Orlistat for the Treatment of Type I Hyperlipoproteinemia

012013-042

Patients with Type I Hyperlipoproteinemia (T1HLP) have a rare form of hypertriglyceridemia marked by significant chylomicronemia and recurrent episodes of acute pancreatitis. T1HLP is caused by a deficiency of lipoprotein lipase or one of its cofactors. Many patients are a challenge to treat, as the only effective therapy available is an extremely low fat diet. This diet is exceedingly difficult to follow, and despite adherence, many patients still have chylomicronemia and develop acute pancreatitis. Specific Aim: To determine the efficacy of a gastric and pancreatic lipase inhibitor, Orlistat, in reducing serum triglyceride levels in patients with T1HLP.

2015-12-01

2018-05-31

2022-12-31

Completed

Early phase I

2

Inclusion Criteria: 1. Type I hyperlipoproteinemia 2. Fasting serum triglyceride levels of greater than 1000 mg/dL 3. Age \> 8 years Exclusion Criteria: 1. Secondary hypertriglyceridemias due to diabetes, renal disease, hypothyroidism, alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIVprotease inhibitors, retinoic acid derivatives and interferons 2. Pregnant or lactating women 3. 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) 4. Severe anemia (hematocrit \< 24%) 5. Drug use (cocaine, marijuana, LSD, etc.) 6. Major surgery in the past three months 7. Congestive heart failure 8. Serum creatinine greater than 2.5 mg/dL 9. Cancer within the past five years 10. Gastrointestinal surgery in the past 11. Current therapy with anti-coagulants, digoxin, and anti-arrhythmics 12. Current therapy with cyclosporine 13. Chronic malabsorption syndromes 14. Cholestasis 15. Acute illnesses such as acute pancreatitis in the last 8 weeks

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'CROSSOVER', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 2, 'type': 'ACTUAL'}}

2023-02-08