NCT02955888

Study of Safety, Tolerability & Efficacy in Cystic Fibrosis Patients With Abnormal Glucose Tolerance

A Phase 2, Double-Blind, Placebo Controlled Study to Evaluate the Safety & Tolerability of PBI-4050 and Its Effects on Pancreatic and Pulmonary Function in Cystic Fibrosis Patients With Abnormal Glucose Tolerance

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Liminal BioSciences Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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).

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	PBI4050	Investigational Medicinal Product
DRUG	Placebo	Comparator

Timeline

• Start date: 2017-01-03
• Primary completion: 2018-02-01
• Completion: 2018-02-01
• First posted: 2016-11-04
• Last updated: 2020-12-08

Locations

7 sites across 1 country: Canada