Recruiting

Avalglucosidase Alfa

Sponsor:

Sanofi

Code:

NCT05734521

Conditions

Pompe Disease

Pregnancy

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Interventions

avalglucosidase alfa-NGPT (GZ402666) IV

avalglucosidase alfa-NGPT (GZ402666)

Study Details

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.

Conditions

Pompe Disease

Pregnancy

Study ID

NCT05734521

Start date

Oct 26, 2022

Status verified date

Jan, 2026

Completion date

Oct 31, 2032

Anticipated

Primary completion date

Oct 31, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

100 participants

Anticipated

Interventions and Outcome Measures

Arms

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

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

Interventions

avalglucosidase alfa-NGPT (GZ402666) IV

intravenous infusion

avalglucosidase alfa-NGPT (GZ402666)

exposed via pregnancy and lactation

Primary outcome measure

  • Maternal complications [ Time Frame: through study completion, an average of 10 years ]
  • Pregnancy outcome [ Time Frame: through study completion, an average of 10 years ]
  • Infant outcome [ Time Frame: through study completion, an average of 10 years ]

Central Contacts and Locations

Central contacts

Trial Transparency email recommended (Toll free for US & Canada)

800-633-1610Contact-US@sanofi.com

Locations

Investigational site worldwide

Recruiting

Bridgewater, New Jersey, United States, 08807

More Information

Sponsor

Sanofi

Last update posted

Jan 16, 2026

Last verified

Jan, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Sanofi on 2026-01-16.