Limb Girdle Muscular Dystrophy 2I/R9
Conditions
Brief summary
Change from baseline in NSAD at 36 months, Frequency and severity of TEAEs and TESAEs, Results of physical examinations including vital signs, Chemistry and hematology laboratory analyses, 12-lead ECG, including QTc intervals
Detailed description
Change from baseline in 10MWT (velocity, m/s) at 36 months, Change from baseline in pulmonary function as measured by FVC (percent predicted, performed in a sitting position) at 36 months, Change from baseline in the PUL2.0 at 36 months, Change from baseline in NSAD at 36 months, Change from baseline in total glycosylated αDG expression, Change from baseline in glycosylated αDG/ total αDG ratio, Change from baseline in pre-functional assessment serum CK
Interventions
DRUGRibitol
DRUGPlacebo to match BBP-418
Sponsors
ML Bio Solutions Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in NSAD at 36 months, Frequency and severity of TEAEs and TESAEs, Results of physical examinations including vital signs, Chemistry and hematology laboratory analyses, 12-lead ECG, including QTc intervals | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in 10MWT (velocity, m/s) at 36 months, Change from baseline in pulmonary function as measured by FVC (percent predicted, performed in a sitting position) at 36 months, Change from baseline in the PUL2.0 at 36 months, Change from baseline in NSAD at 36 months, Change from baseline in total glycosylated αDG expression, Change from baseline in glycosylated αDG/ total αDG ratio, Change from baseline in pre-functional assessment serum CK | — |
Countries
Denmark, Germany, Italy, Netherlands, Norway
Outcome results
None listed