late-onset Pompe disease

The primary objective of the study is to determine the effect of neoGAA treatment on respiratory muscle strength as measured by Forced Vital Capacity (FVC) % predicted in the upright position, as compared to alglucosidase alfa.

neoGAA

neoGAA

Injection vial

100

Primary endpoint:
Change in FVC % predicted in the upright position from baseline to
12 months.
Secondary endpoint(s):
Change in the following parameters from baseline to 12 months:
Efficacy:
 MIP and MEP (% predicted)
 6MWT distance walked
 Motor function (QMFT)
 Lower extremity muscle strength (HHD), and
 Health-related quality of life (SF-12)
Safety:
 Assessment of adverse events (AEs)/treatment-emergent
adverse events (TEAEs), including infusion associated
reactions (IARs)
 Clinical laboratory evaluations including hematology,
biochemistry, urinalysis
 Physical examination
 Vital signs
 12-lead electrocardiogram (ECG)
 Immunogenicity assessments
Other endpoints:
Tertiary endpoints:
 Motor function (GMFM-88 and GSGC),
 Upper extremity muscle strength (HHD), and
 Health-related quality of life (EQ-5D-5L and PedsQL)
Exploratory endpoints for patient reported outcomes:
 PDSS/PDIS
 R-PAct

Inclusion criteria:
The patient has confirmed GAA enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.

Exclusion criteria:
The patient is <3 years of age, has known Pompe specific cardiac
hypertrophy, is wheelchair dependent, is not able to ambulate
40 meters (approximately 130 feet) without stopping and without an
assistive device, requires invasive-ventilation (non-invasive
ventilation is allowed), is not able to successfully perform repeated
FVC measurements in upright position of ≥40% predicted and
≤85% predicted, or has had previous treatment with
immunomodulation, alglucosidase alfa, or any investigational therapy
for Pompe disease.