Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia

Launched by UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER · Apr 14, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the long-term effects of a medication called orlistat in people with Type 1 Hyperlipoproteinemia (T1HLP), a rare condition that causes very high levels of fats (triglycerides) in the blood. The trial aims to see if orlistat can help lower these fat levels safely and effectively, especially since standard medications haven’t been successful for many patients.

To participate, individuals must be aged between 8 and 70 years and have a confirmed diagnosis of T1HLP, along with fasting triglyceride levels above 750 mg/dL. They should not have taken orlistat for at least two months prior to joining. Participants can expect regular check-ups and monitoring during the study, contributing valuable information to help improve treatment options for this challenging condition. It’s important for potential participants to discuss their health history with the research team to ensure they meet the eligibility requirements.

Gender

ALL

Eligibility criteria

• • Type I hyperlipoproteinemia confirmed by bi-allelic disease-causing variants in any one of the T1HLP genes (LPL, APOC2, APOA5, LMF1, GPIHBP1, or GCKR).
• • Subjects who have a fasting TG greater than or equal to 750 mg/dL at the end of run-in period of 8 weeks will be eligible for randomization.
• • Age ≥ 8 years
• • Well controlled diabetes mellitus with hemoglobin A1c \< 8%
• • Off orlistat for a period of 2 months
• Exclusion Criteria:
• • Secondary hypertriglyceridemia due to diabetes, renal disease, , alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1 protease inhibitors, retinoic acid derivatives, interferons, or lasparaginase.
• • On lomitapide or participating in clinical trial of volanesorsen and olezarsen
• • On cyclosporine
• • Having serum TSH outside of the normal range if on levothyroxine supplementation.
• • Use of levothyroxine to suppress TSH in individuals with thyroid cancer.
• • Pregnant or lactating women
• • Significant liver disease (elevated transaminases \> 2 times upper limit of normal)
• • Alcohol abuse (\> 7 drinks or 84 g per week for women and \> 14 drinks or 168 g per week for men)
• • Severe anemia (hematocrit \< 24%)
• • Illicit drug use (cocaine, marijuana, LSD, etc.)
• • Major surgery in the past three months
• • Congestive heart failure
• • Serum creatinine greater than 2.5 mg/dL
• • Cancer within the past five years
• • Gastrointestinal surgery in the past
• • Current therapy with anti-coagulants, digoxin and anti-arrhythmics
• • Chronic malabsorption syndromes
• • Cholestasis
• • Acute illnesses such as acute pancreatitis in the last 8 weeks
• • Previous history of renal calcium oxalate stones