Hope for pompe patients: could a switch in enzyme therapy slow decline?

NCT ID NCT07652814

Disease control Sponsor: Iris Plug Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for muscle strength, lung function, and quality of life. The trial includes about 6 people aged 5 to 55 in the Netherlands.

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
What this could lead to: If successful, this could offer a better treatment option for people with late-onset Pompe disease whose condition declines on current enzyme therapy.
What could go wrong: This is a small, early-phase study with only 6 participants, so results may not apply to everyone. The drug may not work better or could cause side effects like infusion reactions.

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

About 6 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2022

Expected to finish

Mar 2028

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

5 to 55 years

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: * Age\> 5 years and \< 55 years * Childhood or juvenile/adult onset * Residing in the Netherlands * Current ERT with alglucosidase alfa\> 2 years (dose regimen 20 or 40 mg/kg bi-weekly). * Confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confirmed disease-causing variants in the GAA gene. * Willing and able to adhere to study procedures * Deterioration in pulmonary function and/or 6MWT and/or muscle strength despite current treatment regimen with alglucosidase alfa. * Disease status: measurable pulmonary (dys)function: (F)VC ≤ 80% predicted (mechanic ventilation during the day or night allowed); Measurable muscle weakness in proximal and/or distal muscle groups (non- ambulant/wheelchair bound patients allowed); measurable functional ability Exclusion Criteria: * Age\>55 years * Invasive mechanical ventilation * No remaining useful functional ability, as decided by the treating physician * Unmanageable, sever IAR's on alglucosidase alfa * Deterioration due to high levels of anti-alglucosidase alfa antibodies interfering with treatment efficacy * Female patient of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to be tested for pregnancy

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Conditions

The condition(s) this trial relates to.

glycogen storage disease due to acid maltase deficiency, late-onset 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.

Glycogen storage disease type II late onset Pompe disease Pompe disease (late-onset) Pompe's disease juvenile onset

Hope for pompe patients: could a switch in enzyme therapy slow decline?

What the study statuses mean