Study of Gemcabene in Adults With FPLD

• Status: Completed
• Trial end date: 2019-07-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Elif Oral

Criteria

• 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-γ, 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.