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): A multinational, multi-center, open-label, rater-blinded Phase II study” is open for recruitment at all planned United States trial centers.

IB1001-202 is a multinational study that investigates the effects of the novel drug IB1001 (N-Acetyl-L-Leucine) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). The trial has previously been accepted by the US Food and Drug Administration (IND 142302) as well as National Regulatory Agencies in Germany, Spain, and the United Kingdom.

In the US, patients aged 18 and older with a confirmed genetic diagnosis of Tay-Sachs of Sandhoff disease 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. Patients (and a caregiver) will be reimbursed for reasonable out-of-pocket expenses incurred for participating in the trial, such as travel and parking.

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:

NYU Langone School of Medicine
New York, New York, United States, 10017
Principal Investigator: Dr Heather Lau, MD
Contact: Sara Rodriguez, Research Coordinator
Email: Sara.Rodriguez@nyulangone.org
Tel: 929-455-5108

University of California – Los Angeles
Los Angeles, California, United States, 90095
Principal Investigator: Dr Susan Perlman, MD
Contact: Aaron Fisher, Staff Research Associate Supervisor
Email: ADFisher@mednet.ucla.edu
Tel:  310-206-8153

 The Mayo Clinic
Rochester, Minnesota, United States, 55905
Principal Investigator: Dr Anhar Hassan, MD
Contact: Sandra Looney, Clinical Research Coordinator
Email: Looney.sandra@mayo.edu
Tel: 507-538-4107

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