IB1001-201Effects of N-Acetyl-L-Leucine on Niemann-Pick type C Disease
Research type
Research Study
Full title
Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C: A multinational, multicenter, open-label, rater-blinded Phase II study.
IRAS ID
259038
Contact name
Paul Gissen
Contact email
Sponsor organisation
IntraBio Ltd
Eudract number
2018-004331-71
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
N/A, N/A
Duration of Study in the UK
1 years, 9 months, 30 days
Research summary
The primary purpose of the study is to evaluate the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the treatment of Niemann-Pick type C disease (NPC), and investigating the efficacy in terms of improving symptoms, functioning, and quality of life against the defined endpoints in patients with NPC.
NPC is a rare, distressing, neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD) that predominantly affects paediatric patients.
Patients with neurological onset early in life have more severe symptoms and deteriorate faster. NPC is estimated to affect 1:100,000 live births.
Currently there are limited and no curative treatments approved for NPC worldwide,
therefore, there is a strong need for the development of novel and more effective therapies to treat these intractable diseases.The Extension Phase allows patients who have completed Visit 6 of the Parent Study of IB1001-201 to, at the discretion of the Principal Investigator (PI), continue treatment with N-Acetyl-L-Leucine (IB1001) if the PI determines it is in their best interest. The Extension Phase will collect information on the long-term safety, tolerability, pharmacokinetics (PK), and efficacy of IB1001 as a treatment for NPC for the purpose of further assessing the risk/ benefit balance of the investigational medicinal product in the proposed clinical setting.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
19/EM/0094
Date of REC Opinion
20 Jun 2019
REC opinion
Further Information Favourable Opinion