigator. The PCC Investigator will oversee study operations and the PCC will be responsible for enrolling patients and collecting data as described in the protocol. The PCC will follow patients throughout their pregnancies and/or breastfeeding and infants through 1 year of age. All PCC activities will follow country-specific regulatory and ethics committee procedures. As the study is observational, medical treatment for each patient and infant will be consistent with routine clinical practice, and will not be mandated or required in any way by the study protocol. The study will enroll patients for a minimum of 10 years and may need to extend beyond 10 years if there are few participants compared to pharmacovigilance cases or an increased usage of migalastat in females of reproductive potential. Amicus will discuss with the Agency the status and interim results prior to closing the study.

Study status

Ongoing
Research institutions and networks

Institutions

Contact details

Fabry Pregnancy Registry Pregnancy Coordinating Center fabrypregnancy@ubc.com

Study contact
fabrypregnancy@ubc.com

Janine Collins

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Date of interim report, if expected

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Amicus Therapeutics, Inc.
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

EU RMP category 3 (required)