NL-OMON52076Not yet recruitingPhase 3
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) - baby-COMET
Conditions
Trial Snapshot
- Phase
- Phase 3
- Status
- Not yet recruiting
- Sponsor
- Genzyme Europe BV
- Enrollment
- 1
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 0 to 1 (—)
Inclusion Criteria
- •- Participants must have confirmed diagnosis of infantile-onset Pompe disease
- •defined as:
- •the presence of 2 lysosomal acid a-glucosidase (GAA) pathogenic variants and a
- •documented GAA deficiency from blood, skin, or muscle tissue; or the presence
- •pathogenic variant and a documented GAA deficiency from blood, skin and muscle
- •in 2 separate samples (from either 2 different tissues or from the same tissue
- •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, LVMI
- •equivalent to
- •mean age specific LVMI
- •+1 standard deviation for participants diagnosed by newborn screening or sibling
- •+2 standard deviation for participants diagnosed by clinical evaluation.
- •- Parents or legally authorized representative(s) must be capable of giving
- •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 enzyme-replacement therapy (ERT) with recombinant human
- •glucosidase (rhGAA) from any source.
- •- Participant who has previously been treated in any clinical trial of
- •avalglucosidase alfa.
- •- Participant not suitable for participation, whatever the reason, as judged by
- •Investigator, including medical or clinical conditions, or participants
- •potentially at risk of
- •noncompliance to study procedures.
Investigators
Similar Trials
Recruiting
Phase 1
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaGlycogen storage disease type IIMedDRA version: 20.1Level: PTClassification code: 10053185Term: Glycogen storage disease type II Class: 100000004850CTIS2024-513859-33-00Sanofi-Aventis Recherche & Developpement21
Active, not recruiting
Phase 1
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase AlfaGlycogen storage disease type IIMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2020-004686-39-BESanofi-Aventis Recherche & Développement18
Recruiting
Phase 3
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa2024-513859-33-00Sanofi-Aventis Recherche & Developpement8
Active, not recruiting
Phase 1
Clinical Study for IOPD Participants Less Than or Equal to 6 Months of age to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) WithAvalglucosidase Alfa(Baby-COMET)Glycogen storage disease type IIMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2020-004686-39-ITSANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT20
Active, not recruiting
Phase 1
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of enzyme replacement therapy (ERT) With Avalglucosidase AlfaGlycogen storage disease type IIMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2020-004686-39-DESanofi-Aventis Recherche & Développement18