International Niemann–Pick Disease Alliance

IntraBio: Clinical Trial IB1001-201: US Recruitment

Inc. is pleased to confirm that the Clinical Trial IB1001-201, “Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease (NPC): A multinational, multi-center, open-label, rater-blinded Phase II study” is open for recruitment in the United Kingdom.

IB1001-201 is a multinational study that investigates the effects of the novel drug IB1001 (N-Acetyl-LLeucine) for treatment of Niemann-Pick Disease type C (NPC). The trial has previously been accepted by Independent Ethics Committees (IEC), and the UK Medicines and Healthcare products Regulatory Agency (EudraCT: 2018-004431-71), as well as National Regulatory Agencies in Germany, Slovakia, and Spain, and the US Food and Drug Administration (IND 134369).

In the UK/Europe, patients aged 6 and older with a confirmed diagnosis of NPC may be eligible for recruitment. The trial will consist of three study phases: a baseline period, a 6-week treatment period, and a 6-week post-treatment washout period, followed by a planned one-year extension phase. Patients (and a caregiver) will be reimbursed for reasonable out-of-pocket expenses incurred for participating in the trial, such as travel and parking.

Four trial sites have been established in the United Kingdom for paediatric (ages 6-17) and adult (aged 18+) patients. Patients and families who are interested in learning more about the trial, including the eligibility criteria and enrolment process, should contact their prospective study site:

Great Ormond Street Hospital
Trial Site for Patients Aged 6 – 17 Years
Principal Investigator: Paul Gissen, MD
Contact: Olivia Rosie-Wilkinson
Senior Research Coordinator
Email: Olivia.Rosie-Wilkinson@gosh.nhs.uk
Tel: +44 (0)2074 059200 ext. 47267
Royal Manchester Children’s Hospital
Trial Site for Patients Aged 6 – 17 Years
Principal Investigator: Simon Jones, MD
Contact: Laura Crowther
Clinical Trials Manager
Email: laura.crowther@mft.nhs.uk
Tel: +44 (0)161 701 9137
Royal Free London NHS Foundation Trust
Trial Site for Patients Aged 18 Years +
Principal Investigator: Uma Ramaswami, MD
Contact: Masoud Kazemi
Senior Research Lead
Email: masoud.kazemi@nhs.net
Tel: +44 (0) 207 830 2814
Salford Royal NHS Foundation Trust
Trial Site for Patients Aged 18 Years +
Principal Investigator: Reena Sharma, MD
Contact: Marie Meehan, Senior Clinical
Research Practitioner/ Team Manager
Email: Marie.Meehan@srft.nhs.uk
Tel: +44 (0)161 206 4192

For the complete enrollment criteria, as well as details regarding the study assessments, multinational clinical trial sites, etc., please visit ClinicalTrials.Gov(NCT03759639)

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