An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged ≥ 5 years.

Conditions

Glycogen storage disease type II or acid maltase deficiency, Pompe disease

Brief summary

Safety and tolerability are determined by assessing: • The number of AE’s/treatment-emergent adverse events, including infustion associated reactions • The occurrence of antibodies against avalglucosidase alfa • Clinical laboratory evaluations aimed at liver and muscle function (ASAT/ALAT/CK), Pharmacokinetics: • Single and multiple dose estimates for Cmax, AUC, CL, Efficacy is determined by assessing: • Changes in muscle strength: Manual Muscle Testing (MMT), Hand-Held dynamometry (HHD) • Changes in muscle function: Quick Motor Function Test (QMFT), 6-Minute Walk Test(6-MWT), Timed tests • Changes in the pulmonary function o Forced vital capacity in sitting and supine positions, Maximum Inpiratory Pressure (MIP), Maximum Expiratory Pressure (MEP) • Changes in PRO-Measures: Rasch-build Pompe Activity Scale (R-PaCT), Quality of life, modified Borg scale

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 64 Years

Design outcomes

Primary

Measure	Time frame
Safety and tolerability are determined by assessing: • The number of AE’s/treatment-emergent adverse events, including infustion associated reactions • The occurrence of antibodies against avalglucosidase alfa • Clinical laboratory evaluations aimed at liver and muscle function (ASAT/ALAT/CK), Pharmacokinetics: • Single and multiple dose estimates for Cmax, AUC, CL, Efficacy is determined by assessing: • Changes in muscle strength: Manual Muscle Testing (MMT), Hand-Held dynamometry (HHD) • Changes in muscle function: Quick Motor Function Test (QMFT), 6-Minute Walk Test(6-MWT), Timed tests • Changes in the pulmonary function o Forced vital capacity in sitting and supine positions, Maximum Inpiratory Pressure (MIP), Maximum Expiratory Pressure (MEP) • Changes in PRO-Measures: Rasch-build Pompe Activity Scale (R-PaCT), Quality of life, modified Borg scale	—

Measure

Time frame

Safety and tolerability are determined by assessing: • The number of AE’s/treatment-emergent adverse events, including infustion associated reactions • The occurrence of antibodies against avalglucosidase alfa • Clinical laboratory evaluations aimed at liver and muscle function (ASAT/ALAT/CK), Pharmacokinetics: • Single and multiple dose estimates for Cmax, AUC, CL, Efficacy is determined by assessing: • Changes in muscle strength: Manual Muscle Testing (MMT), Hand-Held dynamometry (HHD) • Changes in muscle function: Quick Motor Function Test (QMFT), 6-Minute Walk Test(6-MWT), Timed tests • Changes in the pulmonary function o Forced vital capacity in sitting and supine positions, Maximum Inpiratory Pressure (MIP), Maximum Expiratory Pressure (MEP) • Changes in PRO-Measures: Rasch-build Pompe Activity Scale (R-PaCT), Quality of life, modified Borg scale

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Countries

Netherlands

Outcome results

None listed