A prospective cross-sectional and longitudinal study with additional restrospective chart review to evaluate clinical and biochemical characteristics and disease progression in patients with mucopolysaccharidosis type IIIB

Hospital de Clínicas de Porto Alegre0 个研究点开始时间: 2015年7月6日最近更新: 2023年5月29日

适应症

试验速览

暂无简介。

•Definitive diagnosis of MPS IIIB; at least 1 years of age (biological age) at the time of written informed consent; understands the full nature and purpose of the study, including possible risks of study procedures; informed consent signature.

•Visual or hearing impairments sufficient to preclude cooperation with neurodevelopmental testing; history of poorly-controlled seizure disorder; currently receiving medication which, in the Investigator’s opinion, would be likely to substantially confound interpretation of the results, or has been on the current dose of psychotropic medication for less than 3 months; receiving a newly increased dose of melatonin (e.g., less than 3 months on current dose); previously received an investigational therapy for the treatment of MPS IIIB or has had hematopoietic stem cell transplant (HSCT); in the opinion of the Investigator, the patient has any other prior or ongoing medical condition that may present a safety risk, interfere with study compliance, or confound data interpretation.

发起方

Hospital de Clínicas de Porto Alegre