Safety and Tolerability of PBI-4050 and Its Effects on the Biomarkers in Subjects With Alström Syndrome

• Status: Completed
• Trial end date: 2018-06-04
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome for a treatment duration of 24 weeks.

Subjects who complete the initial 24 weeks of treatment may continue treatment for an additional 36 or 48 weeks, provided the subject signs informed consent.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Liminal BioSciences Ltd.; ProMetic BioSciences Inc.

Criteria

Inclusion Criteria :

• Subject is 16 years of age or older at screening.

• Subject has signed informed consent.

• Subject has a documented diagnosis of Alström syndrome

• Subject on diabetes treatment has been receiving the same antidiabetic agent(s) for a minimum of 1 month before screening.

• Subject is able and willing to self-monitor blood glucose level at home or can obtain adequate assistance from care givers.

• 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 has recent or on-going infection requiring systemic treatment with an anti-infective agent within 30 days before screening.

• Subject has had at least two documented episodes of severe hypoglycaemia within 12 months before screening

• Subject has uncontrolled hypertension with BP > 170/100 mmHg as determined at screening.

• Subject has alanine transaminase (ALT) or aspartate transaminase (AST) level ≥ 5 × upper limit of normal (ULN) at screening.

• Subject is currently using weight loss medications at screening. Subjects may be re-screened after stopping the weight loss medication for a period of at least 5 half-lives.

• Subject has used any moderate/potent inducer or inhibitor of CYP2C9 isozyme or strong inducer or inhibitor of cytochrome P450 (CYP) 3A isozyme within 30 days prior to the first study drug administration.

• Subject has a history of chronic alcohol or other substance abuse 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.