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Safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged ≥ 5 years.
EU CTIS ID: 2024-518215-18-00
What this study is testing
To explore safety, tolerability and efficacy of avalglucosidase alfa in patients with nonclassic Pompe disease aged ≥ 5 years of whom clinical condition deteriorates while on standard treatment with alglucosidase alfa.
- Therapeutic use (Phase IV)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Age ≥ 5 years and ≤ 55 years.
- Childhood or juvenile/young adult onset.
- Residing in the Netherlands
- Current enzyme-replacement therapy 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 (incl. patient and/or parent/guardian signed informed consent).
You likely can't join if
- Age >55 years.
- Invasive mechanical ventilation.
- No remaining useful functional ability (e.g. (almost) tetraplegic), as decided by the treating physician.
- Unmanageable, severe IARs 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
See the full eligibility criteria
- Age ≥ 5 years and ≤ 55 years.
- Childhood or juvenile/young adult onset.
- Residing in the Netherlands
- Current enzyme-replacement therapy 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 (incl. patient and/or parent/guardian signed informed consent).
- 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.
- Age >55 years.
- Invasive mechanical ventilation.
- No remaining useful functional ability (e.g. (almost) tetraplegic), as decided by the treating physician.
- Unmanageable, severe IARs 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.