IB1001 Clinical Trials

IB1001 Clinical Trials

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)

Trial Status

The IB1001-201 (NPC) clinical trial completed enrollment in January 2020. Topline results were was 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.

Based on these findings, IntraBio is conducting a Phase III pivotal trial for patients with NPC (IB1001-301).

The IB1001-202 (GM2) clinical trial completed enrollment in October 2020. Topline results were was 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.

The IB1001-202 (GM2) and IB1001-203 (A-T) clinical trials have been significantly impacted by the global outbreak of COVID-19.  Recruitment for the IB1001-203 is delayed, given respiratory diseases are characteristics of A-T. Enrollment will continue when it is safe for patients and clinical trial sites.

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

IB1001-301Effects 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)

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)

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