약학회지

ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)

Table. 5.

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