THOR - Tübingen Choroideremia gene therapy trial open label Phase 2 clinical trial using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1) - THOR

niversitätsklinikum Tübingen, STZ eyetrial am Department für Augenheilkunde0 个研究点目标入组 6 人开始时间: 2015年5月21日最近更新: 2022年5月9日

适应症

试验速览

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•1.Participant is willing and able to give informed consent for participation in the study.
•2.Male aged 18 years or above.
•3.Genetically confirmed diagnosis of choroideremia. Patients without a confirmed mutation in the CHM gene, but who have the clinical phenotype typical of choroideremia can only be enrolled if they meet all the following three criteria: (i) family history consistent with X-linked inheritance, (ii) absent REP1 protein on Western blot of a blood sample and, (iii) normal RPE65 gene on sequencing.
•4.Active disease visible clinically within the macula region
•5.Best-corrected visual acuity equal to or worse than 6/9 (20/32; Decimal 0.63; LogMAR 0.2) but better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 6
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 6

•1.Female and child participants (under the age of 18)
•2.Participants with a history of amblyopia in the study eye
•3.Men unwilling to use barrier contraception methods, if relevant
•4.Grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control
•5.Any other significant ocular and non-ocular disease/disorder or retinal surgery which, in the opinion of the Investigator, may either put the participants at risk because of participation in the study, or may influence the results of the study, or the participant’s ability to participate in the study. This would include not taking or having a contraindication to oral prednisolone, such as a history of gastric ulcer or significant side effects.
•6.Participants who have participated in another research study involving an investigational product in the past 12 weeks, or having had gene or cellular therapy at any time prior to this study

发起方

niversitätsklinikum Tübingen, STZ eyetrial am Department für Augenheilkunde