Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome

Study Overview
=================
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.

Official Title
-----------------
An Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome

Conditions
-----------------
Alström Syndrome

Intervention / Treatment
-----------------
* Drug: PBI-4050


Participation Criteria
=================
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.

Ages Eligible for Study
-----------------
Minimum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PBI-4050<br> | Drug: PBI-4050<br>* Four 200 mg capsules (800 mg total) administered orally, once daily<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment |  | 96 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in fasting plasma glucose over time |  | 96 weeks | 
| Change from baseline in plasma insulin over time |  | 96 weeks | 
| Change from baseline in glycated hemoglobin (HbA1c) over time |  | 96 weeks | 
| Change from baseline in blood glucose as measured by weekly 4 point profile |  | 96 weeks | 
| Change from baseline in the liver stiffness | Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan | 96 weeks |