Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia

• Status: Not yet recruiting
• Trial end date: 2025-07-30
• Target enrollment: 0
• Participant gender: All

Summary

Type I hyperlipoproteinemia (T1HLP, also known as familial chylomicronemia syndrome or FCS) is a rare diseasewhere the blood triglycerides (fats) are very high. It is caused by lack of certain enzymes and proteins in the bodythat are important in disposing circulating fats from blood. Treatment of T1HLP patients who have very high levels of blood fats (≥ 1,000 mg/dL) is challenging as conventional triglyceride-lowering medications, such as fibrates and fishoil, are ineffective.

The purpose of this trial is to study the long-term efficacy and safety of orlistat for reducing blood triglyceride levels in patients with T1HLP.

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

Details

• Lead Sponsor: University of Texas Southwestern Medical Center
• Treatments: Orlistat

Criteria

Inclusion Criteria:

1. Type I hyperlipoproteinemia confirmed by bi-allelic disease-causing variants in any one of the T1HLP genes (LPL, APOC2, APOA5, LMF1, GPIHBP1, or GCKR).

2. Fasting serum triglyceride levels of greater than 1000 mg/dL.

3. Age 8-70 years

4. Effective contraception for males and females of childbearing age.

5. Off orlistat for a period of 2 months.

Exclusion Criteria:

• Secondary hypertriglyceridemias due to diabetes, renal disease, hypothyroidism, alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1 protease inhibitors, retinoic acid derivatives, interferons, or l-asparaginase.

• On lomitapide or participating in clinical trial of volanesorsen

• 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