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Trial intelligence / Profile preview
RO7248824 open-label phase 1 study in children with Angelman syndrome
NL-OMON54298Phase 1Recruiting
- Sponsor
- Roche
- Start
- Jan 2021
- 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, open-label trial of rugonersen in Angelman syndrome (AS) (10 arms), sponsored by Roche. Primary endpoints: Frequency and severity of adverse events, Abnormal laboratory findings and 4 more. Started Jan 2021; status: recruiting.
02
Drugs
rugonersen
03
Conditions
Angelman syndrome (AS)
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