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

Study Description

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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.

Condition or disease	Intervention/treatment	Phase
Alström Syndrome	Drug: PBI-4050	Phase 2; Phase 3

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 Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	20 participants
Intervention Model:	Single Group Assignment
Masking:	None (Open Label)
Primary Purpose:	Treatment
Official Title:	An Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Actual Study Start Date :	October 9, 2017
Estimated Primary Completion Date :	July 2020
Estimated Study Completion Date :	September 2020

Arms and Interventions

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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

Outcome Measures

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Primary Outcome Measures :

1. Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment [ Time Frame: 96 weeks ]

Secondary Outcome Measures :

1. Change from baseline in fasting plasma glucose over time [ Time Frame: 96 weeks ]
2. Change from baseline in plasma insulin over time [ Time Frame: 96 weeks ]
3. Change from baseline in glycated hemoglobin (HbA1c) over time [ Time Frame: 96 weeks ]
4. Change from baseline in blood glucose as measured by weekly 4 point profile [ Time Frame: 96 weeks ]
5. Change from baseline in the liver stiffness [ Time Frame: 96 weeks ]
   Measured in kilopascal (kPa) correlated to fibrosis by using a FibroScan

Eligibility Criteria

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Ages Eligible for Study:	16 Years and older (Child, Adult, Senior)
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Locations

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

Sponsors and Collaborators

ProMetic BioSciences Inc.

More Information

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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party:	ProMetic BioSciences Inc.
ClinicalTrials.gov Identifier:	NCT03184584 History of Changes
Other Study ID Numbers:	PBI-4050-CT-9-10
First Posted:	June 12, 2017
Last Update Posted:	April 10, 2018
Last Verified:	April 2018

Studies a U.S. FDA-regulated Drug Product:		No
Studies a U.S. FDA-regulated Device Product:		No

Additional relevant MeSH terms:

Syndrome; Alstrom Syndrome; Disease; Pathologic Processes; 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; Genetic Diseases, Inborn