NCT04926129已完成不适用
Natural History of the Progression of X-Linked Retinitis Pigmentosa
NightstaRx Ltd, a Biogen Company1 个研究点 分布在 1 个国家目标入组 201 人开始时间: 2017年9月13日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 201
- 试验地点
- 1
- 主要终点
- Change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) Best-corrected Visual Acuity (BCVA)
研究概览
简要总结
The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).
详细描述
This study was previously posted by NightstaRx Ltd. In October, 2020, sponsorship of the trial was transferred to Biogen.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 7 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Have documentation of a pathogenic mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene.
- •Are willing and able to undergo ophthalmic examinations, as required by protocol, for up to 24 months
- •Have an ETDRS BCVA in at least 1 eye of ≥34 letters (Equivalent to Snellen ≥ 6/60 or 20/200; decimal 0.1; LogMAR 1.0).
- •Mean retinal sensitivity in the eligible eye as assessed by microperimetry:
- •Males with a mean retinal sensitivity of 68 loci ranging from ≥0.1 decibels (dB) and ≤20 dB.
- •Females with a mean retinal sensitivity of 68 loci ranging from ≥0.1 dB and ≤25 dB.
- •If female, have symptomatic disease with impairment of visual function.
排除标准
- •Have a history of amblyopia in the eligible eye.
- •Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may put the participant at risk because of participation in the study, may influence the results of the study, may influence the participant's ability to perform study diagnostic tests, or impact the participant's ability to participate in the study. This includes clinically significant cataracts.
- •Have participated in another research study involving an investigational medicinal product in the past 12 weeks or received a gene/cell-based therapy at any time previously (including but not limited to Intelligent Implant System implantation, ciliary neurotrophic factor therapy, nerve growth factor therapy).
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
结局指标
主要结局
Change from Baseline in Early Treatment Diabetic Retinopathy Study (ETDRS) Best-corrected Visual Acuity (BCVA)
时间窗: Up to Month 24
BCVA will be assessed for both eyes using the ETDRS visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If \<20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant.
Change from Baseline in Retinal Sensitivity Assessed with Microperimetry
时间窗: Up to Month 24
Macular analyser integrity assessment (MAIA) microperimetry will be conducted for both eyes to assess changes in retinal sensitivity within the macula.
次要结局
- Change from Baseline in Contrast Sensitivity(Up to Month 24)
- Change from Baseline in Low Luminance Visual Acuity (LLVA)(Up to Month 24)
- Change from Baseline in Health Utilities Index Mark 3 (HUI3)(Up to Month 24)
- Change from Baseline in Intraocular Pressure as Assessed by Goldmann Applanation Tonometry(Up to Month 24)
- Change from Baseline in Retinitis Pigmentosa (RP)-Specific Patient-Reported Outcome (PRO) Questionnaire(Up to Month 24)
- Change from Baseline in Multi-Luminance Mobility Test (MLMT) Readings(Up to Month 24)
- Change from Baseline in Fundus Autofluorescence (FAF)(Up to Month 24)
- Change from Baseline in 25-item Visual Function Questionnaire (VFQ-25) Score(Up to Month 24)
- Change from Baseline in EuroQol-5 Dimension 5-level (EQ-5D-5L)(Up to Month 24)
- Change from Baseline in Visual Field Readings(Up to Month 24)
- Change from Baseline in Microperimetry Readings(Up to Month 24)
- Change from Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT)(Up Month 24)
- Change from Baseline in Fundus Photography(Up to Month 24)
- Change from Baseline in Morphology of Eye as Assessed by Slit-lamp Examination(Up to Month 24)
- Change from Baseline in Adaptive-Optics Scanning Laser Ophthalmoscopy (AO-SLO)(Up to Month 24)
- Change from Baseline in Morphology of Eye as Assessed by Dilated Ophthalmoscopy(Up to Month 24)
- Change from Baseline in Morphology of Eye as Assessed by Lens Opacities Classification System (LOCS) III Lens Grading(Up to Month 24)
研究者
研究点 (1)
Loading locations...
相似试验
已完成
不适用
Wolman/CESD Natural History Chart Review and Longitudinal Follow-UpWolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2NCT01884220Children's Hospital Medical Center, Cincinnati4
已完成
不适用
Natural History Study of Patients With VCP-related DiseaseIBMPFDNCT04823143Nationwide Children's Hospital44
招募中
不适用
Immunological Characteristics of Preclinical IBDUlcerative ColitisIBDCrohn DiseaseNCT05698745Grupo Espanol de Trabajo en Enfermedad de Crohn y Colitis Ulcerosa450
已完成
不适用
Natural History Study of SLC25A46 Mutation-related MitochondriopathyOptic AtrophyNeurodegenerative Disease, HereditaryMitochondrial DiseasesNCT04594590State University of New York at Buffalo9
已完成
不适用
Natural History Study of FDXR Mutation-related MitochondriopathyNeurodegenerative Disease, HereditaryMitochondrial DiseasesOptic AtrophyNCT04580979State University of New York at Buffalo33