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IntraBio Ltd: Nov 301 NPC UK Recruitment Update

Posted: 03.11.22

IntraBio Ltd. is pleased to share that the Phase III pivotal trial with N-acetyl-L-leucine (IB1001-301) clinical trial has enrolled over 70% of the target number of patients in the second month of recruitment.

The study, which investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), will enroll a total of approximately 52 patients across all international sites.

Recruitment is expected to be completed by December 2022.


To ensure all patients have the opportunity to participate, interested patients are encouraged to contact their nearest study center as soon as possible to schedule a screening visit before enrollment is complete.   In the United Kingdom, three trial sites have been established. Information on each site, including contact details, can be found below:


Royal Manchester Children’s Hospital

Trial Site for Patients Aged 4 – 17 Years

Principal Investigator: Simon Jones, MD

Contact: Laura Crowther

Clinical Trials Manager


Tel: +44 (0)161 701 9137


Royal Free London NHS Foundation Trust

Trial Site for Patients Aged 13 Years +

Principal Investigator: Uma Ramaswami, MD

Contact: Masoud Kazemi

Senior Research Lead


Tel: +44 (0) 207 830 2814


Great Ormond Street Hospital

Trial Site for Patients Aged 4 – 17 Years

Principal Investigator: Paul Gissen, MD

Contact: Giuseppe Rizzetti

Lead Clinical Research Nurse


Tel: +44 (0)2074 059200 ext. 34228



IB1001-301 is a multinational, randomized, placebo-controlled, double-blinded, crossover Phase III study. Patients will receive treatment with both IB1001 orally-administered sachet) and a matching Placebo in two, 12-week treatment periods. Patients who complete the study will have the option to participate in an open-label extension phase, where patients will receive treatment with IB1001 for a minimum of 1-year.

Patients aged 4 years + may be eligible for recruitment at all trial sites. Patients are required to have neurological symptoms and cannot be using any other investigational agent (including investigational drugs in expanded access programs). Patients are permitted to use a stable dose of miglustat.

For the complete enrolment criteria, as well as details regarding the study assessments, multinational clinical trial sites, etc., please visit ClinicalTrials.Gov (NCT05163288).


If you have any additional questions on the study/recruitment, please contact:

Taylor Fields

Chief Product Development Officer & Senior Vice-President, IntraBio