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Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

NCT ID: NCT05164055

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE4

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-11

Study Completion Date

2026-09-30

Brief Summary

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This long-term open label safety and efficacy study is intended to follow up, and to provide post-trial access to enzyme replacement therapy (ERT) with avalglucosidase alfa to patients with Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first.

\- Study visit frequency: every 2 weeks

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Detailed Description

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Treatment duration approximately 4 years and 3 months: until reimbursement of avalglucosidase alfa in France or until September 2026, whichever comes first

Conditions

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Glycogen Storage Disease Type II

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Avalglucosidase alfa

Administered intravenously every other week

Group Type EXPERIMENTAL

Avalglucosidase alfa (GZ402666)

Intervention Type DRUG

Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Interventions

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Avalglucosidase alfa (GZ402666)

Pharmaceutical form: Sterile lyophilized powder Route of administration: intravenous (IV) infusion

Intervention Type DRUG

Other Intervention Names

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Nexviadyme®

Eligibility Criteria

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Inclusion Criteria

* Patient with LOPD or IOPD who has previously completed Study EFC14028, LTS13769, or ACT14132 in France, and reimbursement for avalglucosidase alfa is not yet granted in France.
* The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if \<18 years of age, is willing to provide assent if deemed able to do so.
* The patient (and patient's legal guardian if patient is \<18 years of age) must have the ability to comply with the clinical protocol.
* The patient, if female and of childbearing potential, must have a negative pregnancy test result \[urine beta-human chorionic gonadotropin (β-HCG)\] at enrollment.
* Sexually active female patients of childbearing potential and male patients are required to practice true abstinence in line with their preferred and usual lifestyle or to use 2 acceptable effective methods of contraception.

Exclusion Criteria

* Patient with life-threatening hypersensitivity (anaphylactic reaction) to one of avalglucosidase alfa's excipients.
* Patient who permanently discontinued avalglucosidase alfa in a previous clinical study
* Pregnant or breastfeeding female patient
* The patient is concurrently participating in another clinical study of investigational treatment.
* The patient, in opinion of the Investigator, is unable to comply with the requirements of the study.
* 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.
* Individuals accommodated in an institution because of regulatory or legal order; prisoners, or patients who are legally institutionalized.

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Minimum Eligible Age

6 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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Investigational Site Number : 2500004

Bordeaux, , France

Site Status

Investigational Site Number : 2500005

Brest, , France

Site Status

Investigational Site Number : 2500008

Clermont-Ferrand, , France

Site Status

Investigational Site Number : 2500009

Lille, , France

Site Status

Investigational Site Number : 2500003

Lyon, , France

Site Status

Investigational Site Number : 2500001

Marseille, , France

Site Status

Investigational Site Number : 2500006

Nantes, , France

Site Status

Investigational Site Number : 2500007

Nice, , France

Site Status

Investigational Site Number : 2500002

Paris, , France

Site Status

Investigational Site Number : 2500010

Paris, , France

Site Status

Investigational Site Number : 2500011

Tours, , France

Site Status

Countries

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France

Related Links

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https://www.trialsummaries.com/Study/StudyDetails?id=25338&tenant=MT_SNY_9011

PTA17333 Plain Language Results Summary

Other Identifiers

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U1111-1266-5434

Identifier Type: REGISTRY

Identifier Source: secondary_id

2024-514773-22

Identifier Type: REGISTRY

Identifier Source: secondary_id

2021-002590-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

PTA17333

Identifier Type: -

Identifier Source: org_study_id

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