Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease
interventional
N/A
1 country
1
The primary objective is to explore whether less frequent treatment with ERT (once every 4 weeks instead of once every 2 weeks) does not lead to increased progression of muscle strength, muscle function and pulmonary function in a selected group of elderly patients with LOPD who is in a stable condition (not requiring wheelchair use and respiratory support during the day while awake and seated), and who is at no immediate risk of losing the ability to carry out important activities of daily living (ADLs). If this less frequent treatment regimen (once every 4 weeks instead of once every 2 weeks) proves to be safe for a period of 9 months, and does not lead to increased disease progression, we aim to investigate whether treatment can eventually be discontinued.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
Not reported
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 20, 2024
Actual (reported)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)
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Formulated product
Opfolda 65 mg hard capsules
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
CAPSULE, HARD
View registry recordThe registry names these ingredients. The formulation’s exact chemical form has not been confirmed.
Opfolda 65 mg hard capsules
Named ingredient: miglustat
View registry recordErasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, 3015 GD, Netherlands
Ina Barzel
First Submitted
August 20, 2024
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