A Phase 4 interventional study of Avalglucosidase alfa (GZ402666) in Glycogen Storage Disease Type II, sponsored by Genzyme, a Sanofi Company. Active, not recruiting at 11 sites in France. Open to participants aged 6 Months and older. Per ClinicalTrials.gov, last updated 2026-09-09.
Sponsored by Genzyme, a Sanofi Company (part of Sanofi) · Phase 4, Interventional, and Treatment
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Among 10 studies testing avalglucosidase alfa in Glycogen Storage Disease Type II — 1 open to participants now, 5 under way, 1 of them phase 3. avalglucosidase alfa · Glycogen Storage Disease Type II.
Among the 10 studies testing avalglucosidase alfa in Glycogen Storage Disease Type II, this one is the 6th largest by enrollment, is one of 2 that started in 2022, is one of 3 in phase 4, and was the 8th registered.
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
Browse Glycogen Storage Disease Type II studies →1 of the 10 studies testing avalglucosidase alfa in Glycogen Storage Disease Type II is open now, in 1 country. Recruiting first, the newest first:
Genzyme, a Sanofi Company · Sites in China
Ages up to 17
By country, each count listing its studies:
avalglucosidase alfa: first FDA approval 2021 (NEXVIAZYME), first EMA approval 2022. What it is approved for, and how studies give it, is on its page →
Of the 23 Glycogen Storage Disease Type II studies recruiting now, 16 (70%) set no upper age limit and 6 (26%) stop at 75 or younger; 15 take children under 18; none take healthy volunteers.
This study: 6 Months and older · all sexes · no healthy volunteers.
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10 studies; the count open now beside each. This study's row is marked.
First in 2001; this study’s year is marked. 2 started in 2022.
This study's enrollment of 17 is below the median of 20 across 89 interventional studies indexed under Glycogen Storage Disease Type II.
Median enrollment 18 across the 10 interventional studies. Of the 5 that completed or were terminated, 3 (60%) have results posted.
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9 of 10 have an industry lead sponsor. The 4 most frequent:
This study is registered under Genzyme, a Sanofi Company, one of the names counted as Sanofi. Under that name alone:
Genzyme, a Sanofi Company is the lead sponsor of 308 studies on the registry; 5 are open to participants now.
Of its 25 completed or terminated interventional studies of FDA-regulated products, 19 (76%) have results posted.
41 studies in Glycogen Storage Disease Type II by Sanofi: 4 open now, 8 under way, 1 of them phase 3. Of 31 finished, 12 (39%) have results posted.
Among the 41 studies in Glycogen Storage Disease Type II by Sanofi, this one is the 11th largest by enrollment of the 29 interventional ones that give a count, is one of 2 that started in 2022, is one of 13 in phase 4, and was the 39th registered.
Sanofi’s Glycogen Storage Disease Type II studies by the year they started, since 2001.
1 of the studies testing avalglucosidase alfa in Glycogen Storage Disease Type II that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05164055Genzyme, a Sanofi CompanyAvalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) | 2027-06-30 |
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Among the 10 studies testing avalglucosidase alfa in Glycogen Storage Disease Type II, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among Sanofi’s 41 in Glycogen Storage Disease Type II:
Nothing recorded among them since 2026-09-25.
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This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to ERT with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until the conclusion of negotiations of avalglucosidase alfa in France under the standard reimbursement framework on each indication independently, infantile (IOPD) and late (LOPD), or until June 2027, whichever comes first.
- Study visit frequency: every 2 weeks
From market authorization until the conclusion of negotiations of avalglucosidase alfa in France under the standard reimbursement framework on each indication independently, infantile (IOPD) and late (LOPD), or until June 2027, whichever comes first.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Administered intravenously every other week
Drug: Avalglucosidase alfa (GZ402666)
Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion
Also known as: Nexviadyme®
Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months
Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants
The primary measurement is the distance in meters walked by the participant on a flat, hard surface in 6 minutes. The distance walked in meters will be recorded and the corresponding percent predicted value will be calculated. The greater the distance (that a participant could walk in 6 minutes), the greater the endurance.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Assessment of quick motor function test (QMFT) for LOPD participants
The QMFT is an observer administered test to evaluate changes in motor function. QMFT comprises of 16 items specifically difficult for participants with Pompe disease. Each item is scored separately on a 5-point ordinal scale (ranged from 0 to 4, higher score indicated better outcome). Total QMFT score is obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), higher score represented better outcome.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position, also tested in supine position. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) \* 100. Maximum Inspiratory Pressure (MIP) is a quick and non-invasive test to measure strength of inspiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MIP refers to how much air pressure force an individual creates by inhaling through the mouth as hard as possible. Maximum Expiratory Pressure (MEP) is a quick and non-invasive test to measure strength of expiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MEP is the greater pressure generated during maximal expiration.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants
SF-12, a 12 item-questionnaire, used to assess health-related quality of life in participants aged \>=18 years at screening/baseline. SF-12 consisted of 12 items, which were categorized into eight domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health), higher scores indicated good health condition. These eight domains were further summarized into 2 summary scores, physical component summary (PCS) and mental component summary (MCS). The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), higher scores indicated a better health-related quality of life.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants
The 24-hour recall PDSS (V1.1) is a self-administered questionnaires specifically designed to capture the symptoms impacts relevant to patients with LOPD. The PDSS includes 12 questions with responses on a scale from 0 (none) to 10 (as bad as I can imagine) The data from PDSS scale will be analyzed separately and as a composite with PDIS scale.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants
The 24-hour recall PDIS (V1.2) is a self-administered questionnaires specifically designed to capture disease impacts relevant to patients with LOPD. The PDIS includes 15 questions with varying scales implemented depending on question type. The data from PDIS scale will be analyzed separately and as a composite with PDSS scale.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants
The Pompe-PEDI consists of a Functional Skills Scale and a Caregiver Assistance Scale. Both scales have 3 domains: self-care; mobility; and social function. The Mobility Domain was selected to measure change in mobility secondary to changes in muscle strength. The domain consists of 160 mobility items.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
PedsQL score for IOPD participants
The 23item PedsQL Generic Core Scale encompasses 4 subscales including physical, emotional, school, and social functionings. Scores are transformed to a 0-100 scale, higher scores indicate better HRQOL. Infant scale will not be used since this is a scale for up to 24 months of age and patients are older now than that.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Left Ventricular Mass Index (LVMI) Z-score in IOPD participants
Left Ventricular Mass Index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations.
Time frame: From study enrolment to the final study visit/telephone contact, up to 4 years and 11 months (at enrolment and every 6 months)
Plan to share: 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
No publications or documents are linked to this record.
Started Jul 2022 (actual). Primary completion is estimated for Jun 2027, 59 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Avalglucosidase alfa
Glycogen Storage Disease Type II →