Cystic FibrosisPhase 2NCT02955888
Study of Safety, Tolerability & Efficacy in Cystic Fibrosis Patients With Abnormal Glucose Tolerance
A Phase 2 study testing PBI4050 in people with Cystic Fibrosis, run by Liminal BioSciences Ltd. at 7 sites in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- PBI4050
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 11 participants
- Where
- 7 sitesCanada
- Study dates
- 3 Jan 2017to Feb 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
- 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.
- 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.
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- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Canada7 sites
Queen Elizabeth II Health Science Center
Halifax, Nova Scotia
Institut de Recherches Cliniques de Montréal (IRCM)
Montreal, Quebec
Ottawa Hospital Research Institute
Ottawa, Ontario
Institut Universitaire de Cardiologie et de Pneumologie de l'Université Laval (IUCPQ)
Québec
Centre de Recherche du Centre Hospitalier Universitaire de Sherbrooke
Sherbrooke, Quebec
St. Michael's Hospital
Toronto, Ontario
St. Paul's Hospital - Pacific Lung Health Centre (PLHC)
Vancouver, British Columbia
About this study
Official 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
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.
Read the 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
- Being tested
- PBI4050, Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBI4050Drug
Investigational Medicinal Product
PlaceboDrug
Comparator
| Study group | Role | What they receive |
|---|---|---|
| PBI4050 | Experimental | Four 200 mg capsules (total 800 mg) administered orally, once daily. Receives: Drug: PBI4050 |
| Placebo | Placebo comparator | Four 200 mg capsules (total 800 mg) administered orally, once daily. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Measured: Up to 1 Year
Change from Baseline of insulin-secretion following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)
Measured: Up 1 Year
Change from Baseline of HbA1C following Oral Glucose Tolerance Test (OGTT) (Combined Main Study and Extension Study Participation)
Measured: Up to 1 Year
Change from Baseline in pulmonary function parameters (FEV1 ) (Combined Main Study and Extension Study Participation)
Measured: Up to 1 Year
Change from Baseline in pulmonary function parameters ( FVC) (Combined Main Study and Extension Study Participation)
Measured: Up to 1 Year
Change from Baseline in pulmonary function parameters (FEV1/FVC ratio) (Combined Main Study and Extension Study Participation)
Measured: Up to 1 Year
Change from Baseline in pulmonary function parameters (Forced Expiratory Flow (FEF25%-75%)) (Combined Main Study and Extension Study Participation)
Measured: Up to 1 Year
Change from baseline in weight (Combined Main Study and Extension Study Participation)
Measured: Up to1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2016 (estimated)
- Study start3 Jan 2017
- Primary completionFeb 2018
- Study completionFeb 2018
Why it stopped: Unsatisfactory rate of recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Liminal BioSciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- John Moran, MD, Study chair, Liminal BioSciences Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02955888
- Sponsor's protocol ID
- PBI-4050-CT-9-07
- Status last verified
- Dec 2020
- First submitted
- 21 Oct 2016
- Last updated
- 8 Dec 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02955888
- Acronym / sponsor ID
- PBI-4050-CT-9-07
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Unsatisfactory rate of recruitment
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Liminal BioSciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 3 Jan 2017 (actual)
- Primary completion
- Feb 2018 (actual)
- Study completion
- Feb 2018 (actual)
- First posted
- 4 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PBI4050 | Experimental | Drug: PBI4050Four 200 mg capsules (total 800 mg) administered orally, once daily. |
| Placebo | Placebo comparator | Drug: PlaceboFour 200 mg capsules (total 800 mg) administered orally, once daily. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (14 listed)
- 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.
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PBI4050Investigational Medicinal Product | Drug | PBI4050 |
| PlaceboComparator | Drug | Placebo |
Primary outcome
Number of Subjects with Adverse Events (Combined Main Study and Extension Study Participation)
Time frame: Baseline to 1 Year
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.
8 secondary outcomes
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
Results and safety
The study was stopped early (reason given: Unsatisfactory rate of recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Moran, MD study chair
Liminal BioSciences Ltd.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PBI4050 in people with Cystic Fibrosis, run by Liminal BioSciences Ltd. at 7 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Unsatisfactory rate of recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects with Adverse Events (Combined Main Study and Extension Study Participation) (time frame: Baseline to 1 Year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2020.