IntraBio is conducting multinational clinical trials with its lead compound, IB1001 (N-acetyl-L-leucine) for the treatment of three orphan diseases:  Niemann-Pick disease type C (NPC);  GM2 Gangliosidosis (Tay-Sachs and Sandhoff);  Ataxia-Telangiectasia (A-T)

Information about each active clinical center, including contact details, can be found on ClinicalTrials.Gov: 

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

IB1001-301 ClinicalTrials.gov (NCT05163288)

IB1001-201 – Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

 IB1001-201 ClinicalTrials.gov (NCT03759639)

The IB1001-201 (NPC) clinical trial completed enrollment in January 2020. Topline results were announced in September 2020 confirming the clinical trial demonstrated a statistically significant and clinically meaningful improvement in both the primary and topline secondary endpoints for the treatment of pediatric and adult patients with NPC.

IB1001-202 – Effects of N-Acetyl-L-Leucine on GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-202 ClinicalTrials.gov (NCT03759665)

The IB1001-202 (GM2) clinical trial completed enrollment in October 2020. Topline results were announced in August 2021 confirming the clinical trial demonstrated a statistically significant and clinically meaningful improvement in both the primary and topline secondary endpoints for the treatment of pediatric and adult patients with GM2.

IB1001-203 Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study

IB1001-203 ClinicalTrials.gov (NCT03759678)

IB1000s Series