A French multicenter Phase 4 open label extension study of long-term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France

Conditions

Glycogen storage disease type II

Brief summary

Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death

Detailed description

Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants, Assessment of quick motor function test (QMFT) for LOPD participants, Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants, Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants, Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants, Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants, Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants, PedsQL score for IOPD participants, Left Ventricular Mass Index (LVMI) Z-score in IOPD participants

Related papers

No related papers found yet.

Interventions

DRUGNexviadyme 100 mg powder for concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Number of adverse events (AE), treatmentemergent adverse events (TEAE), including infusion associated reactions (IAR) and death	—

Secondary

Measure	Time frame
Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants, Assessment of quick motor function test (QMFT) for LOPD participants, Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants, Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants, Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants, Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants, Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants, PedsQL score for IOPD participants, Left Ventricular Mass Index (LVMI) Z-score in IOPD participants	—

Measure

Time frame

Assessment of six-minute walk test (distance in meters and % predicted value) for late-onset Pompe disease (LOPD) and infantile-onset Pompe disease (IOPD) participants, Assessment of quick motor function test (QMFT) for LOPD participants, Pulmonary function tests (forced vital capacity [FVC] (% predicted), maximum expiratory pressure/maximum inspiratory pressure) in upright and supine positions for LOPD and IOPD participants, Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants, Quality of life evaluation: Pompe Disease Symptom Scale (PDSS) for LOPD participants, Quality of life evaluation: Pompe Disease Impact Scale (PDIS) for LOPD participants, Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) score for IOPD participants, PedsQL score for IOPD participants, Left Ventricular Mass Index (LVMI) Z-score in IOPD participants

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Countries

France

Outcome results

None listed