Phase II Study of PBI-4050 in Subjects with Alström Syndrome
Research type
Research Study
Full title
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Tolerability of PBI-4050 and of its Effects on the Inflammatory, Fibrosis, Diabetes and Obesity Biomarkers in Subjects with Alström Syndrome
IRAS ID
186163
Contact name
Tarekegn Hiwot
Contact email
Sponsor organisation
Prometic Biosciences Inc
Eudract number
2015-001625-16
Duration of Study in the UK
1 years, 0 months, 1 days
Research summary
Alström Syndrome is a rare autosomal recessive genetic disorder, with an estimated prevalence of less than one per million. The syndrome is characterised by a multitude of clinical features. Two of the most important are progressive and severe fibrosis affecting the heart,liver, kidney and fat cells, and early onset Type 2 diabetes with severe insulin resistance.
The prognosis of Alström syndrome varies widely. However, life expectancy with the condition is generally reduced and subjects rarely survive beyond 45 years. There is no specific treatment for this genetic disease, and current treatment consists of only supportive therapy.
PBI-4050 has been developed as a potential drug candidate for the treatment of inflammatory/fibrosis-related diseases, including type 2 diabetes with metabolic syndrome. Pre-clinical testing has shown PBI-4050 to be effective in preventing or reversing fibrosis in multiple animal models studied, including models of fibrosis in kidney, heart, lung and liver. Furthermore, pre-clinical results (in mice models) have demonstrated PBI-4050 to be effective in reducing insulin resistance, normalising glycaemia (glucose in the blood) and maintaining (early treatment) or restoring (late treatment) insulin content in the pancreas.
Two Phase I studies have been completed in Canada, the first involving healthy subjects and the second in patients with advanced nephropathy due to type II diabetes. No serious adverse events were reported for doses up to 2,400mg.
This study is an open-label, single-centre, Phase II trial to evaluate the safety and tolerability of PBI-4050 in subjects with Alström syndrome. In order to assess the effects of PBI-4050 on fibrosis, diabetes, inflammation and weight, a number of assessments have been incorporated into the study, which include analysis of blood and urine for specific biomarkers.
Approximately 18 patients with documented diagnosis of Alström syndrome will be enrolled from one site UK national centre.REC name
East of England - Cambridgeshire and Hertfordshire Research Ethics Committee
REC reference
15/EE/0334
Date of REC Opinion
29 Oct 2015
REC opinion
Further Information Favourable Opinion