Failed primary in VALOR; FDA accelerated approval April 2023 based on plasma neurofilament-light reduction. Surrogate-endpoint case study.
Details
- nct_id
- NCT02623699
- phase
- phase_3
- status
- completed
- domain
- als
- indication
- SOD1-mutant ALS
- intervention
- Tofersen (antisense oligonucleotide targeting SOD1)
- sponsor
- Biogen
- n_enrolled
- 108
- primary_endpoint
- ALSFRS-R change at week 28
Raw fields (1)
- metadata
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