A pivotal study of N-Acetyl-L-Leucine on Niemann- Pick disease type C
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study
IRAS ID
1004498
Contact name
Taylor Fields
Contact email
Sponsor organisation
IntraBio Ltd
Eudract number
2021-005356-10
Clinicaltrials.gov Identifier
Research summary
The goal of this study is to demonstrate that N-Acetyl-L-Leucine is efficacious in improving symptoms, functioning, and quality of life in patients with NPC. A recent, Phase II clinical trial with N-Acetyl-L- Leucine (IB1001) for NPC (IB1001-201) demonstrated rapidly improved symptoms, functioning, and quality of life after 6-weeks of treatment in pediatric and adult patients with NPC. IB1001-201 met its primary CI-CS endpoint, as well as the secondary Scale for the Assessment and Rating of Ataxia (SARA) and Clinical Global Impression of Change endpoints, demonstrating statistically significant and clinically meaningful improvements in cerebellar signs and symptoms, functioning, and quality of life. N-Acetyl-L-Leucine was observed to be safe and well-tolerated, with no drug-related serious adverse events, establishing a very favourable risk- benefit profile for IB1001 for the treatment of NPC.
Based on these findings, IntraBio is conducting a Phase III, placebo-controlled, double-blind, randomized, crossover study investigating the efficacy and safety of N-Acetyl-L-Leucine for the treatment of NPC. Patients will only be included in the study if they meet the inclusion and exclusion criteria described in the study protocol approved by the relevant ethics committees and the regulatory authorities and informed consent is obtained. To further minimize risks, patients will be monitored closely throughout the study. The study will occur in approximately 14 sites in Australia, Czech Republic, Germany, the Netherlands, Slovakia, Switzerland, the United Kingdom and the United States. The sites in the UK will be NHS sites. There will be a screening period of up to 56 days and then the study duration, from the first day of dosing (Visit 2) to the final day of dosing at the end of Period II (Visit 6), is expected to be approximately 168 days.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
22/EM/0015
Date of REC Opinion
7 Mar 2022
REC opinion
Further Information Favourable Opinion