Orlistat for the Treatment of Type I Hyperlipoproteinemia (T1HLP)
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|ClinicalTrials.gov Identifier: NCT02767531|
Recruitment Status : Recruiting
First Posted : May 10, 2016
Last Update Posted : June 19, 2017
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.
|Condition or disease||Intervention/treatment||Phase|
|Hyperlipoproteinemia Type I Hypertriglyceridemia||Drug: Orlistat||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||None (Open Label)|
|Official Title:||Orlistat for the Treatment of Type I Hyperlipoproteinemia|
|Study Start Date :||December 2015|
|Estimated Primary Completion Date :||July 31, 2019|
|Estimated Study Completion Date :||July 31, 2019|
120 mg of Orlistat will be given 3 times to patients weighing greater than 50 kg and patients weighing less than 40 kg will be given 60 mg of Orlistat 3 times a day for 3 months.
Orlistat is a gastric and pancreatic lipase inhibitor that is approved by the FDA for weight loss. It is available over-the-counter as 60 mg tablets under the trade name Alli, and available by prescription as 120 mg capsules under the trade name Xenical.
Other Name: Alli
No Intervention: Off drug
Standard therapy will be given for three months
- Fasting Serum Triglyceride [ Time Frame: 3 months ]Fasting blood samples will be collected on three consequetive days at the end of three months period
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02767531
|Contact: Abhimanyu Garg, MDfirstname.lastname@example.org|
|Contact: Claudia Quittner, RNemail@example.com|
|United States, Texas|
|UT Southwestern Medical Center 5323 Harry Hines Blvd||Recruiting|
|Dallas, Texas, United States, 75390-8537|
|Principal Investigator: Abhimanyu Garg, MD|
|Sub-Investigator: Nivedita Patni, M.D.|
|Principal Investigator:||Abhimanyu Garg, MD||University of Texas|