Pompe disease
Conditions
Brief summary
Efficacy: - Muscle strength: manual muscle testing (MMT), hand-held dynamometry (HHD) - Muscle function: 6-minute walk test (6MWT), quick motor function test (QMFT) - Pulmonary function: forced vital capacity (FVC) in sitting and supine position, maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP) - Patient reported outcome measures (PROMs): modified Rasch-built Pompe-specific activity (mR-PAct) scale, SF-36 (QoL, quality of life), Safety: - Vital signs: heart rate, blood pressure, respiratory rate - Weight - Assessment of treatment-emergent adverse events (TEAEs), including infusion associated reactions (IARs)
Interventions
DRUGOpfolda 65 mg hard capsules
DRUGPombiliti 105 mg powder for concentrate for solution for infusion
Sponsors
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Efficacy: - Muscle strength: manual muscle testing (MMT), hand-held dynamometry (HHD) - Muscle function: 6-minute walk test (6MWT), quick motor function test (QMFT) - Pulmonary function: forced vital capacity (FVC) in sitting and supine position, maximum inspiratory pressure (MIP), maximum expiratory pressure (MEP) - Patient reported outcome measures (PROMs): modified Rasch-built Pompe-specific activity (mR-PAct) scale, SF-36 (QoL, quality of life), Safety: - Vital signs: heart rate, blood pressure, respiratory rate - Weight - Assessment of treatment-emergent adverse events (TEAEs), including infusion associated reactions (IARs) | — |
Countries
Netherlands
Outcome results
None listed