Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia

NCT05816343 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STU-2019-0776
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

Type I hyperlipoproteinemia (T1HLP, also known as familial chylomicronemia syndrome or FCS) is a rare diseasewhere the blood triglycerides (fats) are very high. It is caused by lack of certain enzymes and proteins in the bodythat are important in disposing circulating fats from blood. Treatment of T1HLP patients who have very high levels of blood fats (≥ 1,000 mg/dL) is challenging as conventional triglyceride-lowering medications, such as fibrates and fishoil, are ineffective. The purpose of this trial is to study the long-term efficacy and safety of orlistat for reducing blood triglyceride levels in patients with T1HLP.

Conditions

Type 1 Hyperlipoprotenemia

Outcome Measures

Primary Outcomes (1)

• Change in Triglycerides

  Change in fasting serum TG levels weeks compared to the baseline fasting serum TG levels .

  24 weeks

Study Arms (2)

Orlistat Drug

ACTIVE COMPARATOR

Drug will be given orally.

Drug: Orlistat

Placebo

PLACEBO COMPARATOR

Placebo will be given orally.

Drug: Placebo

Interventions

Orlistat DRUG

Orlistat is an inhibitor of gastric and pancreatic lipases and can reduce dietary fat absorption by 30%. Orlistat at a dose of 2 capsules (each containing 60 mg of active drug) three times a day with each meal (a total dose of 360 mg daily).

Also known as: Alli

Orlistat Drug

Placebo DRUG

Placebo at a dose of 2 capsules (each containing 60 mg placebo) three times a day with each meal (a total dose of 360 mg daily).

Placebo

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

• Secondary hypertriglyceridemia due to diabetes, renal disease, , alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1 protease inhibitors, retinoic acid derivatives, interferons, or lasparaginase.
• On lomitapide or participating in clinical trial of volanesorsen and olezarsen
• On cyclosporine
• Having serum TSH outside of the normal range if on levothyroxine supplementation.
• Use of levothyroxine to suppress TSH in individuals with thyroid cancer.
• 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%)
• Illicit 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

Sponsors & Collaborators

• University of Texas Southwestern Medical Center: lead

Study Sites (1)

UT southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

MeSH Terms

Interventions

Orlistat

Intervention Hierarchy (Ancestors)

Lactones; Organic Chemicals

Study Officials

• Abhimanyu G Garg, MD

  University of Texas Southwestern Medical Center

  PRINCIPAL INVESTIGATOR

• Chandna Vasandani, Ph.D

  University of Texas Southwestern Medical Center

  STUDY DIRECTOR

Central Study Contacts

Abhimanyu G [agarg], MD

CONTACT

2146482895; abhimanyu.garg@utsouthwestern.edu

Abhimanyu G [agarg]

CONTACT

2146482895; abhimanyu.garg@utsouthwestern.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: A randomized, double-blind, placebo-controlled, parallel, study design will be employed for the first 24 weeks (Phase 1) followed by an open-labeled period of 24 weeks (Phase 2).

Study Record Dates

• First Submitted: April 1, 2023
• First Posted: April 18, 2023
• Study Start: January 26, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): May 30, 2029
• Last Updated: May 11, 2025

Record last verified: 2025-05

Locations

US (1)