Phase 2 N=5 Randomized Treatment

Study of Gemcabene in Adults With FPLD

Familial Partial Lipodystrophy · Hypertriglyceridemia · Fatty Liver · NASH - Nonalcoholic Steatohepatitis

Bottom Line

View on ClinicalTrials.gov: NCT03508687 ↗

Enrolled (actual)

Serious AEs

20.0%

Results posted

Aug 2020

Primary outcome: Primary: Change in Fasting Serum Triglyceride (at 12 Weeks) — -0.44; -20.27 percent change — p=0.517

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: 300mg Gemcabene (Drug); 600mg Gemcabene (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Elif Oral
Primary completion: Jul 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Change in Fasting Serum Triglyceride (at 12 Weeks)	-0.44; -20.27	0.517
SECONDARY Change in Fasting Serum Triglycerides (Through 24 Weeks)	9.25; -126.22; -10.75; -189.33	—
SECONDARY Percent Change in Fasting Serum Triglycerides (Through 24 Weeks)	5.56; -18.91; -0.44; -19.91	—
SECONDARY Change in Liver Fat Content as Measured by Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF)	2.71; 1.01; 12.75; -1.27	—
SECONDARY Percent Change in Liver Fat Content as Measured by Magnetic Resonance Imaging - Protein Density Fat Fraction (MRI-PDFF)	25.66; 11.57; 104.88; -3.36	—
SECONDARY Change in Liver Fibrosis	-0.63; 0.96; -0.03; .45	—
SECONDARY Percent Change in Liver Fibrosis	-19.07; 27.84; -0.37; 41.55	—
SECONDARY Change in NAS (Non-alcoholic Steatohepatitis)	2; NA	—
SECONDARY Percent Change in NAS (Non-alcoholic Steatohepatitis)	100; NA	—
SECONDARY Change in Cholesterol	-7.00; -16.67; 20.50; 23.67	—
SECONDARY Percent Change in Cholesterol	-4.08; -7.61; 12.52; 6.73	—
SECONDARY Change in Apolipoprotein	-4.35; -8.76; 6.74; 7.57	—
SECONDARY Percent Change in Apolipoprotein	-5.38; -6.04; 8.60; 5.02	—
SECONDARY Change in High-Sensitivity C-Reactive Protein (hsCRP)	-0.70; -0.33; 0.15; 0.77	—
SECONDARY Percent Change in High-Sensitivity C-Reactive Protein (hsCRP)	-42.36; 3.71; 15.28; 43.41	—
SECONDARY Change in Alanine Aminotransferase (ALT)	2.50; 13.33; 22.50; 40.67	—
SECONDARY Percent Change in Alanine Aminotransferase (ALT)	6.32; 40.10; 58.78; 82.17	—
SECONDARY Change in Aspartate Aminotransferase (AST)	6.00; 8.00; 11.00; 62.67	—
SECONDARY Percent Change in Aspartate Aminotransferase (AST)	19.25; 25.44; 35.56; 160.41	—

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.

Eligibility 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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03508687). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.