Orlistat for the Treatment of Type I Hyperlipoproteinemia

NCT02767531 | Completed Phase 2 Results DMC

• 1 other identifier: 012013-042
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Hyperlipoproteinemia Type I; Hypertriglyceridemia

Outcome Measures

Primary Outcomes (1)

• Fasting Serum Triglycerides

  Fasting blood samples were collected on three consequetive days at the end of each three month period. Mean values of the three days were calculated.

  3 days

Study Arms (2)

Orlistat

EXPERIMENTAL

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.

Drug: Orlistat

Off drug

NO INTERVENTION

Standard therapy will be given for three months

Interventions

Orlistat DRUG

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.

Also known as: Alli

Orlistat

Eligibility Criteria

• Age: 8 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (1)

UT Southwestern Medical Center 5323 Harry Hines Blvd

Dallas, Texas, 75390-8537, United States

Location

Related Publications (1)

• Patni N, Quittner C, Garg A. Orlistat Therapy for Children With Type 1 Hyperlipoproteinemia: A Randomized Clinical Trial. J Clin Endocrinol Metab. 2018 Jun 1;103(6):2403-2407. doi: 10.1210/jc.2018-00369.

  PMID: 29659879 DERIVED

MeSH Terms

Conditions

Hyperlipoproteinemia Type I; Hypertriglyceridemia

Interventions

Orlistat

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Lactones; Organic Chemicals

Results Point of Contact

• Title: Abhimanyu Garg, MD
• Organization: UT Southwestern Medical Center

abhimanyu.garg@utsouthwestern.edu

2146482895

Study Officials

• Abhimanyu Garg, MD

  University of Texas

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: May 2, 2016
• First Posted: May 10, 2016
• Study Start: December 1, 2015
• Primary Completion: May 31, 2018
• Study Completion: December 31, 2022
• Last Updated: February 8, 2023
• Results First Posted: February 1, 2019

Record last verified: 2023-02

Locations

US (1)