| Autor: |
Tarnopolsky, M, Attarian, S, Borges, J, Bouhour, F, Choi, Y, Clemens, P, Day, J, Díaz-Manera, J, Erdem-Ozdamar, S, Goker-Alpan, O, Illarioshkin, S, Kishnani, PS, Kostera-Pruszczyk, A, Kushlaf, H, Ladha, S, Mozaffar, T, Roberts, M, Straub, V, Toscano, A, van der Ploeg, AT, An Haack, K, Hug, C, Huynh-Ba, O, Johnson, J, Zhou, T, Dimachkie, MM, Schoser, B |
| Zdroj: |
The Canadian Journal of Neurological Sciences; November 2021, Vol. 48 Issue: Supplement 3 pS27-S27, 1p |
| Abstrakt: |
Background:Phase 3 COMET trial (NCT02782741) compares avalglucosidase alfa (n=51) with alglucosidase alfa (n=49) in treatment-naïve LOPD. Methods:Primary objective: determine avalglucosidase alfa effect on respiratory muscle function. Secondary/other objectives include: avalglucosidase alfa effect on functional endurance, inspiratory/expiratory muscle strength, lower/upper extremity muscle strength, motor function, health-related quality of life, safety. Results:At Week 49, change (LSmean±SE) from baseline in upright forced vital capacity %predicted was greater with avalglucosidase alfa (2.89%±0.88%) versus alglucosidase alfa (0.46%±0.93%)(absolute difference+2.43%). The primary objective, achieving statistical non-inferiority (p=0.0074), was met. Superiority testing was borderline significant (p=0.0626). Week 49 change from baseline in 6-minute walk test was 30.01-meters greater for avalglucosidase alfa (32.21±9.93m) versus alglucosidase alfa (2.19±10.40m). Positive results for avalglucosidase alfa were seen for all secondary/other efficacy endpoints. Treatment-emergent adverse events (AEs) occurred in 86.3% of avalglucosidase alfa-treated and 91.8% of alglucosidase alfa-treated participants. Five participants withdrew, 4 for AEs, all on alglucosidase alfa. Serious AEs occurred in 8 avalglucosidase alfa-treated and 12 alglucosidase alfa-treated participants. IgG antidrug antibody responses were similar in both. High titers and neutralizing antibodies were more common for alglucosidase alfa. Conclusions:Results demonstrate improvements in clinically meaningful outcome measures and a more favorable safety profile with avalglucosidase alfa versus alglucosidase alfa. Funding:Sanofi Genzyme |
| Databáze: |
Supplemental Index |
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