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

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

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.

Study Overview

• Status: Terminated
• Conditions: Alström Syndrome
• Intervention / Treatment: Drug: 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.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B15 2PR
    • University Hospitals Birmingham NHS Foundation Trus

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Liminal BioSciences Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): October 9, 2017
• Primary Completion (Actual): May 8, 2020
• Study Completion (Actual): May 8, 2020

Study Registration Dates

• First Submitted: June 9, 2017
• First Submitted That Met QC Criteria: June 9, 2017
• First Posted (Actual): June 12, 2017

Study Record Updates

• Last Update Posted (Actual): November 3, 2020
• Last Update Submitted That Met QC Criteria: October 30, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Eye Diseases; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Neuromuscular Diseases; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Eye Diseases, Hereditary; Heredodegenerative Disorders, Nervous System; Nervous System Malformations; Abnormalities, Multiple; Ciliopathies; Polyneuropathies; Retinitis Pigmentosa; Hereditary Sensory and Motor Neuropathy; Syndrome; Alstrom Syndrome
• Other Study ID Numbers: PBI-4050-CT-9-10

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No