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

Glycogen Storage Disease Type II Clinical Trial

— Baby-COMET  

Official 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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04910776
• Other study ID #: EFC14462
• Secondary ID: U1111-1246-66452
• Status: Recruiting
• Phase: Phase 3
• Start date: September 1, 2021
• Est. completion date: August 28, 2026

Study information

• Verified date: May 2024
• Source: Sanofi
• Contact: Trial Transparency email recommended (Toll free number for US &
• Phone: 800-633-1610
• Email: Contact-US[@]sanofi.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 18
• Est. completion date: August 28, 2026
• Est. primary completion date: December 27, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Days to 12 Months

Eligibility

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 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.

Related Conditions & MeSH terms

• Glycogen Storage Disease
• Glycogen Storage Disease Type II

Intervention

Drug:
• avalglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion

Locations

Country	Name	City	State
Belgium	Investigational Site Number : 0560001	Leuven
China	Investigational Site Number : 1560002	Qingdao
China	Investigational Site Number : 1560001	Shanghai
Germany	Investigational Site Number : 2760001	Gießen
Italy	Investigational Site Number : 3800001	Firenze
Italy	Investigational Site Number : 3800002	Monza	Lombardia
Netherlands	Investigational Site Number : 5280001	Rotterdam
Spain	Investigational Site Number : 7240001	Esplugues de Llobregat	Catalunya [Cataluña]
Taiwan	Investigational Site Number : 1580001	Taipei
United Kingdom	Investigational Site Number : 8260001	London	London, City Of
United Kingdom	Investigational Site Number : 8260002	Manchester
United States	Cincinnati Children's Hospital Medical Center - PIN Site Number : 8400001	Cincinnati	Ohio
United States	Duke University Medical Center Site Number : 8400004	Durham	North Carolina
United States	Advanced Medical Genetics Site Number : 8400002	Hawthorne	New York
United States	Seattle Childrens Hospital and Regional Medical Center Site Number : 8400003	Seattle	Washington
United States	Stanford University Site Number : 8400006	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Countries where clinical trial is conducted

United States,  Belgium,  China,  Germany,  Italy,  Netherlands,  Spain,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of participants who are alive and free of invasive ventilation at Week 52		Week 52
Secondary	Proportion of participants who are alive and free of invasive ventilation at 12 and 18 months of age		at 12 and 18 months of age
Secondary	Proportion of participants who are alive at Week 52		Week 52
Secondary	Proportion of participants who are alive at 12 and 18 months of age		at 12 and 18 months of age
Secondary	Proportion of participants who are free of ventilator use (invasive and non-invasive separate and combined) at Week 52		Week 52
Secondary	Proportion of participants who are free of supplemental oxygen use at Week 52		Week 52
Secondary	Change from baseline to Week 52 in left ventricular mass (LVM)-Z score		Week 52
Secondary	Change from baseline to Week 52 in Alberta Infant Motor Scale (AIMS) score		Week 52
Secondary	Change from baseline to Week 52 in body length Z-scores		Week 52
Secondary	Change from baseline to Week 52 in body weight Z-scores		Week 52
Secondary	Change from baseline to Week 52 in head circumference Z-scores		Week 52
Secondary	Change from baseline to Week 52 in body length percentiles		Week 52
Secondary	Change from baseline to Week 52 in body weight percentiles		Week 52
Secondary	Change from baseline to Week 52 in head circumference percentiles		Week 52
Secondary	Change from baseline to Week 52 in urinary Hex4		Week 52
Secondary	Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR)		Week 52, Week 212
Secondary	Number of participants with abnormalities in physical examinations		Week 52, Week 208
Secondary	Number of participants with potentially clinically significant abnormality (PCSA) in clinical laboratory results		Week 52, Week 208
Secondary	Number of participants with PCSA in vital signs measurements		Week 52, Week 208
Secondary	Number of participants with PCSA in 12-lead electrocardiogram (ECG)		Week 52, Week 208
Secondary	Incidence of treatment-emergent anti-drug antibodies (ADA)		Week 52, Week 208
Secondary	Plasma concentration of avalglucosidase alfa		at Day 1, Week 12, and Week 52