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Pompe disease patients get continued enzyme therapy in Long-Term safety study

NCT ID NCT05164055

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 2 times

Summary

This study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the drug is covered by French health insurance or until September 2026.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Avalglucosidase alfa (Nexviadyme), an enzyme replacement therapy given by IV infusion every 2 weeks
What this could lead to
If successful, this study could confirm that long-term use of avalglucosidase alfa is safe and helps manage Pompe disease symptoms.
What could go wrong
This is a small, open-label extension study with only 17 participants, so results may not apply to everyone. Side effects like infusion reactions are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Participants

17 people

The number who actually took part.

Started

Jul 2022

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Investigational Site Number : 2500001

    Marseille, 13385, France

  • Investigational Site Number : 2500002

    Paris, 75013, France

  • Investigational Site Number : 2500003

    Lyon, 69003, France

  • Investigational Site Number : 2500004

    Bordeaux, 33000, France

  • Investigational Site Number : 2500005

    Brest, 29609, France

  • Investigational Site Number : 2500006

    Nantes, 44093, France

  • Investigational Site Number : 2500007

    Nice, 06200, France

  • Investigational Site Number : 2500008

    Clermont-Ferrand, 63000, France

  • Investigational Site Number : 2500009

    Lille, 59037, France

  • Investigational Site Number : 2500010

    Paris, 75015, France

  • Investigational Site Number : 2500011

    Tours, 37044, France

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