This is a Phase 2, multi-center, double blind, placebo controlled study to evaluate the safety and tolerability of PBI-4050, and its effects on the pancreatic, pulmonary functions and on various biomarkers in Cystic Fibrosis patients with abnormal glucose tolerance. Patients with abnormal glucose tolerance have elevated glucose level either at 1 hour or 2 hour during an Oral Glucose Tolerance Test (OGTT). The Main study will include 24 weeks of treatment with PBI-4050 or matching placebo. At the end of the treatment period, patients will have the option of participating in a 24-week Extension study.
This is a Phase 2, multi-center, double blind, placebo controlled study to evaluate the safety and tolerability of PBI-4050, and its effects on the pancreatic, pulmonary functions and on various biomarkers in Cystic Fibrosis patients with abnormal glucose tolerance. Patients with abnormal glucose tolerance have elevated glucose level either at 1 hour or 2 hour during an Oral Glucose Tolerance Test (OGTT). A total of 90 patients will be enrolled for study participation. A Data Safety Monitoring Board (DSMB) will continually review individual patients safety data obtained from the 90 patients. When the first 15 patients have completed at least 1 month of study treatment, the DSMB will meet formally to determine whether additional patients may be enrolled, the study should continue with changes or if the study should be stopped. In addition, the DSMB will review the PK data and may recommend dose adjustment based on the PK results. The total duration of study participation for each patient is at least 32 weeks, including up to 4 weeks of a screening period, 24 weeks of study treatment and 4 weeks of safety follow-up. Patients who choose to participate in the open label extension will be in the study for an additional 24 weeks of study treatment and 4 weeks of safety follow-up (for a total of 56 weeks).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
11
St. Paul's Hospital - Pacific Lung Health Centre (PLHC)
Vancouver, British Columbia, Canada
Queen Elizabeth II Health Science Center
Halifax, Nova Scotia, Canada
Ottawa Hospital Research Institute
Ottawa, Ontario, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
Institut de Recherches Cliniques de Montréal (IRCM)
Montreal, Quebec, Canada
Centre de Recherche du Centre Hospitalier Universitaire de Sherbrooke
Sherbrooke, Quebec, Canada
Institut Universitaire de Cardiologie et de Pneumologie de l'Université Laval (IUCPQ)
Québec, Canada
Number of Subjects with Adverse Events (Combined Main Study and Extension Study Participation)
Adverse Event data (including abnormal laboratory values) collected up to final follow-up (30 days after last dose of study drug). Serious Adverse Events that were ongoing at the follow-up visit will be followed until the event resolved, returned to baseline, or was determined to be a stable or chronic condition.
Time frame: Baseline to 1 Year
Change from Baseline in glucose level following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from Baseline of insulin-secretion following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)
Time frame: Up 1 Year
Change from Baseline of HbA1C following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from Baseline in pulmonary function parameters (FEV1 ) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from Baseline in pulmonary function parameters ( FVC) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from Baseline in pulmonary function parameters (FEV1/FVC ratio) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from Baseline in pulmonary function parameters (Forced Expiratory Flow (FEF25%-75%)) (Combined Main Study and Extension Study Participation)
Time frame: Up to 1 Year
Change from baseline in weight (Combined Main Study and Extension Study Participation)
Time frame: Up to1 Year
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