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

Sanofi

Status and phase

Active, not recruiting

Phase 3

Conditions

Glycogen Storage Disease Type II

Treatments

Drug: avalglucosidase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT04910776

U1111-1246-6645 (Registry Identifier)

EFC14462

2020-004686-39 (EudraCT Number)

2024-513859-33 (Registry Identifier)

Details and patient eligibility

About

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

Full description

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

Enrollment

17 patients

Sex

All

Ages

Under 12 months old

Volunteers

No Healthy Volunteers

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.