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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

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.

Condition or disease Intervention/treatment
Fabry Disease Drug: 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.

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Actual Study Start Date : April 17, 2020
Estimated Primary Completion Date : August 2029
Estimated Study Completion Date : February 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Breastfeeding

Group/Cohort Intervention/treatment
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.
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.

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.



Primary Outcome Measures :
  1. Number of major birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]

Secondary Outcome Measures :
  1. Incidence of spontaneous abortion [ Time Frame: : up to 20 weeks ]
  2. Number of elective or induced abortion [ Time Frame: Through the pregnancy, an average of 40 weeks ]
  3. Number of fetal death or stillbirth [ Time Frame: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week ]
  4. Number of live birth [ Time Frame: at the delivery, an average of 40 weeks of pregnancy ]
  5. Number of neonatal death [ Time Frame: up to 28 days of neonatal life ]
  6. Number of minor birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
  7. 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.

  8. Neurodevelopmental problems [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]
  9. 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 ]
  10. Number of obstetric and delivery complications [ Time Frame: At the delivery, an average of 40 weeks of pregnancy ]
  11. Occurrence of Jaundice cases in Infants [ Time Frame: Up to 1 year ]
  12. Number of hospitalizations in infants [ Time Frame: Up to 1 year ]
  13. Mortality in Infants [ Time Frame: Up to 1 year ]
  14. Head circumference in Infants (cm) [ Time Frame: Up to 1 year ]
  15. Weight in Infants (kilograms) [ Time Frame: Up to 1 year ]
  16. Length in Infants (cm) [ Time Frame: Up to 1 year ]
  17. Occurrence of milk allergic reaction in breastfed or formula supplemented infants [ Time Frame: Up to 1 year ]
  18. Occurrence of allergic reaction in patients who are breastfeeding [ Time Frame: Up to 1 year ]
  19. Frequency of adverse events effecting lactation [ Time Frame: Up to 1 year ]
  20. Incidence of all serious adverse events [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Pregnant and/or breastfeeding patients with Fabry disease
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04252066


Contacts
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Contact: Pregnancy Registry Call Center 888-239-0758 galafoldpregnancy@ubc.com

Locations
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United States, New Jersey
Amicus Therapeutics, Inc. Pregnancy Registry Recruiting
Cranbury, New Jersey, United States, 08512
Sponsors and Collaborators
Amicus Therapeutics
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04252066    
Other Study ID Numbers: AT1001-037
First Posted: February 5, 2020    Key Record Dates
Last Update Posted: April 28, 2020
Last Verified: April 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amicus Therapeutics:
migalastat
Fabry Disease
Women with Fabry Disease
Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders