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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT04910776
Other study ID # EFC14462
Secondary ID U1111-1246-66452
Status Recruiting
Phase Phase 3
First received
Last updated
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
Health authority
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.

Study Design


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
See also
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Completed NCT03687333 - Evaluate Efficacy and Safety in Chinese Patients With Infantile-Onset Pompe Disease With One Year Alglucosidase Alfa Treatment Phase 4