Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

Running, not enrolling · Phase 3

Conditions studied: Glycogen Storage Disease Type II

In brief

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: * Study duration: Screening - up to 4 weeks; * Primary Analysis Period (PAP) - 52 weeks; * Extended Treatment Period (ETP) - 52 weeks; * Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. * Treatment duration: Up to 4 years * Visit frequency: every other week and potentially every week

Key facts

Study ID

NCT04910776

Run by

Sanofi

People needed

17

Starts

2021-09-01

Expected to finish

2027-08-10

Last updated by the study team

2026-04-08

Who can join

Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participants must have confirmed diagnosis of infantile-onset Pompe disease defined as: the presence of 2 lysosomal acid α-glucosidase (GAA) pathogenic variants and a documented GAA deficiency from blood, skin, or muscle tissue; or the presence of 1 GAA pathogenic variant and a documented GAA deficiency from blood, skin and muscle tissue in 2 separate samples (from either 2 different tissues or from the same tissue but at 2 different sampling dates).
• Participants must have established cross-reactive immunological material (CRIM) status available prior to enrollment.
• Participants must have cardiomyopathy at the time of diagnosis: ie, left ventricular mass index (LVMI) equivalent to mean age specific LVMI
• +1 standard deviation for participants diagnosed by newborn screening or sibling screening;
• +2 standard deviation for participants diagnosed by clinical evaluation.
• Parents or legally authorized representative(s) must be capable of giving signed informed consent.

You may not qualify if…

• Participants with symptoms of respiratory insufficiency, including any ventilation use (invasive or noninvasive) at the time of enrollment.
• Participants with major congenital abnormality.
• Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).
• Participant received any Pompe disease specific treatment, eg enzyme-replacement gene therapy (ERT).
• Participant who has previously been treated in any clinical trial of avalglucosidase alfa.
• Participant 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.

Where it is running

• Stanford Hospital- Site Number : 8400006 — Stanford, California, United States
• Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 — Minneapolis, Minnesota, United States
• Advanced Medical Genetics- Site Number : 8400002 — Hawthorne, New York, United States
• Duke University Medical Center- Site Number : 8400004 — Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center- Site Number : 8400001 — Cincinnati, Ohio, United States
• Seattle Children's Hospital- Site Number : 8400003 — Seattle, Washington, United States
• Investigational Site Number : 0560001 — Leuven, Belgium
• Investigational Site Number : 1560002 — Qingdao, China
• Investigational Site Number : 1560001 — Shanghai, China
• Investigational Site Number : 2760001 — Bad Oeynhausen, Germany
• Investigational Site Number : 3800002 — Monza, Lombardy, Italy
• Investigational Site Number : 5280001 — Rotterdam, Netherlands
• Investigational Site Number : 7240001 — Esplugues de Llobregat, Catalunya [Cataluña], Spain
• Investigational Site Number : 1580001 — Taipei, Taiwan
• Investigational Site Number : 8260001 — London, London, City of, United Kingdom
• Investigational Site Number : 8260002 — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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