IntraBio Completes NPC Clinical Trial Enrollment

IntraBio Inc is pleased to share that it has completed enrollment for its IB1001-201 Clinical Trial.  


IB1001-201 investigates N-Acetyl-L-Leucine (IB1001) for both the symptomatic, and long-term, neuroprotective effect of treatment of Niemann-Pick disease Type C (NPC). 

Recruitment for IB1001-201 commenced in September 2019 and has since enrolled patients across multinational trial sites in the USA, UK, Germany, Spain, and Slovakia. Naïve patients with confirmed screening visits through January 2020 remain eligible for enrollment, and the Parent Study is expected to be completed by early May 2020.  


“We are very appreciative of the dedicated Principal Investigators and Study Teams, as well as NPC Patient Organizations worldwide who have helped us reach this milestone,” said IntraBio’s Chairman Mallory Factor. “We are thrilled to have completed recruitment and continue to make progress in the development of IB1001 to help meet the NPC community’s overwhelming medical need. IntraBio believes it is well-positioned for clinical, regulatory, and commercial success.”


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. NPC 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 for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease; IB1001-202), and Ataxia-Telangiectasia (A-T; IB1001-203). Enrollment in the IB1001-202 and IB1001-203 studies is ongoing.  


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