International Niemann–Pick Disease Alliance

IB1001-301 Recruitment Complete

IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288).

Enrolment for IB1001-301 commenced in September 2022. Over 70 NPC patients were screened across 13 multinational trial sites in Australia, Europe, the United Kingdom, and the United States. Given the NPC community’s extremely high interest in IB1001-301, and their high unmet medical need, IntraBio over-recruited the trial by approximately 130%. Data readout is anticipated before the end of the second quarter 2023.

“We are thrilled to have completed recruitment and thankful to the dedicated Principal Investigators and Study Teams, as well as NPC Patient Organizations worldwide, who helped us reach this milestone” said Taylor Fields, IntraBio’s Chief Product Development Officer. “We are also very appreciative for the NPC community’s tremendous enthusiasm and support of our development of IB1001, which enabled us to complete recruitment so quickly. We remain determined to get IB1001 approved as quickly as possible to help meet NPC patients’ urgent unmet medical need.”

If you have any additional questions, please contact:


Taylor Fields

Chief Product Development Officer & Senior Vice-President, IntraBio


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