Pompe DiseaseNCT05734521
Avalglucosidase Alfa Pregnancy Study
An observational study of Pompe Disease and Pregnancy, run by Sanofi at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Pompe Disease, Pregnancy
- What is being tested?
- avalglucosidase alfa-NGPT (GZ402666) IV and avalglucosidase alfa-NGPT (GZ402666)
- Type of treatment
- Injection or infusion
- How many people
- 100 participants
- Where
- 1 siteUnited States
- Study dates
- 26 Oct 2022to 31 Oct 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Trial Transparency email recommended (Toll free for US & Canada) contact
United States1 site, 1 recruiting
- Recruiting
Investigational site worldwide
Bridgewater, New Jersey, 08807
About this study
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
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.
Read the detailed description
Study Design Time Perspective: Retrospective and Prospective
- Conditions
- Pompe Disease, Pregnancy
- Being tested
- avalglucosidase alfa-NGPT (GZ402666) IV, avalglucosidase alfa-NGPT (GZ402666)
- Medical subject headings
- Glycogen Storage Disease Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
avalglucosidase alfa-NGPT (GZ402666) IVBiological
Also called NEXVIAZYME
intravenous infusion
avalglucosidase alfa-NGPT (GZ402666)Biological
Also called NEXVIAZYME
exposed via pregnancy and lactation
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Biological: 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 Receives: Biological: avalglucosidase alfa-NGPT (GZ402666) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maternal complications
Prevalence of pregnancy/labor/delivery/postpartum maternal complications
Measured: through study completion, an average of 10 years
Pregnancy outcome
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
Measured: through study completion, an average of 10 years
Infant outcome
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.
Measured: through study completion, an average of 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2023
- Study start26 Oct 2022
- Primary completion31 Oct 2032 (estimated)
- Study completion31 Oct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pompe DiseaseStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05734521
- Sponsor's protocol ID
- OBS17641
- Status last verified
- Jan 2026
- First submitted
- 3 Feb 2023
- Last updated
- 16 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Study at a glance
From the registry record, last updated by the sponsor on 16 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05734521
- Acronym / sponsor ID
- OBS17641
- Conditions
- Pompe Disease, Pregnancy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 26 Oct 2022 (actual)
- Primary completion
- 31 Oct 2032 (estimated)
- Study completion
- 31 Oct 2032 (estimated)
- First posted
- 21 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Design Time Perspective: Retrospective and Prospective
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pregnant women exposed to avalglucosidase alfa | – | Biological: avalglucosidase alfa-NGPT (GZ402666) IVPregnant 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 | – | Biological: avalglucosidase alfa-NGPT (GZ402666)Infants born to mother/father with a confirmed diagnosis of Pompe disease and exposed to avalglucosidase alfa |
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Women and their offspring exposed to avalglucosidase alfa during pregnancy and/or lactation
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| avalglucosidase alfa-NGPT (GZ402666) IVAlso: NEXVIAZYMEintravenous infusion | Biological | Pregnant women exposed to avalglucosidase alfa |
| avalglucosidase alfa-NGPT (GZ402666)Also: NEXVIAZYMEexposed via pregnancy and lactation | Biological | Infants born to mother/father exposed to avalglucosidase alfa |
Primary outcome
Maternal complications
Time frame: through study completion, an average of 10 years
Prevalence of pregnancy/labor/delivery/postpartum maternal complications
Pregnancy outcome
Time frame: 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
Time frame: 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jan 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Related trials for Pompe Disease
RecruitingObservational study
Pompe Disease Registry Protocol
Glycogen Storage Disease Type II, Pompe Disease
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingPhase 1/2
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)
Pompe Disease (Late-onset), Pompe Disease Late-Onset
Not yet recruitingPhase 2
An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)
Pompe Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Trial Transparency email recommended (Toll free for US & Canada) contact
Clinical Sciences & Operations study director
Sanofi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pompe Disease and Pregnancy, run by Sanofi at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Transparency email recommended (Toll free for US & Canada) (800-633-1610 ext. option 6, Contact-US@sanofi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maternal complications (time frame: through study completion, an average of 10 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jan 2026.