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

Phase 1

Recruiting

• Conditions: iemann-Pick disease type C; MedDRA version: 20.0Level: SOCClassification code: 10010331Term: Congenital familial and genetic disorders Class: 21; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-510278-14-00
• Lead Sponsor: Intrabio Limited

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-01-26
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified