First-in-human trialBasket designIntegrated protocol designOrphan diseaseComplement pathwayInnovative trial designs are sought to streamline drug development in rare diseases. Basket- and integrated protocol designs are two of these new strategies and have been applied in a handful oncologic trials. We ...
首次人体试验( first-in-human clinical trial,FIH) 是创新药物研发过程中的重要环节,以健康受试者或拟研究适应症患者为受试对象,对新药用于人体时的耐受、安全性,以及药动学、药效学进行初步探索。 2021-06-06 21:37 2021-12-02贝达药业最新管线进度:增加了4个1期临床入组! A.贝达药业基本分析及研报: 【1...
Parental Factors Associated With the Decision to Participate in a Neonatal Clinical Trial. JAMA Netw Open. 2021;4:e2032106. PubMed PubMed Central Google Scholar U.S. Department of Health and Human Services, Food and Drug Administration, Office of Good Clinical Practice, Center for Drug ...
案例3: First‐in‐human trial of the PI3Kβ‐selective inhibitor SAR260301 in patients with advanced solid tumors https://doi.org/10.1002/cncr.31044 磷酸肌醇3激酶(PI3K)β是许多磷酸酶和张力蛋白同系物(PTEN)缺陷型肿瘤模...
The first-in-human trial initiated last year in healthy adults studied single and multiple doses of NaQuinate. The primary objective was to assess the safety, tolerability and pharmacokinetics. ''We are delighted that the first-in-human study has completed its las...
MR-guided focused ultrasound (MRgFUS) is an emerging technology that can accurately and transiently permeabilize the blood-brain barrier (BBB) for targeted drug delivery to the central nervous system. We conducted a single-arm, first-in-human trial to in
Advanced Research Projects Agency (DARPA), which is an agency of the Department of Defense, is now supporting the first-in-human trial of the system.Florida International University, with Jung as principal investigator, will receive up to$2.2 millionin support of thi...
The PROBE trial is a first-in-human Phase I trial of ATG-101 in patients with metastatic/advanced solid tumors and mature B-cell non-Hodgkin lymphomas (B-NHL). The study will be conducted in the Dose Escalation Phase and Dose Expansion Phase, with the primary objective to eval...
Trial watch: First-in-human studies with cardiac myosin activatorsThe first in a new potential class of drugs to treat heart failure — cardiac myosin activators — has shown encouraging results in a Phase I trial and a small Phase II trial. Omecamtiv mecarbil, developed by Cytokinetics and...
Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a primary photoreceptor degeneration that leads to severe sight loss in young people. In the present study, we report the first-in-human phase 1/2, dose-escalation clinical trial for X-linked RP caused by muta...