Pompe disease patients get continued enzyme therapy in Long-Term safety study
NCT ID NCT05164055
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
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Phase 4
Runs after approval, following long-term safety and how well the treatment works in everyday use.
- Participants
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17 people
The number who actually took part.
- Started
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Jul 2022
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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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
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6 months and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Investigational Site Number : 2500001
Marseille, 13385, France
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Investigational Site Number : 2500002
Paris, 75013, France
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Investigational Site Number : 2500003
Lyon, 69003, France
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Investigational Site Number : 2500004
Bordeaux, 33000, France
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Investigational Site Number : 2500005
Brest, 29609, France
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Investigational Site Number : 2500006
Nantes, 44093, France
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Investigational Site Number : 2500007
Nice, 06200, France
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Investigational Site Number : 2500008
Clermont-Ferrand, 63000, France
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Investigational Site Number : 2500009
Lille, 59037, France
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Investigational Site Number : 2500010
Paris, 75015, France
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Investigational Site Number : 2500011
Tours, 37044, France
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