Alström Syndrome MedDRA version: 20.0 Level: LLT Classification code 10068814 Term: Alstrom syndrome System Organ Class: 100000004850
Conditions
Interventions
Product Name: PBI-4050
Pharmaceutical Form: Capsule, soft
CAS Number: 1254472-97-3
Current Sponsor code: PBI-4050
Other descriptive name: 3-pentylbenzeneacetic acid sodium salt
Concentration unit: mg
Sponsors
Prometic Biosciences Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject completed the End of Treatment visit of the preceding Prometic-sponsored ALMS study with PBI-4050. 2. Subject has signed informed consent. 3. Subject has a documented diagnosis of ALMS. 4. Subject must be willing to forego other forms of experimental drug treatment during the study. 5. Female subjects of child-bearing potential who are sexually active with a nonsterile male partner must agree to use adequate birth control throughout the study and for 30 days after the last dose of PBI-4050. Male subjects who have not been vasectomized and partner with a woman of childbearing potential must be willing to use an acceptable contraceptive method throughout the study and for 30 days after the last dose of PBI-4050. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Subject discontinued PBI-4050 for safety reasons from any preceding Prometic-sponsored ALMS study with PBI-4050. 2. Subject has had a documented episode of severe hypoglycaemia within 12 months before the Week 1, Day 1 visit and investigator judges that the subject is unable to adequately monitor his/her glucose levels in accordance with local standard practice. Severe hypoglycaemia is defined as an episode of confirmed hypoglycaemia (glucose < 3.0 mmol/L) that required parenteral treatment with intramuscular injection of glucagon or intravenous injection of dextrose, an episode of confirmed hypoglycaemia that did not require parenteral treatment but involved severe neuroglycopenic symptoms, or an episode of unconfirmed hypoglycaemia that resulted in seizure or coma. 3. Subject has uncontrolled hypertension with 170/100 mm Hg at the Week 1, Day 1 visit that, in the judgment of the investigator, makes the subject inappropriate for entry into the study. 4. Woman who is pregnant, breast-feeding, or planning a pregnancy during the course of the study as determined at the Week 1, Day 1 visit. 5. Subject has any condition that, in the investigator’s opinion, is likely to interfere with study conduct and compliance. 6. Subject has a history of an allergic reaction to PBI-4050 or any of its excipients.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the long-term safety and tolerability of PBI-4050, administered orally once daily, in subjects with Alström syndrome (ALMS) who have completed treatment in a preceding Prometic-sponsored ALMS study with PBI-4050. ;Secondary Objective: The secondary objectives are: - To evaluate the effect of PBI-4050 on metabolic syndrome parameters (fasting plasma glucose, fasting insulin, and HbA1c) - To assess the effect of PBI-4050 on liver stiffness using transient elastography (FibroScan®) - To assess the effect of PBI-4050 on the fat content and fibrosis burden in the liver using magnetic resonance imaging (MRI) - To assess the effect of PBI-4050 on cardiac fibrosis using MRI ;Primary end point(s): The primary endpoint is safety of PBI-4050. The following safety variables will be analysed descriptively. • Adverse Events (AEs): All AEs will be coded to a Preferred Term and associated Lower Level Term, and System Organ Class according to the Medical Dictionary for Regulation Activities (MedDRA version 18.0) before the database lock. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The following secondary endpoints will be analysed for efficacy and summarized descriptively at each time point as applicable. • Changes from baseline in metabolic syndrome parameters: o FPG o HbA1c o Fasting insulin o C-peptide o HOMA-B and HOMA-S • Change from baseline in the liver stiffness (measured in kPa correlated to fibrosis) by using a transient elastography (FibroScan) • Change from baseline in the fat content and fibrosis burden in liver MRI • Change from baseline in cardiac fibrosis on cardiac MRI. | — |
Countries
United Kingdom
Contacts
Public ContactCatherine Simcock
Prometic Biosciences Inc.
Outcome results
None listed