Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050.
This is a Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed the end-of-treatment (EoT) visit in a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050. This study will also evaluate the efficacy and pharmacological effects of PBI 4050 in this multi faceted disorder. Approximately 20 to 30 subjects will be enrolled to receive 800 mg PBI-4050 once daily for 96 weeks or until product licensing or study termination by the Sponsor, whichever occurs first.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Four 200 mg capsules (800 mg total) administered orally, once daily
University Hospitals Birmingham NHS Foundation Trus
Birmingham, United Kingdom
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
Time frame: 96 weeks
Change from baseline in fasting plasma glucose over time
Time frame: 96 weeks
Change from baseline in plasma insulin over time
Time frame: 96 weeks
Change from baseline in glycated hemoglobin (HbA1c) over time
Time frame: 96 weeks
Change from baseline in blood glucose as measured by weekly 4 point profile
Time frame: 96 weeks
Change from baseline in the liver stiffness
Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan
Time frame: 96 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.