Hypertriglyceridemia Clinical Trial
Official title:
An Investigator-Initiated Open-Label, Randomized Study of Gemcabene in Adults With Familial Partial Lipodystrophy Disease (FPLD)
Verified date | July 2020 |
Source | University of Michigan |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The overall objective of this study is to assess the efficacy and safety of two dosing regimens of gemcabene (300 mg once daily for 24 weeks or 300 mg daily for 12 weeks followed by 600 mg daily for 12 weeks) in up to eight patients with Familial Partial Lipodystrophy with high triglycerides and Non-Alcoholic Fatty Liver Disease. The study will consist of a six week Wash Out Period, up to a 28 day Screening Period, a 24 week Treatment Period, and a follow-on safety assessment four weeks post final dose. Study participation will last approximately 4 months and includes at least 9 study visits, and can be as many as 11 study visits.
Status | Completed |
Enrollment | 5 |
Est. completion date | July 31, 2019 |
Est. primary completion date | July 31, 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
- Clinical diagnosis of lipodystrophy based on a lack of body fat in a partial fashion
assessed by physical examination, and at least 1 MAJOR criterion (below): - Low skinfold thickness in anterior thigh by caliper measurement: men (= 10 mm) and women (= 22 mm) OR - Historic genetic diagnosis of familial partial lipodystrophy (e.g. mutations in LMNA, PPAR-?, AKT2, or PLIN1 genes) as supported by source documentation - Hepatic steatosis (>10% - Stage 2 or 3) as demonstrated by MRI-PDFF; - Alcohol intake of less than 20 g per day in females and 30 g per day in males (one 12 oz beer, one glass of wine, or 2 oz of spirits or liquor equals roughly 10 g of alcohol; - Mean fasting triglyceride value = 250 mg/dL at the Screening Visit; - Background lipid lowering medications must be stable for at least 6 weeks prior to the Screening Visit; - Women patients must not be pregnant or lactating and women of child-bearing potential must agree to use acceptable methods of contraception throughout the duration of the study and for 30 days after the last dose of study drug. Male patients must agree to use contraception by means of a condom and may not donate sperm throughout the duration of the study and for 8 days after the last dose of study drug. - Weight greater than 50 kg (~110 lbs); with a body mass index (BMI) of no more than 45 kg/m²; - Have not used a fibrate with in the last 6 weeks and/or thiazolidinediones (TZDs) within the last 12 weeks prior to the Screening visit. - Do not have a hypersensitivity or a history of significant reactions of fibrates. - Are not currently taking potent CYP3A4 inhibitors such as itraconazole or a macrolide antibiotic. - Have a condition or finding which, in the opinion of the Investigator, would compromise the patient's safety or participation in the study. |
Country | Name | City | State |
---|---|---|---|
United States | University of Michigan | Ann Arbor | Michigan |
Lead Sponsor | Collaborator |
---|---|
Elif Oral |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in Fasting Serum Triglyceride (at 12 Weeks) | This is measured by percent change in fasting serum triglyceride from baseline to week 12 | Baseline to week 12 | |
Secondary | Change in Fasting Serum Triglycerides (Through 24 Weeks) | This is measured by change in fasting serum triglyceride from baseline to average of weeks 6 and 12, and week 24 and change in fasting serum triglyceride from baseline to week 12 | Baseline, week 6 and week 12, week 24 | |
Secondary | Percent Change in Fasting Serum Triglycerides (Through 24 Weeks) | This is measured by percent change in fasting serum triglyceride from baseline to average of weeks 6 and 12, and week 24 and change in fasting serum triglyceride from baseline to week 12 | Baseline, week 6 and week 12, week 24 | |
Secondary | Change in Liver Fat Content as Measured by Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF) | This is measured by change in liver fat content using Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF) from baseline to week 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Percent Change in Liver Fat Content as Measured by Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF) | This is measured by percent change in liver fat content using Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF) from baseline to week 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Change in Liver Fibrosis | This is measured by change in liver fibrosis using MR-elastography from baseline to week 12 and week 24 | Baseline, Week 12, and Week 24 | |
Secondary | Percent Change in Liver Fibrosis | This is measured by percent change in liver fibrosis using MR-elastography from baseline to week 12 and week 24 | Baseline, Week 12, and Week 24 | |
Secondary | Change in NAS (Non-alcoholic Steatohepatitis) | This is measured by change in NAS via non-alcoholic fatty liver disease activity score. NAS is the unweighted sum of steatosis, lobular inflammation and hepatocyte ballooning from baseline to week 24. Total NAS scores can range from 0 to 8. The higher the NAS score, the more severe the liver disease. | Baseline to week 24 | |
Secondary | Percent Change in NAS (Non-alcoholic Steatohepatitis) | This is measured by change in NAS via non-alcoholic fatty liver disease activity score. NAS is the unweighted sum of steatosis, lobular inflammation and hepatocyte ballooning from baseline to week 24. Total NAS scores can range from 0 to 8. The higher the NAS score, the more severe the liver disease. | Baseline to week 24 | |
Secondary | Change in Cholesterol | This will be measured by change in total, HDL and LDL levels in mg/dL | Baseline, week 6 and week 12, week 24 | |
Secondary | Percent Change in Cholesterol | This will be measured as percent change in total, HDL and LDL levels in mg/dL. | Baseline, week 6 and week 12, week 24 | |
Secondary | Change in Apolipoprotein | This will be measured by change in apolipoprotein A and B in mg/dL | Baseline, week 6 and week 12, week 24 | |
Secondary | Percent Change in Apolipoprotein | This will be measured by percent change in apolipoprotein A and B in mg/dL | Baseline, week 6 and week 12, week 24 | |
Secondary | Change in High-Sensitivity C-Reactive Protein (hsCRP) | This is measured by change in high-sensitivity C-reactive protein (hsCRP) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Percent Change in High-Sensitivity C-Reactive Protein (hsCRP) | This is measured by percent change in high-sensitivity C-reactive protein (hsCRP) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Change in Alanine Aminotransferase (ALT) | This is measured by change in alanine aminotransferase (ALT) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Percent Change in Alanine Aminotransferase (ALT) | This is measured by percent change in alanine aminotransferase (ALT) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Change in Aspartate Aminotransferase (AST) | This is measured by change in aspartate aminotransferase (AST) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 | |
Secondary | Percent Change in Aspartate Aminotransferase (AST) | This is measured by percent change in aspartate aminotransferase (AST) from baseline to weeks 12 and week 24 | Baseline, week 12, week 24 |
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