A multi-center, non-randomized study to characterize biomarkers in cerebrospinal fluid (CSF) of patients with infantile-onset (IOPD) or late-onset Pompe disease (LOPD) - ASY17795

Sanofi BV0 个研究点目标入组 3 人开始时间: 待定最近更新: 2024年4月9日

适应症

试验速览

暂无简介。

•I 01. Participants who have a confirmed diagnosis of LOPD and clinical signs or
•symptoms OR
•participants who have a confirmed diagnosis of IOPD.
•I 02. Capable of giving signed informed consent as described in Appendix 1
•(Section 10.1) of
•the protocol which includes compliance with the requirements and restrictions
•listed in the
•informed consent form (ICF) and in this protocol; OR If participant is <18
•parent(s) or legally authorized representative(s) (LAR) must be capable of
•giving signed
•informed consent as described in Appendix 1 (Section 10.1) which includes
•with the requirements and restrictions listed in the ICF and in this protocol.

•E 01. Chronic illness, except for LOPD, IOPD, or directly resulting from Pompe
•disease, that, at
•the discretion of the Investigator, could have an impact on Hex4 and
•inflammatory markers in the CSF.
•E 02. Any acute illness that, at the discretion of the Investigator, could have
•an impact on Hex4
•and inflammatory markers in the CSF.
•E 03. High risk of complications from lumbar puncture or potentially associated
•sedation, as judged by the Investigator.
•E 04. Concurrent treatment with any experimental drug or experimental vaccine.
•E 05. Participation in another clinical trial with any investigational drug
•within 30 days or 5 half-lives, whichever is greater, prior to study start (Day
•E 06. Previous participation in any clinical trial involving gene therapy.

发起方

Sanofi BV