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

Phase 3Active Not RecruitingNCT04910776

Sanofi17 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Glycogen Storage Disease Type II

Interventions

Eligibility

Sex: ALLMin age: 0 DaysMax age: 12 Months

Medical Language ↔ Plain English

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.

Locations (16)

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

...and 6 more locations

Outcomes

Primary Outcomes

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

Time frame: Week 52

Secondary Outcomes

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

Proportion of participants who are alive at Week 52

Time frame: Week 52

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

Time frame: at 12 and 18 months of age

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

Time frame: Week 52

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

Time frame: Week 52

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

Time frame: Week 52

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

Time frame: Week 52

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

Time frame: Week 52

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

Time frame: Week 52

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

Time frame: Week 52

Change from baseline to Week 52 in body length percentiles

Time frame: Week 52

Change from baseline to Week 52 in body weight percentiles

Time frame: Week 52

Change from baseline to Week 52 in head circumference percentiles

Time frame: Week 52

Change from baseline to Week 52 in urinary Hex4

Time frame: Week 52

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

Time frame: Week 52, Week 212

Number of participants with abnormalities in physical examinations

Time frame: Week 52, Week 208

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

Time frame: Week 52, Week 208

Number of participants with PCSA in vital signs measurements

Time frame: Week 52, Week 208

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

Time frame: Week 52, Week 208

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

Time frame: Week 52, Week 208

Plasma concentration of avalglucosidase alfa

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