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Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years

Active Not RecruitingPhase 4

Sponsor: Iris Plug

Conditions: Glycogen Storage Disease Type II Late Onset, Pompe Disease (Late-onset), Pompe Disease Late-Onset, Pompe's Disease Juvenile Onset, Pompe Disease

Interventions: Avalglucosidase Alfa

Countries: Netherlands

The goal of this clinical trial is to study the efficacy and safety of treatment with avalglucosidase alfa in patients with late onset Pompe disease that previously deteriorated on alglucosidase alfa. The main question it aims to answer is: * Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa safe? * Is switching to avalglucosidase alfa in late-onset Pompe patients deteriorating on alglucosidase alfa potentially more effective? Participants will switch to biweekly avalglucosidase alfa infusions (instead of alglucosidase alfa infusions) and perform assessment for: * Efficacy: muscle strength and function, pulmonary function, patient-reported outcomes. * Safety: Adverse events assessment, physical examination, clinical laboratory evaluations, vital signs, ECGs and immunogenicity assessments. * Pharmacokinetic assessments during 2 avalglucosidase alfa infusions.

Eligibility overview

Sex: ALL

Age: 5 Years to 55 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria

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

Locations (1)

Rotterdam, Netherlands