An open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/8-hG1.7p.coCNGA3) for gene therapy of children with achromatopsia owing to defects in CNGA3 - Gene Therapy for Achromatopsia (CNGA3)

MeiraGTx UK II Limited0 个研究点目标入组 36 人开始时间: 2019年4月23日最近更新: 2019年12月10日

试验速览

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•Key Inclusion Criteria:
•Aged 3 to 15 years old
•Achromatopsia caused by mutations in CNGA3
•Evidence of preservation of photoreceptors at the macula
•Able to undertake age-appropriate clinical assessments
•Willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune response to Advanced Therapy Investigational Product (ATIMP).
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 36
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Key Exclusion Criteria:
•Female adolescents who are pregnant or breastfeeding
•Intra-ocular surgery within 6 months of screening
•Ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results.
•Participated in another research study involving an investigational therapy for ocular disease within the last 6 months
•Have any other condition that the Principal Investigator (PI) considers makes them inappropriate for entry into the trial
•Are unwilling to consider the possibility of entry into a subsequent longer term follow up study

发起方