A Rollover Study of PBI-4050 in Subjects with Alström Syndrome
Research type
Research Study
Full title
An Open-Label Rollover Study of PBI-4050 in Subjects with Alström Syndrome
IRAS ID
224450
Contact name
Tarekegn Hiwot
Contact email
Sponsor organisation
ProMetic BioSciences Inc.
Eudract number
2016-004823-21
Duration of Study in the UK
2 years, 8 months, 15 days
Research summary
Alström Syndrome (ALMS) is a rare autosomal recessive genetic disorder, with an estimated prevalence of less than one per million. The syndrome is characterised by the progressive development of multi-organ fibrosis, as well as a number of other clinical features. Wide clinical variability is observed among individuals with ALMS, even within the same family. Symptoms first typically appear in infancy and development of multi-organ pathology leads to a much-reduced life expectancy, rarely exceeding 50 years. There is no specific treatment for this genetic disease, with current treatment consisting of supportive therapy.
PBI-4050 has been developed as a potential drug candidate for the treatment of inflammatory/fibrosis-related diseases. A Phase 2 study is currently ongoing within the UK, which involves ALMS patients receiving once daily dosing of PBI-4050 (up to 800mg). Preliminary efficacy results have shown an improvement in liver fibrosis. Furthermore, preliminary safety data has not identified any significant risks being observed with the use of PBI-4050.
This is a Phase 2, open-label, rollover study that aims to evaluate the safety and tolerability of PBI-4050 in patients with ALMS, who have completed treatment in preceding ProMetic-sponsored ALMS studies. At present, only one study in the UK is ongoing (PBI-4050-ATX-9-05), from which subjects can rollover and be eligible for this study. However, the study design allows for ALMS patients to be included from future ProMetic-sponsored ALMS studies (with PBI-4050).
Approximately 20-30 ALMS patients can be enrolled from ProMetic-sponsored ALMS studies, globally. Approximately 12 patients are expected to be enrolled from the UK.
REC name
South Central - Berkshire B Research Ethics Committee
REC reference
17/SC/0179
Date of REC Opinion
20 Apr 2017
REC opinion
Favourable Opinion