TOPICAL GENTAMICIN TREATMENT OF PATIENTS WITH EPIDERMOLYSIS BULLOSA DUE TO NONSENSE MUTATIONS (THE GENTELBULL STUDY) - GENTELB

Oslo University Hospital0 个研究点目标入组 6 人开始时间: 2020年6月29日最近更新: 2022年5月9日

试验速览

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•Part A 1.The patient has EB caused by nonsense mutation 2.The patient has a symmetrical distribution of wounds which allows for wounds on one side of the body to serve as control wounds, while study therapy is applied to the other side. 3.The patient (if an adult) or parent(s)/guardian(s) is capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol
•Part B 1.The patient has EB caused by splice site mutations 2.The patient (if an adult) or parent(s)/guardian(s) is capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 4
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 4
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Part A 1.Known contact allergy against gentamicin sulfate or other ingredients in the ointment 2.Known intolerance to gentamicin sulfate of any sort 3.Moderate or severely reduced kidney function (eGFR <30) 4.Use other experimental therapy against EB 5.Receiving systemic aminoglycosides during the last 3 months
•Part B 1.The patient also carries a nonsense mutation that can be the cause of EB

发起方

Oslo University Hospital