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临床试验/NCT04770415
NCT04770415Unknown不适用

Ophthalmic Manifestations of Patients With Mucopolysaccharidosis

Shrouk Shaban Bakr1 个研究点 分布在 1 个国家目标入组 28 人开始时间: 2021年4月1日最近更新:
适应症

试验速览

阶段
不适用
发起方
入组人数
28
试验地点
1
主要终点
early detection and management of ophthalmic manifestations in patients with MPS

研究概览

简要总结

Mucopolysaccharidosis (MPSs) are a group of disorders caused by inherited defects in lysosomal enzymes resulting in widespread intra- and extra-cellular accumulation of glycosaminoglycan(1,2). They have been subdivided according to enzyme defect and systemic manifestations and include MPS IH (Hurler)(3) , MPS IS (Scheie), MPS IH/S (Hurler/Sheie), MPS II(4,5) (Hunter), MPS III (Sanfilippo)(6) , MPS IV (Morquio)(7,8), MPS VI (Maroteaux-Lamy)(9), MPS VII (Sly)(10,11) and MPS IX (Natowicz)(12). Mucopolysaccharidosis have a spectrum of systemic manifestations, including airway and respiratory compromise, skeletal deformities, intellectual and neurological impairment, cardiac abnormalities, gastrointestinal problems and ocular manifestations(13). Ocular manifestation are common in the mucopolysaccharidosis and may result in significant visual impairment(14). Corneal opacification of varying severity is frequently seen, as well as retinopathy, optic nerve swelling and atrophy, ocular hypertension, and glaucoma(14). New treatment modalities for the systemic manifestations of the mucopolysaccharidosis include bone marrow transplant and enzyme replacement therapy have resulted in an improved prognosis in many cases(15).

研究设计

研究类型
Observational
观察模型
Case Crossover
时间视角
Prospective

入排标准

年龄范围
— 至 18 Years(Child, Adult)
性别
All
接受健康志愿者

入选标准

  • All patients at any age up to 18 years with any type of MPS confirmed by the enzymatic assay

排除标准

  • Any patient other than MPS

结局指标

主要结局

early detection and management of ophthalmic manifestations in patients with MPS

时间窗: 1 week

次要结局

未报告次要终点

研究者

发起方
Shrouk Shaban Bakr
申办方类型
Other
责任方
Sponsor Investigator
主要研究者

Shrouk Shaban Bakr

Resident doctor

Assiut University

研究点 (1)

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