An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I

Kawashima Satoshi0 个研究点目标入组 14 人开始时间: 2021年6月16日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•1. Patients who have completed the Part 2 of JR-171-101 study.
•2. A patient from whom written informed consent can be obtained. If the patient is aged under 20 years at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) needs to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible.
•3. Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted.

•1. A patient who is unable to perform the study procedures, except for 6-minute walk test, neurocognitive testing, BVMT-R, HVLT-R, and T.O.V.A.
•2. A patient who is judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process.
•3. A patient who is judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or hypersensitivity to any drugs.

发起方

Kawashima Satoshi