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Safety and PK/PD of Rugonersen in Angelman Syndrome
NCT04428281Phase 1Completed
- Sponsor
- Roche
- Start
- Aug 2020
- Primary completion
- Jul 2025
- Modality
- Modified DNA Oligonucleotides, Single-strand DNA, Antisense Oligonucleotides (ASOs)
- Countries
- 4
01
Overview
Phase 1, open-label trial of rugonersen in Angelman syndrome (AS) (19 cohorts), sponsored by Roche. Primary endpoints: Frequency and Severity of Adverse Events (AEs), Frequency and Severity of Serious Adverse Events (SAEs) and 9 more. Started Aug 2020; status: completed.
02
Drugs
rugonersen
03
Conditions
Angelman syndrome (AS)
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