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NCT Number: NCT04910776

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Glycogen Storage Disease Type II Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glycogen Storage Disease Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

0 day–12 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Investigational Site Number : 0560001, Leuven, Belgium

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About this study

Study duration may be variable by country, including at least completion of the PAP and ETP, and up to 4.08 years.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

Avalglucosidase alfa

Drug

Sterile lyophilized powder intravenous (IV) infusion

Other names: GZ402666

Primary outcomes

  1. Proportion of participants who are alive and free of invasive ventilation at Week 52

    Time frame: Week 52

Secondary outcomes

  1. Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age

    Time frame: at 12 and 18 months of age

  2. Proportion of participants who are alive at Week 52

    Time frame: Week 52

  3. Proportion of participants who are alive at 12 and 18 months of age

    Time frame: at 12 and 18 months of age

  4. Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52

    Time frame: Week 52

  5. Proportion of participants who are free of supplemental oxygen use at Week 52

    Time frame: Week 52

  6. Change from baseline to Week 52 in left ventricular mass (LVM)-Z score

    Time frame: Week 52

  7. Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score

    Time frame: Week 52

  8. Change from baseline to Week 52 in body length Z-scores

    Time frame: Week 52

  9. Change from baseline to Week 52 in body weight Z-scores

    Time frame: Week 52

  10. Change from baseline to Week 52 in head circumference Z-scores

    Time frame: Week 52

  11. Change from baseline to Week 52 in body length percentiles

    Time frame: Week 52

  12. Change from baseline to Week 52 in body weight percentiles

    Time frame: Week 52

  13. Change from baseline to Week 52 in head circumference percentiles

    Time frame: Week 52

  14. Change from baseline to Week 52 in urinary Hex4

    Time frame: Week 52

  15. Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)

    Time frame: Week 52, Week 212

  16. Number of participants with abnormalities in physical examinations

    Time frame: Week 52, Week 208

  17. Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results

    Time frame: Week 52, Week 208

  18. Number of participants with PCSA in vital signs measurements

    Time frame: Week 52, Week 208

  19. Number of participants with PCSA in 12-lead electrocardiogram (ECG)

    Time frame: Week 52, Week 208

  20. Incidence of treatment-emergent anti-drug antibodies (ADA)

    Time frame: Week 52, Week 208

  21. Plasma concentration of avalglucosidase alfa

    Time frame: at Day 1, Week 12, and Week 52

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)

Acronym: Baby-COMET

Important dates

Study start
2021
Primary completion
2026
Study completion
2027
First posted
Jun 2, 2021
Registry last updated
Apr 8, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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