NCT03184584

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

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Liminal BioSciences Ltd. · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

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

Interventions

Type	Name	Description
DRUG	PBI-4050	Four 200 mg capsules (800 mg total) administered orally, once daily

Timeline

• Start date: 2017-10-09
• Primary completion: 2020-05-08
• Completion: 2020-05-08
• First posted: 2017-06-12
• Last updated: 2020-11-03

Locations

1 site across 1 country: United Kingdom