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

NCT ID: NCT02955888

Last Updated: 2020-12-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-03
• Study Completion Date: 2018-02-28

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.

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

PBI4050

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

Group Type: EXPERIMENTAL

PBI4050

Intervention Type: DRUG

Investigational Medicinal Product

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Comparator

Interventions

PBI4050

Investigational Medicinal Product

Intervention Type: DRUG

Placebo

Comparator

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Patient is 18 years of age or older at screening.

2. Patient has a documented and confirmed CF diagnosis.

3. Patient has performed an OGTT for diagnosis of INDENT, IGT and de novo CFRD within 12 months prior to screening visit.

4. Patient has a Body Mass Index (BMI) of at least 17 kg/m2.

5. Patient has signed written informed consent.

6. Patient is able and willing to self-monitor blood glucose level at home.

7. 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.

8. 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.

Exclusion Criteria

1. Patient has recent or ongoing infection requiring intravenous treatment with an anti-infective agent within 30 days before screening.

2. Patient is concurrently taking high dose of ibuprofen (>30 mg/kg) or is using corticosteroids (except inhaled and topical corticosteroids).

3. Patient is currently using weight-loss medications.

4. 4. 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.

5. 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.

6. Patient has a history of chronic alcohol or other substance abuse as determined at screening that may prevent study compliance based on Investigator judgment.

7. History of malignancy of any organ system, treated or untreated, within the past 5 years other than basal or squamous cell skin cancer.

8. Patient has unstable chronic heart failure that has required change in therapy within 2 months prior to screening.

9. Patient with known non-controlled history of infection with Human Immunodeficiency Virus (HIV) and/or active Hepatitis.

10. Woman who is pregnant, breast-feeding or planning a pregnancy during the course of the study.

11. Woman of childbearing potential who is unwilling to use adequate birth control throughout the duration of the study.

12. Patient has any condition that, in the Investigator's opinion, is likely to interfere with study conduct and compliance.

13. Patient has participated in an investigational clinical trial within 30 days (or 5 half-lives, whichever is longer) prior to screening visit.

14. Patient is under insulin and/or repaglinide treatment at screening/baseline

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Liminal BioSciences Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

John Moran, MD

Role: STUDY_CHAIR

Liminal BioSciences Ltd.

Locations

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

Countries

Canada

Other Identifiers

PBI-4050-CT-9-07

Identifier Type: -

Identifier Source: org_study_id