Status:
NOT_YET_RECRUITING
Treatment Frequency Reduction in Pompe Disease
Lead Sponsor:
Erasmus Medical Center
Conditions:
Pompe Disease (Late-onset)
GAA Deficiency
Eligibility:
All Genders
50+ years
Phase:
PHASE4
Brief Summary
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease i...
Detailed Description
All eligible patients with late-onset Pompe disease will be treated with alglucosidase alfa 20 mg/kg once every 4 weeks for 9 months. During the study, patients will be monitored once every 3 months. ...
Eligibility Criteria
Inclusion
- LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and/or 2 confirmed disease-causing variants in the GAA gene)
- Age ≥50 years
- Current treatment with alglucosidase alfa at a standard dose of 20 mg/kg once every 2 weeks for ≥4 years
- Relatively stable clinical condition over the past year
- Able to walk ≥150 m within 6 minutes (6MWT)
- (Forced) vital capacity (FVC) in sitting position: \>55% of expected value and in supine position: \>45% of expected value
- Willing and able to adhere to the study procedures
Exclusion
- Rapidly progressive muscle weakness
- Severely limited muscle strength almost requiring/requiring daily wheelchair use
- Requiring respiratory support (non-invasive/invasive ventilation) or being at high risk to require respiratory support (ventilation) due to further deterioration of current pulmonary function. Using continuous positive airway pressure (CPAP) support only for obstructive sleep apnea syndrome (OSAS) is permitted.
- Comorbidities which are expected to influence the primary outcome measures within the next 2 years
Key Trial Info
Start Date :
October 1 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2027
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT06575829
Start Date
October 1 2024
End Date
December 31 2027
Last Update
August 28 2024
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