Pompe Disease (also known as glycogen storage disease type II)
Conditions
Brief summary
Incidence of adverse and serious adverse events (AEs/SAEs), clinically significant abnormal laboratory values, change in vital signs, change in physical examination (PE), vector shedding in bodily fluids, and liver function tests, Immune responses against the AAV-Spark100 capsid (neutralizing antibody [NAb] assay) and GAA transgene product (binding and NAb assays)
Detailed description
Changes from baseline in Six-Minute Walk Test (6MWT), Changes from baseline in % predicted forced vital capacity (FVC), Peak and steady-state vector-derived GAA enzyme levels assessed by total GAA protein and activity measured in circulation, Biomarkers of skeletal muscle injury, glycogen accumulation (creatine kinase [CK], urine glucose tetrasaccharide [Hex4])
Interventions
DRUGSPK-3006
Sponsors
Genentech Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of adverse and serious adverse events (AEs/SAEs), clinically significant abnormal laboratory values, change in vital signs, change in physical examination (PE), vector shedding in bodily fluids, and liver function tests, Immune responses against the AAV-Spark100 capsid (neutralizing antibody [NAb] assay) and GAA transgene product (binding and NAb assays) | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes from baseline in Six-Minute Walk Test (6MWT), Changes from baseline in % predicted forced vital capacity (FVC), Peak and steady-state vector-derived GAA enzyme levels assessed by total GAA protein and activity measured in circulation, Biomarkers of skeletal muscle injury, glycogen accumulation (creatine kinase [CK], urine glucose tetrasaccharide [Hex4]) | — |
Countries
Germany
Outcome results
None listed