An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing to defects in CNGB3 - Gene Therapy for Achromatopsia: CNGB3

MeiraGTx UK II Limited0 个研究点目标入组 27 人开始时间: 2016年11月17日最近更新: 2019年12月16日

适应症

试验速览

暂无简介。

•Inclusion in the trial will be limited to individuals who:
•Are aged 3 years or older (children will be included only once the maximal tolerated dose has been determined)
•Have Achromatopsia caused by mutations in CNGB3
•Present evidence of preservation of photoreceptors at the macula
•Are able to undertake age-appropriate clinical assessments
•Are willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune response to ATIMP
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 9
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 18
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Individuals will be excluded who:
•Are females who are pregnant or breastfeeding
•Had intra-ocular surgery within 6 months of screening
•Have an ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results.
•Have participated in another research study involving an investigational therapy for ocular disease within the last 6 months
•Have any other condition that the PI considers makes them inappropriate for entry into the trial, inclusive of but not limited to a history of the following: hypertension, diabetes mellitus, tuberculosis, renal impairment, immunocompromised
•state, osteoporosis, gastric ulceration or severe affective disorder
•Are unwilling to consider the possibility of entry into a subsequent longer term follow up study

发起方