CTIS2023-508688-54-00进行中（未招募）1 期
An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene - CCPK850X2202
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
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
- 年龄范围
- 18 至 65+（—）
- 性别
- All
入选标准
- •Written informed consent must be obtained before any assessment is performed., Male and female patients aged 18 to 70 years. All female patients must have negative pregnancy test results at the screening visits and just prior to the treatment day., The patients must have sufficiently clear ocular media and adequate pupil dilation to permit fundus photographs of adequate clarity and performance of vitrectomy and subretinal injection., Patients must meet surgical requirements. In addition, patients must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) <40 kg/m2. BMI = Body weight (kg) / [Height (m)]2., The visual acuity in the study eye at the screening 1 visit should be no better than 60 early treatment diabetic retinopathy study (ETDRS) letters. In Cohort 1, patients with visual acuity from 35 ETDRS letters to Hand Motion visual acuity will be included. For Cohorts 2 and beyond, patients with visual acuity of 60 ETDRS letters to Hand Motion visual acuity will be enrolled. Consideration for inclusion in Cohorts 1 and 2 only may be given to patients with visual acuity as low as Light Perception at the screening 1 visit, if they meet all other inclusion/exclusion criteria and at the agreement of the sponsor and principal investigators., Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa phenotype with biallelic mutations in the RLBP1 gene verified by Clinical Laboratory Improvement Amendments (CLIA), Good Laboratory Practices (GLP) or equivalent molecular genetics testing., Visible photoreceptor (outer nuclear) and retinal pigment epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit as confirmed by the Central Reading Center., Dark adaptation bleaching effect in the study eye at short wavelength stimulus of > 1.0 log unit at 2 of the 3 baseline measures obtained prior to treatment.
排除标准
- •Unable or unwilling to meet requirements of the study, History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period., Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints; for example: glaucoma (IOP =25 mm Hg despite treatment with anti-glaucoma medication or low tension glaucoma), corneal or significant lenticular opacities, retinal vascular occlusion, retinal detachment, macular hole, or choroidal neovascularization of any cause., Any active infection or ocular disease involving ocular adnexa including infectious conjunctivitis, keratitis, scleritis, endophthalmitis, as well as idiopathic or autoimmune-associated uveitis in either eye., Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, primary care physician or designee). Use of systemic anticoagulant therapies during the study, such as warfarin, heparin or similar are to be evaluated by the treating physician as potential exclusions. The use of aspirin is not usually an exclusion criterion unless indicated by the treating physician. Abnormal vital signs and/or Electrocardiograms (ECGs) that suggest potential contraindications for planned study anesthesia are exclusions., Known history of or current clinically significant arrhythmias, myocardial infarction, coronary bypass surgery, or any percutaneous coronary intervention (PCI) within 6 months prior to screening or patients with heart failure New York Heart Association (NYHA) class IIIV at the discretion of the treating physician or cardiologist., Cerebrovascular accident (stroke) within the 12 months prior to screening at the discretion of the treating physician., Complicating systemic diseases or clinically significant abnormal laboratory values. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example radiation treatment of the orbit; leukemia with central nervous system (CNS)/optic nerve involvement). Also patients with immunocompromising diseases would be excluded since they would have susceptibility to opportunistic infections., Women who are pregnant or nursing (lactating), as well as male and female patients of childbearing potential that are unwilling to use contraception as per study requirements.
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