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

• Register: ClinicalTrials.gov
• Last refreshed on: 11 January 2021
• Main ID: NCT02955888
• Date of registration: 21/10/2016
• Prospective Registration: Yes
• Primary sponsor: Liminal BioSciences Ltd.
• Public title: Study of Safety, Tolerability & Efficacy in Cystic Fibrosis Patients With Abnormal Glucose Tolerance
• Scientific title: 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
• Date of first enrolment: January 3, 2017
• Target sample size: 11
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02955888
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Canada

Contacts

• Name: John Moran, MD
• Affiliation: Liminal BioSciences Ltd.

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Placebo

Drug: PBI4050

Primary Outcome(s)

Number of Subjects with Adverse Events (Combined Main Study and Extension Study Participation) [Time Frame: Baseline to 1 Year]

Secondary Outcome(s)

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 pulmonary function parameters ( FVC) (Combined Main Study and Extension Study Participation) [Time Frame: Up 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 in pulmonary function parameters (FEV1/FVC ratio) (Combined Main Study and Extension Study Participation) [Time Frame: Up to 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 of insulin-secretion following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation) [Time Frame: Up 1 Year]

Change from baseline in weight (Combined Main Study and Extension Study Participation) [Time Frame: Up to1 Year]

Secondary ID(s)

PBI-4050-CT-9-07

Source(s) of Monetary Support

Please refer to primary and secondary sponsors