Orlistat for the Treatment of Type I Hyperlipoproteinemia (T1HLP)

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The University of Texas System (UT)

Status and phase

Completed
Phase 2

Conditions

Hyperlipoproteinemia Type I
Hypertriglyceridemia

Treatments

Drug: Orlistat

Study type

Interventional

Funder types

Other

Identifiers

NCT02767531
012013-042

Details and patient eligibility

About

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.

Full description

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.

Enrollment

2 patients

Sex

All

Ages

8 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Type I hyperlipoproteinemia
  • Fasting serum triglyceride levels of greater than 1000 mg/dL
  • Age > 8 years

Exclusion criteria

  • 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
  • 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%)
  • 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
  • Current therapy with cyclosporine
  • Chronic malabsorption syndromes
  • Cholestasis
  • Acute illnesses such as acute pancreatitis in the last 8 weeks

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Crossover Assignment

Masking

None (Open label)

2 participants in 2 patient groups

Orlistat
Experimental group
Description:
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.
Treatment:
Drug: Orlistat
Off drug
No Intervention group
Description:
Standard therapy will be given for three months

Trial documents
1

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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