Avalglucosidase alfa French post-trial access for participants with Pompe disease

A French multicenter Phase 4 open label extension study of long term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France - PTA Avalglucosidase

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-002590-26-FR

Enrollment

Registered

2021-08-16

Start date

Unknown

Completion date

Unknown

Last updated

2024-08-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Glycogen storage disease type II MedDRA version: 20.1 Level: PT Classification code 10053185 Term: Glycogen storage disease type II System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: Avalglucosidase alfa Product Code: GZ402666 Pharmaceutical Form: Powder for concentrate for solution for infusion INN or Proposed INN: Avalglucosidase Alfa Current Sponsor code: GZ402666

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Patient with Pompe Disease who has previously completed Study EFC14028, LTS13769, or ACT14132 of avalglucosidase alfa studies in France. - The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if =65 years) yes F.1.3.1 Number of subjects for this age range 1

Exclusion criteria

Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: - Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients. - The patient is concurrently participating in another clinical study of investigational treatment. - The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatobiliary, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess long-term safety in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until January 2023, whichever comes first.;Secondary Objective: To assess long-term efficacy in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until January 2023, whichever comes first.;Primary end point(s): 1/ Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death 2/ Number of participants with abnormalities in physical examinations 3/ Number of participants with abnormalities in vital signs measurements 4/ Number of participants with abnormalities in clinical laboratory results 5/ Number of participants with abnormalities in 12- lead electrocardiogram (ECG) 6/ Incidence of treatment-emergent anti-drug antibodies (ADA);Timepoint(s) of evaluation of this end point: For all items : From baseline until reimbursement or January 2023, whichever comes first	—

Measure

Time frame

Main Objective: To assess long-term safety in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until January 2023, whichever comes first.;Secondary Objective: To assess long-term efficacy in patients in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until January 2023, whichever comes first.;Primary end point(s): 1/ Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death 2/ Number of participants with abnormalities in physical examinations 3/ Number of participants with abnormalities in vital signs measurements 4/ Number of participants with abnormalities in clinical laboratory results 5/ Number of participants with abnormalities in 12- lead electrocardiogram (ECG) 6/ Incidence of treatment-emergent anti-drug antibodies (ADA);Timepoint(s) of evaluation of this end point: For all items : From baseline until reimbursement or January 2023, whichever comes first

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Secondary

Measure	Time frame
Secondary end point(s): 1/ Evolution of clinical status for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants 2/ Assessment of six-minute walk test (distance in meters and % predicted value) for LOPD and IOPD participants 3/ Assessment of quick motor function test (QMFT) for LOPD participants 4/ Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants 5/ Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants 6/ Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants 7/ Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants 8/ Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants 9/ PedsQL score for IOPD participants 10/ Left Ventricular Mass Index (LVMI) Z-score in IOPD participants ;Timepoint(s) of evaluation of this end point: For all items : Every 6 months, from baseline until reimbursement or January 2023, whichever comes first	—

Measure

Time frame

Secondary end point(s): 1/ Evolution of clinical status for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants 2/ Assessment of six-minute walk test (distance in meters and % predicted value) for LOPD and IOPD participants 3/ Assessment of quick motor function test (QMFT) for LOPD participants 4/ Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants 5/ Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants 6/ Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants 7/ Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants 8/ Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants 9/ PedsQL score for IOPD participants 10/ Left Ventricular Mass Index (LVMI) Z-score in IOPD participants ;Timepoint(s) of evaluation of this end point: For all items : Every 6 months, from baseline until reimbursement or January 2023, whichever comes first

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Countries

France

Contacts

Public ContactDirection des Opérations cliniques

Sanofi-aventis France

Public-Registry-MA-France@sanofi.com0 800 222 555

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026