Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia (NCT05816343) | Clinical Trial Compass
RecruitingPhase 2
Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia
United States28 participantsStarted 2024-01-26
Plain-language 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.
Who can participate
Age range
8 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria:
* Age ≥ 8 years
* Type I hyperlipoproteinemia confirmed by bi-allelic disease-causing variants in any one of the T1HLP genes (LPL, APOC2, APOA5, LMF1, GPIHBP1, or GCKR).
* Subjects with digenic inheritance with heterozygous disease-causing variants in two different T1HLP genes.
* Subjects who have a fasting TG greater than or equal to 750 mg/dL at the end of run-in period of 8 weeks will be eligible for randomization.
* Subjects who do not have confirmed genetic mutation for Type 1 hyperlipoproteinemia but have a fasting TG greater than or equal to 750 mg/dL at the end of run-in period of 8 weeks will be eligible for randomization.
* Well controlled diabetes mellitus with hemoglobin A1c \< 8%
* Off orlistat for a period of 2 months
* Patients on Olezarsen and Plozasiran (APOC3 antisense oligonucleotide) can enroll if on the drug for more than 3 months
* Following methods of contraception for males and females of childbearing age will be employed Males: Being in this research may damage your sperm, which could cause harm to a child that you may father while on this study. If you take part in this study and are sexually active, you must agree to use a medically-acceptable form of birth control. Medically-acceptable forms of birth control include: (1) surgical sterilization (vasectomy), or (2) a condom used with a spermicide (a substance that kills sperm).
Females: If you are part of this study while pregnant or breast-feeding an infant, it is possible that…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Triglycerides
Timeframe: 24 weeks
Trial details
NCT IDNCT05816343
SponsorUniversity of Texas Southwestern Medical Center