X-linked myotubular myopathy
Conditions
Brief summary
Incidence and severity of TEAEs and AESIs, Change from baseline in the following safety assessments through week 52: o clinical laboratory tests o cardiac findings from ECG and ECHO o muscle findings from muscle MRI and histopathology o physical examinations
Detailed description
Change from baseline in hours per day of ventilation support at week 52, ASP2957 vector DNA in serum through week 52, ASP2957 vector DNA in muscle biopsy at week 52, ASP2957 vector DNA in saliva, urine and stool, Immunogenicity of ASP2957 through week 52 as assessed with the following tests: o Anti-MyoAAV3.8 TAb and NAb o Anti-myotubularin Tab
Interventions
DRUGASP2957
Sponsors
Astellas Gene Therapies Inc.
Eligibility
Sex/Gender
Male
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of TEAEs and AESIs, Change from baseline in the following safety assessments through week 52: o clinical laboratory tests o cardiac findings from ECG and ECHO o muscle findings from muscle MRI and histopathology o physical examinations | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in hours per day of ventilation support at week 52, ASP2957 vector DNA in serum through week 52, ASP2957 vector DNA in muscle biopsy at week 52, ASP2957 vector DNA in saliva, urine and stool, Immunogenicity of ASP2957 through week 52 as assessed with the following tests: o Anti-MyoAAV3.8 TAb and NAb o Anti-myotubularin Tab | — |
Outcome results
None listed