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非在研适应症- |
最高研发阶段临床1期 |
首次获批国家/地区- |
首次获批日期1800-01-20 |
A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Intravenously Administered PYC-003, a Peptide-phosphorodiamidate Morpholino Oligonucleotide Conjugate, in Healthy Adult Participants and Adult Participants with Confirmed PKD1 Mutation-associated Autosomal Dominant Polycystic Kidney Disease
This is a Phase 1, First-in-Human study to evaluate the safety and tolerability of PYC-003 in healthy adult participants and adult participants with confirmed PKD1 mutation-associated ADPKD. In Phase A of this study, healthy adult participants will either receive a single dose of PYC-003 or a placebo. Phase B, healthy adult participant will not receive any intervention and will serve as a baseline. Phase C, adult participants with confirmed PKD1 mutation-associated ADPKD will receive a dose of PYC-003.
100 项与 PKD1 x SHANK3 相关的临床结果
100 项与 PKD1 x SHANK3 相关的转化医学
0 项与 PKD1 x SHANK3 相关的专利(医药)