EUCTR2015-003418-25-GB进行中（未招募）1 期
An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with Defects in RPE65 (LCA2) - Gene Therapy Trial for LCA2 OPTIRPE65 (AAV2/5-OPTIRPE65)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 27
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion in the trial will be limited to individuals who:
- •Are aged 3 years or older
- •Have early-onset severe retinal dystrophy consistent with RPE65 deficiency
- •Have homozygous or compound heterozygous missense or null mutations in RPE65 confirmed in accredited laboratory
- •Have functional or structural evidence of photoreceptor preservation as assessed by static perimetry (for visual field assessment) and SD-OCT scanning respectively
- •Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate
- •Are able to undertake age-appropriate clinical assessments
- •If female and of child bearing potential, are willing to use an effective form of birth control (hormonal or barrier method of birth control; or abstinence) for at least 12 months following ATIMP administration
- •If male, are willing to use barrier and spermicide form of contraceptive or maintain sexual abstinence for at least 12 months following ATIMP administration
- •Females of childbearing potential will have a negative pregnancy test on the day of ATIMP administration. Participants are considered not of childbearing potential if they are surgically sterile (i.e. they have undergone a hysterectomy or bilateral oophorectomy) or post-menopausal
- •Are willing to give consent for the use of blood and blood components collected throughout the trial for the investigation of immune responses to the 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 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •Individuals will be excluded who:
- •Are females who are pregnant or breastfeeding
- •Have contraindications for transient immune-suppression by systemic corticosteroids (including uncontrolled hypertension, diabetes mellitus, tuberculosis, renal impairment, osteoporosis, gastric ulceration, severe affective disorder) or are immunocompromised
- •Have a previous history (within 5 years) of gastric or duodenal ulceration, hiatus hernia, uncontrolled gastro-oesphageal reflux or are using non-steroidal anti-inflammatory drugs on a regular basis at the time of screening
- •Have a known allergy to any of the non-investigational drugs to be used in the trial
- •Have participated in another research study involving an investigational medicinal 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
- •Have had intraocular 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
- •Are unwilling to consider the possibility of entry into a subsequent longer term follow up study.
研究者
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