A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) - A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM)

Tohoku University Hospital, Department of Neurology0 个研究点目标入组 19 人开始时间: 2017年3月20日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•-Ingestion of N-acetyl-D-mannosamine (ManNAc), SA (except for IND for the randomised, double-blined, placebo-controlled phase II/III study of NPC-09 (N-acetylneuraminic acid)(UMIN000020683)), or related metabolites; intravenous immunoglobulin (IVIG); or anything that can be metabolized to produce SA in the body within 60 days prior to the Screening Visit -Has had any hypersensitivity to SA or its excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects -Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study -Use of any investigational product or investigational medical device within 30 days prior to Screening, or anticipated requirement for any investigational agent prior to completion of all scheduled study assessments -Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment or may not allow safe participation in the study -Has a concurrent disease, active suicidal ideation, or other condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or would affect safety -More than 400 mL blood donation within 16 weeks -Presence of alcohol or drug dependency -Patients whom the investigator judges not to be appropriate for the subject

发起方

Tohoku University Hospital, Department of Neurology