International Niemann–Pick Disease Alliance

IB1001-301: Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study

We are pleased to share an update from IntraBio, regarding the clinical development of their lead drug, N-acetyl-L-leucine (IB1001) for NPC.  IntraBio is conducting a pivotal Phase III, randomized, double-blind, placebo-controlled trial for patients aged 4 years and older with Niemann-Pick disease type C (NPC).

Trial sites are currently planned in Australia, the Czech Republic, Germany, the Netherlands, Slovakia, Switzerland, the United Kingdom, and the United States. Full study information and eligibility criteria can be found on ClinicalTrials.Gov by searching with the identifier: NCT05163288.  Further updates are expected soon.


IB1001-301 One Pager (00000002)

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