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临床试验/NCT05734521
NCT05734521
招募中
不适用

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

Sanofi1 个研究点 分布在 1 个国家目标入组 100 人2022年10月26日

概览

阶段
不适用
干预措施
avalglucosidase alfa-NGPT (GZ402666) IV
疾病 / 适应症
Pompe Disease
发起方
Sanofi
入组人数
100
试验地点
1
主要终点
Pregnancy outcome
状态
招募中
最后更新
3个月前

概览

简要总结

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.

详细描述

Study Design Time Perspective: Retrospective and Prospective

注册库
clinicaltrials.gov
开始日期
2022年10月26日
结束日期
2032年10月31日
最后更新
3个月前
研究类型
Observational
性别
Female

研究者

发起方
Sanofi
责任方
Sponsor

入排标准

入选标准

  • 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.

排除标准

  • 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.

研究组 & 干预措施

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

干预措施: avalglucosidase alfa-NGPT (GZ402666) IV

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

干预措施: avalglucosidase alfa-NGPT (GZ402666)

结局指标

主要结局

Pregnancy outcome

时间窗: 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

Infant outcome

时间窗: 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.

Maternal complications

时间窗: through study completion, an average of 10 years

Prevalence of pregnancy/labor/delivery/postpartum maternal complications

研究点 (1)

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