Date: 2013-11-12
Type of information: Completion of the trial
phase: 1
Announcement: completion of the study
Company: Gentium (Italy)
Product: defibrotide
Action
mechanism: Defibrotide is a polydeoxyribonucleotide isolated from pig intestine. It is believed to have antithrombotic properties.
Disease: prevention and treatment of hepatic veno-occlusive disease (VOD)
Therapeutic area: Digestive diseases - Liver diseases - Transplantation
Country: Japan
Trial
details: The primary objective of the study was to evaluate the safety and pharmacokinetics of defibrotide in healthy Japanese adult subjects. The secondary objective was to evaluate the effect of defibrotide on the coagulation and fibrinolytic systems in vivo. Two dose levels (3 mg/kg and 6.25 mg/kg, termed Cohort 1 and Cohort 2, respectively) were evaluated, each cohort consisted of 10 subjects (8 subjects were given DF, 2 were given placebo).
Latest
news: * On March 26, 2013, Gentium has announced that the medical team from the National University Corporation Hamamatsu University School of Medicine, Fukushima Medical University have successfully administered defibrotide to the first subject in the phase 1 clinical study. This Investigator led clinical trial aims “to evaluate the Safety and Pharmacokinetics of Defibrotide (DF) in Healthy Adults”. The trial has been adopted as a Health and Labour Sciences Research Grant in 2012 for Practical Use of Medical Technology (categorized as a study project to support practical drug/medical device use during the reconstruction of* On November 12, 2013, Gentium has announced that the medical team from the Phase 1 Trial Unit of the Translational Research Center at the National University Corporation, Hamamatsu University School of Medicine (“HUSM”), Fukushima Medical University (“FMU) has successfully completed the phase 1 clinical study to evaluate the safety and pharmacokinetics of defibrotide in healthy adults. This Investigator led clinical trial was adopted as a Health and Labour Sciences ResearchGrant in 2012 for Practical Use of Medical Technology (categorized as a study project to support practical drug/medical device use during the reconstruction of disaster-hit areas ineastern Japan).All 20 subjects completed the protocol defined treatment and there were no premature discontinuations. No serious adverse events were noted and only one mild, transient and reversible adverse event observed. Moreover, no clinically significant changes were observed in laboratory test results in any of the dosing groups.Gentium provided free drug and technical support to HSUM.
disaster-hit areas in eastern Japan). Gentium is providing defibrotide and technical support to Hamamatsu University School of Medicine.
* On December 5, 2012, Gentium has announced the Company’s entry into an agreement with the National University Corporation Hamamatsu University School of Medicine, Fukushima Medical University, and LINK Healthcare to support a clinical trial to evaluate the safety and pharmacokinetics of defibrotide in healthy adults. An investigational new drug application for defibrotide was recently submitted to Japan’s Pharmaceuticals and Medical Devices Agency. Under the terms of the agreement, Gentium will provide free drug and technical support and LINK will provide administrative and logistical support for the clinical trial conducted by HUSM. If desired outcomes are achieved following the completion of the Phase I clinical trial, a Phase II Clinical Trial will need to be completed before submitting a marketing authorization application for Defibrotide to the PMDA. The clinical trial is supported by a Health and Labour Sciences Research Grant (2012) and is categorized by the grant program as a study to support practical drug/medical device use during the reconstruction of disaster-hit areas in Eastern Japan.
In June 2011, the Company announced the appointment of Link as exclusive distributor of defibrotide in Australia and new Zealand. Gentium and LINK plan to expand the distribution territories covered in their existing agreement to include Asia and Japan as LINK has recently established an operations and distribution facility in Singapore to serve the central Asian markets and purchased a business specializing in named-patient distribution in Japan.
