Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Primary Purpose
Alström Syndrome
Status
Terminated
Phase
Phase 2
Locations
United Kingdom
Study Type
Interventional
Intervention
PBI-4050
Sponsored by
About this trial
This is an interventional treatment trial for Alström Syndrome
Eligibility Criteria
Inclusion Criteria:
- Subject completed a preceding ProMetic-sponsored Alström syndrome study with PBI-4050.
- Subject has signed informed consent
- Subject has a documented diagnosis of Alström syndrome
- Subject receiving antidiabetic medications is able and willing to self-monitor blood glucose levels
- Subject must be willing to forego other forms of experimental drug treatment during the study.
- Female subjects of childbearing potential must have a negative serum pregnancy test at screening and agree to use adequate birth control from screening throughout the study and for 30 days after the last Investigational Medicinal Product (IMP) administration
- If a male subject 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 30 days after the last IMP administration.
Exclusion Criteria:
- Subject discontinued PBI-4050 for safety reasons from any preceding ProMetic-sponsored Alström syndrome study with PBI-4050
- Subject has had a documented episode of severe hypoglycaemia within 12 months before screening and investigator judges that the subject is unable to adequately monitor their glucose levels.
- Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
- Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at screening
- Subject has any condition that, in the investigator's opinion, is likely to interfere with study conduct and compliance
- Subject has a history of an allergic reaction to PBI-4050 or any of its excipients.
Sites / Locations
- University Hospitals Birmingham NHS Foundation Trus
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
PBI-4050
Arm Description
Outcomes
Primary Outcome Measures
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
Secondary Outcome Measures
Change from baseline in fasting plasma glucose over time
Change from baseline in plasma insulin over time
Change from baseline in glycated hemoglobin (HbA1c) over time
Change from baseline in blood glucose as measured by weekly 4 point profile
Change from baseline in the liver stiffness
Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan
Full Information
NCT ID
NCT03184584
First Posted
June 9, 2017
Last Updated
October 30, 2020
Sponsor
Liminal BioSciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03184584
Brief Title
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Official Title
An Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
Terminated
Why Stopped
Terminated early due to redeployment of study site staff during Covid-19 pandemic
Study Start Date
October 9, 2017 (Actual)
Primary Completion Date
May 8, 2020 (Actual)
Study Completion Date
May 8, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Liminal BioSciences Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050.
Detailed Description
This is a Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed the end-of-treatment (EoT) visit in a preceding ProMetic-sponsored Alström Syndrome study with PBI-4050. This study will also evaluate the efficacy and pharmacological effects of PBI 4050 in this multi faceted disorder. Approximately 20 to 30 subjects will be enrolled to receive 800 mg PBI-4050 once daily for 96 weeks or until product licensing or study termination by the Sponsor, whichever occurs first.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alström Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
PBI-4050
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
PBI-4050
Intervention Description
Four 200 mg capsules (800 mg total) administered orally, once daily
Primary Outcome Measure Information:
Title
Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
Time Frame
96 weeks
Secondary Outcome Measure Information:
Title
Change from baseline in fasting plasma glucose over time
Time Frame
96 weeks
Title
Change from baseline in plasma insulin over time
Time Frame
96 weeks
Title
Change from baseline in glycated hemoglobin (HbA1c) over time
Time Frame
96 weeks
Title
Change from baseline in blood glucose as measured by weekly 4 point profile
Time Frame
96 weeks
Title
Change from baseline in the liver stiffness
Description
Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan
Time Frame
96 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subject completed a preceding ProMetic-sponsored Alström syndrome study with PBI-4050.
Subject has signed informed consent
Subject has a documented diagnosis of Alström syndrome
Subject receiving antidiabetic medications is able and willing to self-monitor blood glucose levels
Subject must be willing to forego other forms of experimental drug treatment during the study.
Female subjects of childbearing potential must have a negative serum pregnancy test at screening and agree to use adequate birth control from screening throughout the study and for 30 days after the last Investigational Medicinal Product (IMP) administration
If a male subject 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 30 days after the last IMP administration.
Exclusion Criteria:
Subject discontinued PBI-4050 for safety reasons from any preceding ProMetic-sponsored Alström syndrome study with PBI-4050
Subject has had a documented episode of severe hypoglycaemia within 12 months before screening and investigator judges that the subject is unable to adequately monitor their glucose levels.
Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.
Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at screening
Subject has any condition that, in the investigator's opinion, is likely to interfere with study conduct and compliance
Subject has a history of an allergic reaction to PBI-4050 or any of its excipients.
Facility Information:
Facility Name
University Hospitals Birmingham NHS Foundation Trus
City
Birmingham
ZIP/Postal Code
B15 2PR
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
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