NCT07652814Active, not recruitingPhase 4
Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years
An Open Label, Single Center, Exploratory Study of the Safety and Efficacy of Avalglucosidase Alfa Patients With Non-classic Pompe Disease Aged ≥ 5 Years
Source: ClinicalTrials.gov · Last updated 17 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 6 (estimated)
- First submitted
- 1 Nov 2022
- Start date
- 13 Oct 2022
- Primary completion date
- 9 Mar 2028
- Lead sponsor
- Iris Plug
- Diseases
- Pompe disease
- NCT number
- NCT07652814
- EU CT number
- 2024-518215-18-00
- EudraCT number
- 2019-002251-42
What you commit to
- Study type: Interventional · Phase 4
- Planned participants: 6
- From 13 Oct 2022 to the primary completion date, 9 Mar 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Deterioration due to high levels of anti-alglucosidase alfa antibodies interfering with treatment efficacy
Age
- Age\>55 years
Other
- Invasive mechanical ventilation
- No remaining useful functional ability, as decided by the treating physician
- Unmanageable, sever IAR's on alglucosidase alfa
- 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
Who the study is looking for
- 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
Age in the registry: 5 years to 55 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC) Coordinator of this site? Confirm its status | Rotterdam | Netherlands | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.