Study design & results
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Trial intelligence / Profile preview
BIIB115 phase 1 intrathecal dose-escalation study in healthy volunteers and pediatric SMA participants previously treated with Zolgensma
NL-OMON53796Phase 1Not yet recruiting
- Sponsor
- Biogen
- Start
- Aug 2022
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 1, randomised, double-blind trial of salanersen in spinal muscular atrophy (SMA) (3 arms), sponsored by Biogen. Primary endpoint: Incidence of adverse events and serious adverse events. Planned start Aug 2022; status: not yet recruiting.
02
Drugs
salanersen
03
Conditions
spinal muscular atrophy (SMA)
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Competitive context
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