Clinical Trial: Orlistat for the Treatment of Type I Hyperlipoproteinemia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Orlistat for the Treatment of Type I Hyperlipoproteinemia

Brief Summary:

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.


Detailed Summary:

Type I hyperlipoproteinemia is a rare, autosomal recessive metabolic disorder characterized by extreme hypertriglyceridemia due to a deficiency in lipoprotein lipase or related proteins. Treatment of these patients is challenging as triglyceride-lowering medications are ineffective. A low fat diet is helpful, however, despite good dietary compliance, some patients continue to have severe hypertriglyceridemia and recurrent pancreatitis which can be life threatening. Therefore, Investigator wish to investigate whether inducing dietary fat malabsorption or inhibiting chylomicron formation will cause further lowering of serum triglycerides (TG) beyond the effect of limiting dietary fat intake.

Investigator will study the efficacy and safety of an inhibitor of intestinal lipase (Orlistat) for reducing serum triglyceride levels in patients with Type I hyperlipoproteinemia. Investigator plan to enroll 20 patients with Type I hyperlipoproteinemia in a randomized, double-blind, placebo-controlled, cross-over trial. During the last week of each study period, fasting blood samples will be drawn for three consecutive days for serum lipids and chemistry panel. The primary endpoint will be serum triglycerides; the secondary endpoint variables will be fasting and postprandial serum chylomicron-TG levels, postprandial serum TG levels during a meal tolerance test and retinyl palmitate levels during a meal tolerance test. Repeated measures analysis of variance will be used for statistical comparisons.

These results may help in designing novel therapeutic approaches for patients with Type 1 hyperlipoproteinemia.


Sponsor: University of Texas Southwestern Medical Center

Current Primary Outcome: Fasting Serum Triglyceride [ Time Frame: 3 months ]

Fasting blood samples will be collected on three consequetive days at the end of three months period


Original Primary Outcome: Fasting blood samples will be collected for measuring serum triglyceride every month. At third months, three consecutive fasting blood samples will be collected daily for triglyceride measurement. [ Time Frame: 3 months ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University of Texas Southwestern Medical Center

Dates:
Date Received: May 2, 2016
Date Started: December 2015
Date Completion: December 2017
Last Updated: May 9, 2016
Last Verified: May 2016