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Long-term RBM-007 study in children with achondroplasia
jRCT2031220338Phase 2Active, not recruiting
- Sponsor
- Ribomic Inc.
- Start
- Dec 2023
- Modality
- RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 2, open-label trial of umedaptanib pegol in achondroplasia (ACH) (2 arms), sponsored by Ribomic Inc. Primary endpoints: Comparison of height growth velocity in the observational study and height growth velocity after early phase II study entry, Height growth velocity before administration versus after switching to weekly 1 mg/kg RBM-007 and 10 more. Started Dec 2023; status: active, not recruiting.
02
Drugs
umedaptanib pegol
03
Conditions
achondroplasia (ACH)
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