IntraBio Inc is pleased to share that the Extension Phase for the IB1001-201 Clinical Trial has been approved in all European countries where the trial is being conducted.
IB1001-201 is a multinational, clinical trial that investigates N-acetyl-L-leucine (IB1001) for the treatment of Niemann-Pick disease Type C (NPC). In Europe, IB1001-201 is being conducted in centers across the United Kingdom, Germany, Spain, and Slovakia.
The approval of the Extension Phases provides patients enrolled in the current trial the opportunity to continue treatment with IB1001 if the principal investigator determines it is in their best interest.
The Extension Phase aims to provide further evidence of the symptomatic benefit of IB1001, and demonstrate the disease-modifying, neuroprotective effect of treatment. The long-term benefit of Acetyl Leucine has previously been observed in compassionate use studies and is strongly supported by in vitro and in vivo data.
NPC is a rare, debilitating, inherited lysosomal storage disorder that predominately affects pediatric patients. The disease begins in early childhood and is chronic and progressive in nature, and severely impacts quality of life. The average age of death for NPC patients is approximately 10 years, with half of the patients dying before the age of 12.5 years.
In addition to Clinical Study IB1001-201, IntraBio is running parallel multinational clinical trials involving IB001: IB1001-202 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), and IB1001-203 for the treatment Ataxia-Telangiectasia (A-T). An Extension Phase to both studies is also being conducted.