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Trial intelligence / Profile preview
Phase III rugonersen in Angelman syndrome
NL-OMON61789Phase 3Not yet recruiting
- Sponsor
- OHB Pediatrics
- Start
- Dec 2026
- 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
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Overview
Phase 3, randomised, double-blind trial of rugonersen in Angelman syndrome (AS) (2 arms), sponsored by OHB Pediatrics. Primary endpoint: Change from baseline in Bayley-4 cognition and/or expressive communication raw scores without caregiver input. Planned start Dec 2026; status: not yet recruiting.
02
Drugs
rugonersen
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Conditions
Angelman syndrome (AS)
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