IntraBio Inc. is pleased to confirm that the Clinical Trial IB1001-202, “Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A multinational, multi-center, open-label, rater-blinded Phase II study” is open for recruitment in the United Kingdom.

IB1001-202 is a multinational study that investigates the effects of the novel drug IB1001 (N-Acetyl-L-Leucine) for treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff). IB1001-202 has previously been accepted by Independent Ethics Committees, and the UK Medicines and Healthcare products Regulatory Agency (EudraCT: 2018-004406-25), as well as National Regulatory Agencies in Germany and Spain, and the US Food and Drug Administration (IND 142302).

In the UK/Europe, patients aged 6 and older with a genetically confirmed diagnosis of GM2 Gangliosidosis 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.

Two trial sites have been established in the United Kingdom for pediatric (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 enrollment process, should contact their prospective study site:

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: +44 161 701 9137

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: +44 161 206 4192

For the complete enrollment criteria, as well as details regarding the study assessments, multinational clinical trial sites, etc., please visit ClinicalTrials.Gov(NCT03759665)