Exploratory study to investigate the safety and efficacy of NPJ005 in patients with ATR-X syndrome. - IACT21003

Wada Takahito0 个研究点目标入组 5 人开始时间: 2022年7月14日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•1) meet the Definite diagnostic criteria for the ATR-X syndrome
•2) Age > 2 years at the time of enrolment
•3) Weigh 10 kg or more at the time of enrolment
•4) Have a parent or reliable carer
•5) Have given written consent to participate in this clinical trial. (If the patient is under 18 years of age, consent should be obtained from the individual and his/her legal guardian). If the patient's intellectual disability makes it difficult to obtain consent from the individual, consent should be obtained from the legal guardian.

•1) Those with complications not directly related to the ATR-X syndrome (e.g. perinatal hypoxic encephalopathy, which may affect the assessment)
•2) Those with serious complications (e.g. haematological, renal, hepatic, cardiac, cancer)
•3) Those with a history of major illness or surgery in the past, which is judged to affect the assessment of central nervous system function
•4) Those with no visual function that allows recognition of test indices
•5) Those with concomitant or previous history of photosensitivity or porphyria
•6) With a history of hypersensitivity to 5-ALA, porphyrins or iron preparations
•7) Those with haemochromatosis
•8) have taken supplements containing 5-ALA as a main ingredient within 3 months prior to enrolment (same date 3 months earlier)
•9) Participating in another clinical study or trial involving an intervention within 3 months (same date 3 months earlier) prior to obtaining consent
•10) Any other case that the investigator or sub-investigator deems inappropriate for the safe conduct of this clinical trial.

发起方

Wada Takahito