This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks. Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks, provided the subject signs informed consent.
This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome. Approximately 18 subjects will be enrolled. The duration of study participation is approximately 35 weeks for each subject and comprises of 9 on site visits and telephone contacts in between visits. Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks (Extension Period \[EP\]), provided the subject signs informed consent. The extension period includes a further 3 on site visits and telephone contacts in between visits. The total duration of the study participation is extended to approximately 71 or 83 weeks. The Data Safety Monitoring Board (DSMB) will determine if the safety data continue to support treatment for an additional 36 or 48 weeks. At the completion of the EP End of Treatment, subjects will be allowed to enrol in the Alström Rollover Study PBI-4050-CT-9-10 and continue ongoing study medication without any break in treatment.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Four 200 mg capsules (800 mg total) administered orally, once daily.
University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
Description and number of abnormal laboratory values and adverse events that are related to treatment.
Time frame: Primary on 24 weeks; Final on all data (including Extension Period)
Change from baseline in metabolic syndrome parameters over time.
Change from baseline in fasting plasma glucose over time. Change from baseline in fasting plasma insulin over time. Change from baseline in glycated hemoglobin (HbA1c) over time. Change from baseline in Homeostasis Model Assessment for steady state beta cell function (HOMA-B) and insulin sensitivity (HOMA-S) over time.
Time frame: 24 weeks and end of Extension Period
Change from baseline in biomarkers in blood and urine over time
Percentage of reduction and/or increase of level of biomarkers
Time frame: 24 weeks and end of Extension Phase
Change from baseline in cardiac function parameter: NT-proBNP
Time frame: 24 weeks and end of Extension Phase
Change from baseline of antidiabetic treatment
Dosing change, new medication added or treatment discontinuation
Time frame: 24 weeks and end of Extension Phase
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