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

NCT ID: NCT03184584

Last Updated: 2020-11-03

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-10-09
• Study Completion Date: 2020-05-08

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.

Conditions

Alström Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PBI-4050

Group Type: EXPERIMENTAL

PBI-4050

Intervention Type: DRUG

Four 200 mg capsules (800 mg total) administered orally, once daily

Interventions

PBI-4050

Four 200 mg capsules (800 mg total) administered orally, once daily

Intervention Type: DRUG

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.

• Minimum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Liminal BioSciences Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

University Hospitals Birmingham NHS Foundation Trus

Birmingham, , United Kingdom

Countries

United Kingdom

Other Identifiers

PBI-4050-CT-9-10

Identifier Type: -

Identifier Source: org_study_id