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Avalglucosidase Alfa Pregnancy Study

Sponsor
Sanofi (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05734521
Collaborator
(none)
100
Enrollment
1
Location
119.2
Anticipated Duration (Months)
0.8
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation, to assess the risks of avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.

  • Outcomes in exposed infants, including growth and development, will be assessed through at least the first year of life.

  • Data will be collected for approximately 10 years.

Condition or Disease Intervention/Treatment Phase
  • Biological: avalglucosidase alfa-NGPT (GZ402666) IV
  • Biological: avalglucosidase alfa-NGPT (GZ402666)

Detailed Description

Study Design Time Perspective: Retrospective and Prospective

Study Design

Study Type:
Observational
Anticipated Enrollment :
100 participants
Observational Model:
Cohort
Time Perspective:
Other
Official Title:
A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting
Actual Study Start Date :
Oct 26, 2022
Anticipated Primary Completion Date :
Oct 1, 2032
Anticipated Study Completion Date :
Oct 1, 2032

Arms and Interventions

Arm Intervention/Treatment
Pregnant women exposed to avalglucosidase alfa

Pregnant women with a confirmed diagnosis of Pompe disease and avalglucosidase alfa exposure during the pregnancy and/or lactation

Biological: avalglucosidase alfa-NGPT (GZ402666) IV
intravenous infusion
Other Names:
  • NEXVIAZYME

    		•
    Infants born to mother/father exposed to avalglucosidase alfa

    Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa

    Biological: avalglucosidase alfa-NGPT (GZ402666)
    exposed via pregnancy and lactation
    Other Names:
  • NEXVIAZYME

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Maternal complications [through study completion, an average of 10 years]

      Prevalence of pregnancy/labor/delivery/postpartum maternal complications

    2. Pregnancy outcome [through study completion, an average of 10 years]

      Prevalence of live births, spontaneous abortions (<20 weeks of gestation), elective terminations, ectopic pregnancies, early fetal deaths (20 to 27 weeks of gestation), late fetal deaths (≥28 weeks of gestation), stillbirths and maternal deaths

    3. Infant outcome [through study completion, an average of 10 years]

      Number of occurrences of major congenital anomalies, neonatal deaths, development delays (growth, motor, neurologic, behavioral) through first year of life. Major congenital anomalies defined using the US Centers for Disease Control and Prevention (CDC) and growth and development assessments using CDC's Developmental Milestones 2021.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    Female
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation for whom an Individual Case Safety Report (ICSR) has been submitted to the Sanofi Global Pharmacovigilance (PV) department, and/or,

    • Women exposed to avalglucosidase alfa during pregnancy and/or lactation who have provided informed consent to enroll in the Pompe Pregnancy Sub-registry.

    Exclusion Criteria:

    There are no exclusion criteria in this study.

    The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Investigational site worldwide Bridgewater New Jersey United States 08807

    Sponsors and Collaborators

    • Sanofi

    Investigators

    • Study Director: Clinical Sciences & Operations, Sanofi

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Sanofi
    ClinicalTrials.gov Identifier:
    NCT05734521
    Other Study ID Numbers:
    • OBS17641
    First Posted:
    Feb 21, 2023
    Last Update Posted:
    Feb 21, 2023
    Last Verified:
    Feb 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Glycogen Storage Disease Type II

    Study Results

    No Results Posted as of Feb 21, 2023