RIBOMIC Announces Completion of IND Submission for an Observational Study for Continuous Phase 2 Trial of RBM-007 for Treatment of Achondroplasia
19 Apr 2022
Umedaptanib pegolAchondroplasia
Ribomic, Inc., a clinical stage pharmaceutical company specializing in aptamer therapeutics (TOKYO:4591), today announced that it submitted an Investigational New Drug Application (IND) for an observational study to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan. The purpose of the observational study is to obtain clinical basic data, including height growth, and to select subjects for the early phase II study.
If there is no comment from PMDA for 14 days after submission, the observational study will be initiated.
Overview of the study
Subjects：atients with achondroplasia
Target number of subjects：14
Purpose of the study ：The purpose of this study is to obtain clinical basic data including height growth in children (aged 5-14 years) with achondroplasia, which will enable the evaluation of efficacy and safety in the continuous early Phase II study using RBM-007 as a comparative data, and to select subjects for the early phase II study appropriately.
Study design：Observational study (no therapeutic intervention)
Observation period：26 weeks or more, up to 2 years
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