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A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04252066
Recruitment Status : Recruiting
First Posted : February 5, 2020
Last Update Posted : April 28, 2020
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Tracking Information
First Submitted Date December 18, 2019
First Posted Date February 5, 2020
Last Update Posted Date April 28, 2020
Actual Study Start Date April 17, 2020
Estimated Primary Completion Date August 2029   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 30, 2020)
Number of major birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: January 30, 2020)
  • Incidence of spontaneous abortion [ Time Frame: : up to 20 weeks ]
  • Number of elective or induced abortion [ Time Frame: Through the pregnancy, an average of 40 weeks ]
  • Number of fetal death or stillbirth [ Time Frame: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week ]
  • Number of live birth [ Time Frame: at the delivery, an average of 40 weeks of pregnancy ]
  • Number of neonatal death [ Time Frame: up to 28 days of neonatal life ]
  • Number of minor birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
  • Number of ectopic or molar pregnancy [ Time Frame: Through the pregnancy, an average of 40 weeks ]
    an ectopic or molar pregnancy occurs outside of the uterus.
  • Neurodevelopmental problems [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
  • Adverse fetal outcomes other than birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
  • Number of obstetric and delivery complications [ Time Frame: At the delivery, an average of 40 weeks of pregnancy ]
  • Occurrence of Jaundice cases in Infants [ Time Frame: Up to 1 year ]
  • Number of hospitalizations in infants [ Time Frame: Up to 1 year ]
  • Mortality in Infants [ Time Frame: Up to 1 year ]
  • Head circumference in Infants (cm) [ Time Frame: Up to 1 year ]
  • Weight in Infants (kilograms) [ Time Frame: Up to 1 year ]
  • Length in Infants (cm) [ Time Frame: Up to 1 year ]
  • Occurrence of milk allergic reaction in breastfed or formula supplemented infants [ Time Frame: Up to 1 year ]
  • Occurrence of allergic reaction in patients who are breastfeeding [ Time Frame: Up to 1 year ]
  • Frequency of adverse events effecting lactation [ Time Frame: Up to 1 year ]
  • Incidence of all serious adverse events [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Official Title A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Brief Summary 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.
Detailed 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. The PCC will follow patients throughout their pregnancies and/or breastfeeding and infant through 1 year of age.

There will be 2 cohorts enrolled in the study. Cohort 1 will be pregnant and/or breastfeeding patients who have Fabry Disease, and have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding. Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.

This is an observational study, it will enroll patients and collect data as described in this protocol for a minimum of 10 years.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Pregnant and/or breastfeeding patients with Fabry disease
Condition Fabry Disease
Intervention Drug: migalastat
This is an observational study. Patients as described in Cohort 1 should have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding.
Study Groups/Cohorts
  • Cohort 1
    Cohort 1 will be pregnant and/or breastfeeding patients who have Fabry Disease, and have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding.
    Intervention: Drug: migalastat
  • Cohort 2
    Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: January 30, 2020)
20
Original Estimated Enrollment Same as current
Estimated Study Completion Date February 2030
Estimated Primary Completion Date August 2029   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

Female patients meeting the following criteria will be eligible for study enrollment:

  1. Patients with Fabry disease who are pregnant and/or breastfeeding, whether or not they are exposed to migalastat
  2. Able and willing to provide informed consent or assent, if applicable.
  3. Able and willing to provide HCP contact information.

Exclusion Criteria:

None

Sex/Gender
Sexes Eligible for Study: Female
Gender Based Eligibility: Yes
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Pregnancy Registry Call Center 888-239-0758 galafoldpregnancy@ubc.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04252066
Other Study ID Numbers AT1001-037
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Amicus Therapeutics
Study Sponsor Amicus Therapeutics
Collaborators Not Provided
Investigators Not Provided
PRS Account Amicus Therapeutics
Verification Date April 2020