약학회지

ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
Table. 5. Comparative overview of Luxturna, Zolgensma, and Hemgenix regarding clinical study design and subject sample size
Trade Name Subject Sample Size Phase III Clinical Study Design
Phase I Phase II Phase III Total Study Design Control Arm Primary Efficacy Endpoint
Luxturna 12 N/A 31 43 Open-label
Randomized
Cross-over		 Cross-over Multi-luminance mobility test (MLMT) score change using both eyes from baseline to Year 1
Zolgensma 15 N/A 21 36 Open-label
Single-arm		 Natural history Survival at 14 months of age and the proportion of subjects able to sit independently for ≥30 seconds by 18 months of age
Hemgenix *(10) 3 51 54 Open-label
Single-arm		 Study lead-in period Annualized Bleeding Rate (ABR) during 7-18 months after treatment with AMT-061 compared with the ABR during the lead-in period

*Non-commercial lot

N/A: Non-applicable
Yakhak Hoeji 2023;67:342-53 https://doi.org/10.17480/psk.2023.67.6.342
© 2023 Yakhak Hoeji