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

NCT03184584 | Terminated Phase 2 DMC

• 1 other identifier: PBI-4050-CT-9-10
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Alström Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment

  96 weeks

Secondary Outcomes (5)

• 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

  96 weeks

Study Arms (1)

PBI-4050

EXPERIMENTAL

Drug: PBI-4050

Interventions

PBI-4050 DRUG

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

PBI-4050

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Liminal BioSciences Ltd.: lead

Study Sites (1)

University Hospitals Birmingham NHS Foundation Trus

Birmingham, B15 2PR, United Kingdom

Location

MeSH Terms

Conditions

Alstrom Syndrome

Interventions

setogepram

Condition Hierarchy (Ancestors)

Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Retinitis Pigmentosa; Eye Diseases, Hereditary; Eye Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2017
• First Posted: June 12, 2017
• Study Start: October 9, 2017
• Primary Completion: May 8, 2020
• Study Completion: May 8, 2020
• Last Updated: November 3, 2020

Record last verified: 2020-10

Locations

GB (1)