Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study - IB1001-301

Intrabio Limited0 个研究点目标入组 60 人开始时间: 2024年1月26日最近更新: 2024年7月22日

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•Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness, Male or female aged =4 years with a confirmed diagnosis of NPC at the time of signing informed consent, Patients must fall within: a)A SARA score of 7 = X = 34 points (out of 40) AND b)Either: i.Within the 2-7 range (0-8 range) of the Gait subtest of the SARA scale OR ii.Be able to perform the 9-Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 = X =150 seconds., Weight =15 kg at screening

•Patients who have any known hypersensitivity or history of hypersensitivity to Acetyl leucine (DL, L, D) or derivatives, and or Excipients in the IB1001 or placebo sachets, Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product, Patients with a physical or psychiatric condition which, at the investigator’s discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient’s participation in the clinical study, i.e. reliably perform study assessments, Patients who have been diagnosed with arthritis or other musculoskeletal disorders affecting joints, muscles, ligaments, and/or nerves that by themselves affects patient’s mobility and, at the investigator’s discretion, interferes with their ability to perform study assessments

发起方

Intrabio Limited