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

NCT ID: NCT04910776

Last Updated: 2025-03-13

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-01
• Study Completion Date: 2027-08-10

Brief 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

Detailed Description

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

Conditions

Glycogen Storage Disease Type II

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Avalglucosidase alfa

Administered intravenously every 2 weeks

Group Type: EXPERIMENTAL

avalglucosidase alfa

Intervention Type: DRUG

Sterile lyophilized powder intravenous (IV) infusion

Interventions

avalglucosidase alfa

Sterile lyophilized powder intravenous (IV) infusion

Intervention Type: DRUG

Other Intervention Names

GZ402666

Eligibility Criteria

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.

• Minimum Eligible Age: 0 Days
• Maximum Eligible Age: 12 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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

Investigational Site Number : 3800002

Monza, Lombardy, Italy

Investigational Site Number : 5280001

Rotterdam, , Netherlands

Investigational Site Number : 7240001

Esplugues de Llobregat, Catalunya [Cataluña], Spain

Investigational Site Number : 1580001

Taipei, , Taiwan

Investigational Site Number : 8260001

London, London, City of, United Kingdom

Investigational Site Number : 8260002

Manchester, , United Kingdom

Countries

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

Other Identifiers

U1111-1246-6645

Identifier Type: REGISTRY

Identifier Source: secondary_id

2024-513859-33

Identifier Type: REGISTRY

Identifier Source: secondary_id

2020-004686-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EFC14462

Identifier Type: -

Identifier Source: org_study_id