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

NCT02955888 | Terminated Phase 2 DMC

• 1 other identifier: PBI-4050-CT-9-07
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 7

Brief 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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline to 1 Year

Secondary Outcomes (8)

• Change from Baseline in glucose level following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)

  Up to 1 Year

• Change from Baseline of insulin-secretion following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)

  Up 1 Year

• Change from Baseline of HbA1C following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)

  Up to 1 Year

• Change from Baseline in pulmonary function parameters (FEV1 ) (Combined Main Study and Extension Study Participation)

  Up to 1 Year

• Change from Baseline in pulmonary function parameters ( FVC) (Combined Main Study and Extension Study Participation)

  Up to 1 Year

Other Outcomes (2)

• Changes from baseline in fibrotic and inflammatory biomarkers in blood (Combined Main Study and Extension Study Participation)

  Up to 1 Year

• Changes from baseline in fibrotic and inflammatory biomarkers in urine (Combined Main Study and Extension Study Participation)

  Up to1 Year

Study Arms (2)

PBI4050

EXPERIMENTAL

Four 200 mg capsules (total 800 mg) administered orally, once daily.

Drug: PBI4050

Placebo

PLACEBO COMPARATOR

Four 200 mg capsules (total 800 mg) administered orally, once daily.

Drug: Placebo

Interventions

PBI4050 DRUG

Investigational Medicinal Product

PBI4050

Placebo DRUG

Comparator

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient is 18 years of age or older at screening.
• Patient has a documented and confirmed CF diagnosis.
• Patient has performed an OGTT for diagnosis of INDENT, IGT and de novo CFRD within 12 months prior to screening visit.
• Patient has a Body Mass Index (BMI) of at least 17 kg/m2.
• Patient has signed written informed consent.
• Patient is able and willing to self-monitor blood glucose level at home.
• Female patients of childbearing potential must have a negative pregnancy test (serum or urine) and agree to use adequate birth control from screening throughout the study and for the 30 days after the last study drug administration.
• If a male patient has not been vasectomized at least 6 months before screening and partners with a woman of childbearing potential, he must be willing to use an acceptable contraceptive method throughout the study and for the 30 days after the last study drug administration.

You may not qualify if:

• Patient has recent or ongoing infection requiring intravenous treatment with an anti-infective agent within 30 days before screening.
• Patient is concurrently taking high dose of ibuprofen (\>30 mg/kg) or is using corticosteroids (except inhaled and topical corticosteroids).
• Patient is currently using weight-loss medications.
• \. Patient has used any moderate/potent inhibitor of cytochrome P450 (CYP) 2C9 isozyme or strong inhibitor of CYP3A isozyme within 30 days prior to the first study drug administration.
• Patient has significantly elevated liver enzyme levels, defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 2.5 Upper Limit of Normal (ULN) or total bilirubin above ULN at screening.
• Patient has a history of chronic alcohol or other substance abuse as determined at screening that may prevent study compliance based on Investigator judgment.
• History of malignancy of any organ system, treated or untreated, within the past 5 years other than basal or squamous cell skin cancer.
• Patient has unstable chronic heart failure that has required change in therapy within 2 months prior to screening.
• Patient with known non-controlled history of infection with Human Immunodeficiency Virus (HIV) and/or active Hepatitis.
• Woman who is pregnant, breast-feeding or planning a pregnancy during the course of the study.
• Woman of childbearing potential who is unwilling to use adequate birth control throughout the duration of the study.
• Patient has any condition that, in the Investigator's opinion, is likely to interfere with study conduct and compliance.
• Patient has participated in an investigational clinical trial within 30 days (or 5 half-lives, whichever is longer) prior to screening visit.
• Patient is under insulin and/or repaglinide treatment at screening/baseline

Sponsors & Collaborators

• Liminal BioSciences Ltd.: lead

Study Sites (7)

St. Paul's Hospital - Pacific Lung Health Centre (PLHC)

Vancouver, British Columbia, Canada

Location

Queen Elizabeth II Health Science Center

Halifax, Nova Scotia, Canada

Location

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Location

St. Michael's Hospital

Toronto, Ontario, Canada

Location

Institut de Recherches Cliniques de Montréal (IRCM)

Montreal, Quebec, Canada

Location

Centre de Recherche du Centre Hospitalier Universitaire de Sherbrooke

Sherbrooke, Quebec, Canada

Location

Institut Universitaire de Cardiologie et de Pneumologie de l'Université Laval (IUCPQ)

Québec, Canada

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

setogepram

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• John Moran, MD

  Liminal BioSciences Ltd.

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2016
• First Posted: November 4, 2016
• Study Start: January 3, 2017
• Primary Completion: February 1, 2018
• Study Completion: February 1, 2018
• Last Updated: December 8, 2020

Record last verified: 2020-12

Data Sharing

• IPD Sharing: Will not share

Locations

CA (7)