2024-515553-17-00进行中（未招募）1/2 期
Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observer-masked trial (Colour Bridge)
Universitaetsklinikum Tuebingen AöR1 个研究点 分布在 1 个国家目标入组 14 人开始时间: 2024年7月16日最近更新:
相关药物
试验速览
- 阶段
- 1/2 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
研究概览
简要总结
To proof the safety and efficacy of rAAV.hCNGA3 in patients with achromatopsia if applied bilaterally in adults and minors.
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 年龄范围
- 0 years 至 65+ years（18-64 Years, 0-17 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •clinical diagnosis of achromatopsia
- •Female patients of childbearing potential must agree to use an effective method ofbirth control during the first 6 months post treatment.
- •negative pregnancy test in women with childbearing potential (a woman who istwo years post-menopausal or surgically sterile is not considered to be ofchildbearing potential)
- •6-12 years of age
- •≥ 18 years of age
- •bi-allelic pathogenic or likely pathogenic mutation in CNGA3
- •BCVA ≥ 20/400
- •a minimal outer nuclear layer thickness of 10μm at 3° eccentricity (normal =38±6μm)
- •ability to understand and willingness to consent to study protocol
- •no infection with Human Immundeficiency Virus (HIV)
- •Male patients must agree to use condoms during the first 6 months post treatment.
排除标准
- •any other retinopathy due to other diseases e.g. (but not limited to) arterialhypertension, trauma or acquired inflammatory diseases (uveitis serology) ,retinopathy of the premature
- •causal mutations in other genes for hereditary retinal diseases
- •contraindications in view of the planned surgery (e.g. anaemia Hb<8g/dl, severecoagulopathy, severe blood pressure fluctuations) including intolerance andcontraindications to general anaesthesia
- •ocular opacity and mature cataract
- •ocular infection with herpes simplex virus in medical history
- •history of ocular malignancies
- •disorders of the inner retina (e.g. retinal vascular occlusions in the patients history)
- •glaucoma defined as damage of the optic nerve
- •history of poorly controlled (HbA1c > 7%) Diabetes Mellitus type 1 or type 2
- •patients treated with systemic corticoids within 14 days prior inclusion
- •systemic illness or medically significant abnormal laboratory values >3 UNL in bloodanalysis including renal and hepatic functions at inclusion
- •systemic conditions (e.g. coronary heart disease, congenital/genetic conditions,autoimmune disorders) which may affect study participation or outcome measures
- •absence of vision on the contralateral eye
- •contraindication to pharmacological mydriasis (e.g. history of angle blockglaucoma)
- •current or recent participation in other study or administration of biologic agentwithin the last three months
- •recent (within the last 6 months) ocular surgery, intravitreal or subretinalimplantation of a medical device
- •known sensitivity to any compound used in the study
- •contraindications to systemic immunosuppression
- •subject/partner of childbearing potential unwilling to use adequate contraceptionfor six months after dosing
- •nursing or pregnant female subject
- •any other cause that, in the investigator‘s opinion, renders potential subjects notsuitable for the study
结局指标
主要结局
Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
Contrast sensitivity (Pelli Robson 3 m) 6 months after treatment
次要结局
- FrACT (Freiburg Visual Acuity & Contrast Test)
- Roth FM28 sat
- Contrast sensitivity (Pelli Robson 3 m)
- BCVA assessed using the ETDRS visual acuity protocol
- VA-CAL (Visual acuity under different conditions of contrast and ambient light)
- Patient reported outcomes (VFQ25/CVFQ, A3-PRO)
- Electroretinography
- Chromatic pupil campimetry (CPC) cone protocol – exploratory - functional
研究者
Dr. med Tobias Peters
Scientific
Universitaetsklinikum Tuebingen AöR
研究点 (1)
Loading locations...
相似试验
招募中
1 期
Safety and efficacy of a bilateral single subretinal injection of rAAV.hCNGA3 in adult and minor patients with CNGA3-linked achromatopsia investigated in a randomized, wait list controlled, observer-masked trialCNGA3-linked achromatopsiaMedDRA version: 20.0Level: LLTClassification code: 10000454Term: Achromatopsia Class: 10010331CTIS2024-515553-17-00niversitaetsklinikum Tuebingen AöR14
进行中（未招募）
1 期
Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked AchromatopsiaAchromatopsiaNCT02610582STZ eyetrial13
招募中
1 期
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expressioretinitis pigmentosaMedDRA version: 20.0Level: PTClassification code: 10038914Term: Retinitis pigmentosa Class: 100000004850CTIS2024-511687-90-00eyeDNA Therapeutics23
招募中
2 期
Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression2024-511687-90-00eyeDNA Therapeutics23
招募中
不适用
Single tube drainage versus double tube drainage for reduction of jaundice in cancers of bile duct.CTRI/2015/04/005725nil120