Glycogen Storage Disease Type IIPhase 4NCT06666413
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
A Phase 4 study testing Avalglucosidase alfa in people with Glycogen Storage Disease Type II and Pompe's Disease, run by Genzyme, a Sanofi Company at 1 site in China.
Trial summary
This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Glycogen Storage Disease Type II, Pompe's Disease
- What is being tested?
- Avalglucosidase alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteChina
- Study dates
- 7 May 2025to 2 May 2028
- Phase
- Phase 4
- 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 Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participant must be <18 years of age, at the time of signing the informed consent.
- Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
- Participants must have documented cardiomyopathy at the time of diagnosis.
- Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.
- Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:
- Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.
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- Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
- Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.
- Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.
- Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.
- Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Who cannot join
None listed.
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
China1 site, 1 recruiting
- Recruiting
Investigational Site Number: 1560001
Shanghai, 200127
About this study
Official title: A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)
This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.
Study details include:
- The study duration: total study duration is approximately 64 weeks.
- Screening period of up to 8 weeks
- Treatment period of 52 weeks
- Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)
- The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
- Conditions
- Glycogen Storage Disease Type II, Pompe's Disease
- Being tested
- Avalglucosidase alfa
- 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 alfaDrug
Also called GZ402666, Nexviazyme
Pharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion
| Study group | Role | What they receive |
|---|---|---|
| avalglucosidase alfa | Experimental | Avalglucosidase alfa will be administered by IV infusion following reconstitution and dilution at a dose of 20 mg/kg body weight qow Receives: Drug: Avalglucosidase alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs, SAEs, AESIs including IARs during the TE period
Measured: From baseline to Week 56
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
Measured: From baseline to Week 56
Secondary outcomes (7)
Proportion of participants who are alive and free of invasive ventilation at Week 52
Measured: At Week 52
Change from baseline to Week 52 in LVM Z score evaluated by echocardiography
Measured: From baseline to Week 52
The absolute change in body growth Z scores from baseline to Week 52
Measured: From baseline to Week 52
The percentiles of the body growth Z scores changes from baseline to Week 52
Measured: From baseline to Week 52
Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)
Measured: From baseline to Week 52
Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)
Measured: From baseline to Week 52
Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52
Measured: At Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2024
- Study start7 May 2025
- Primary completion2 May 2028 (estimated)
- Study completion2 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Glycogen Storage Disease Type IIStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genzyme, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06666413
- Sponsor's protocol ID
- LPS18086
- Other IDs
- U1111-1295-2947
- Status last verified
- Mar 2026
- First submitted
- 16 Oct 2024
- Last updated
- 23 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- 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 23 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)
- ClinicalTrials.gov ID
- NCT06666413
- Acronym / sponsor ID
- LPS18086
- Conditions
- Glycogen Storage Disease Type II, Pompe's Disease
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 13 participants (estimated)
- Lead sponsor
- Genzyme, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Study start
- 7 May 2025 (actual)
- Primary completion
- 2 May 2028 (estimated)
- Study completion
- 2 May 2028 (estimated)
- First posted
- 30 Oct 2024 (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 single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.
Study details include:
- The study duration: total study duration is approximately 64 weeks.
- Screening period of up to 8 weeks
- Treatment period of 52 weeks
- Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)
- The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| avalglucosidase alfa | Experimental | Drug: Avalglucosidase alfaAvalglucosidase alfa will be administered by IV infusion following reconstitution and dilution at a dose of 20 mg/kg body weight qow |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Participant must be <18 years of age, at the time of signing the informed consent.
- Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.
- Participants must have documented cardiomyopathy at the time of diagnosis.
- Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent.
- Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply:
Key exclusion criteria (0 listed)
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 alfaAlso: GZ402666, NexviazymePharmaceutical form: Sterile lyophilized powder Route of administration: IV infusion | Drug | avalglucosidase alfa |
Primary outcome
Incidence of AEs, SAEs, AESIs including IARs during the TE period
Time frame: From baseline to Week 56
Abnormality in clinical laboratory, vital signs, and ECG parameters during the TE period
Time frame: From baseline to Week 56
7 secondary outcomes
Proportion of participants who are alive and free of invasive ventilation at Week 52
Time frame: At Week 52
Change from baseline to Week 52 in LVM Z score evaluated by echocardiography
Time frame: From baseline to Week 52
The absolute change in body growth Z scores from baseline to Week 52
Time frame: From baseline to Week 52
The percentiles of the body growth Z scores changes from baseline to Week 52
Time frame: From baseline to Week 52
Change from baseline to Week 52 in distance walked during six-minute walk test (6MWT) (in applicable participants)
Time frame: From baseline to Week 52
Change from baseline to Week 52 in quick motor function test (QMFT) score (in applicable participants)
Time frame: From baseline to Week 52
Number of motor development milestones achieved as evaluated by Motor Milestone Checklist at Week 52
Time frame: At Week 52
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 23 Mar 2026.
- LPS18086 Plain Language Results SummaryLink provided by the sponsor.
- 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
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Avalglucosidase alfa in people with Glycogen Storage Disease Type II and Pompe's Disease, run by Genzyme, a Sanofi Company at 1 site in China.
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 12 inclusion and 0 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 23 Mar 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 China. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Incidence of AEs, SAEs, AESIs including IARs during the TE period (time frame: From baseline to Week 56). The study lists 2 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 23 Mar 2026.