NL-OMON55731已完成不适用
atural History Study of CEP290-Related Retinal Degeneration - EDIT-NHS01
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 4
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational non invasive
入排标准
- 年龄范围
- 2 至 64（—）
入选标准
- •1. pariticipants and/or parent/legal guardian must complete/sign an informed
- •consent form (ICF). If
- •required on a per participants basis, provisions can be made for alternative
- •forms of consent
- •(eg, witnessed consent). Where required by the IRB/IEC, minors must also
- •verbalize or sign a
- •confirmation of assent. Refer to Section 11.3;
- •2. Male or female participants >=6 years of age at Screening;
- •3. Has abnormally decreased vision, defined as having light perception to 20/50
- •best-corrected visual acuity (BCVA) in
- •each eye, with examination and test results consistent with an inherited
- •retinal degeneration
- •due to mutations in the CEP290 gene;
- •4. Has CEP290-related retinal degeneration caused by a compound heterozygous or
- •intron 26 c.2991+1655A>G mutation (ie, 1 or 2 copies of the intron 26
- •c.2991+1655A>G
- •mutation) confirmed by deoxyribonucleic acid sequencing;
- •5. Has ability to cooperate with assessments relative to age;
- •6. Has clear ocular media and adequate pupil dilation in at least 1 eye, to
- •permit good quality
- •fundus examination and optical coherence tomography (OCT) imaging; and
- •7. For females of childbearing potential: is not pregnant as confirmed by a
- •negative urine pregnancy test at Screening and is not planning to become
- •pregnant during the course of the study.
排除标准
- •1. Has history or current evidence of a medical condition (systemic or
- •ophthalmic disease,
- •metabolic dysfunction, physical examination finding, or clinical laboratory
- •finding) that may,
- •in the opinion of the Investigator, preclude adherence to the scheduled study
- •visits, safe
- •participation in the study, or affect the results of the study (eg,
- •uncontrolled systemic
- •hypertension, autoimmune disease, advanced coronary artery disease, or cerebral
- •disease, other unstable or progressive cardiovascular, pulmonary, Parkinson's,
- •liver or renal
- •disease, cancer, or dementia);
- •2. Has history or current evidence of ocular disease in either eye that, in the
- •opinion of the
- •Investigator, may confound assessment of this inherited retinal disease or the
- •assessments
- •utilized herein (eg, glaucoma, age-related macular degeneration, diabetic
- •retinopathy, uveitis,
- •or the presence of any condition that precludes adequate visualization of the
- •fundus such as
- •dense cataracts or corneal scarring);
- •3. Achieves a passing score for the Visual Function Navigation Test at the
- •maximum level of difficulty (ie. passes the most challenging Visual Function
- •Navigation Test under the dimmest lighting conditions) with each eye
- •independently and both eyes together;
- •4. Is currently receiving gene therapy and/or has received gene therapy or
- •oligonucleotide therapeutics;
- •5. Is currently enrolled in an investigational or interventional drug or device
- •study and/or has
- •participated in such a study within 30 days of Screening.
- •6. For females of childbearing potential: is pregnant (or planning to become
- •pregnant) or breastfeeding.
研究者
相似试验
已完成
不适用
Natural History Study of CEP290-Related Retinal DegenerationBlindnessLeber Congenital Amaurosis 10Vision DisordersEye DiseasesEye Diseases, HereditaryEye Disorders CongenitalRetinal DiseaseRetinal DegenerationNCT03396042Editas Medicine, Inc.26
已完成
不适用
Clinical studies in the association with retina vitreous disease and intraocular cytokineretina vitreous diseaseJPRN-UMIN000003376Yachiyo Medical Center, Tokyo Womens Medical University57
已完成
不适用
Investigation of the Etiology of Eye DiseasesMacular diseasesJPRN-UMIN000038063Keio University School of Medicine22
招募中
不适用
Retrospective study of ocular surface diseasesocular surface diseasesJPRN-UMIN000026746Kyoto Prefectural University of Medicine2,500
招募中
不适用
Retrospective study of ocular surface diseasesocular surface diseasesJPRN-UMIN000024891Baptist Eye Institute2,500