International Niemann–Pick Disease Alliance
IntraBio Inc. Pivotal Trial Announcement: 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 the news that recruitment to IntraBio’s Pivotal trial: 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” will open in July 2022 and will be conducted at 16 multinational sites in Australia, Europe, the United Kingdom, and United States.
With a high level of interest, it is expected that recruitment will be completed by December 2022. Patients and families who are interested in learning more about the trial, including the eligibility criteria and enrolment process, are encouraged to contact their specialist consultant, or INPDA representative in your home country where appropriate.
You can find a brief summary of the eligibility criteria, trial design, and trial sites provided in the attached document here.