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Tests treatment safety and results for Type 1 Hyperlipoprotenemia
Official title Long Term Efficacy and Safety of Orlistat for Type 1 Hyperlipoproteinemia
ClinicalTrials.gov ID: NCT05816343
What this study is testing
What is Orlistat?
Orlistat is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for type 1 hyperlipoprotenemia.
Also referred to as Alli.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 56 weeks
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 8 and older
You may be able to join if
- 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...
- people with digenic inheritance with heterozygous disease-causing variants in two different T1HLP genes.
- people 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.
- people 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...
You likely can't join if
- Secondary hypertriglyceridemia due to diabetes, renal disease, alcoholism and drug therapy such as estrogens and estrogen analogues, steroids, HIV-1...
- On lomitapide or participating in clinical trial of volanesorsen.
- 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
See the full eligibility 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).
- people with digenic inheritance with heterozygous disease-causing variants in two different T1HLP genes.
- people 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.
- people 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...
- 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...
- On lomitapide or participating in clinical trial of volanesorsen.
- 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
- Current therapy with anti-coagulants, digoxin and anti-arrhythmics
- Chronic malabsorption syndromes
- Cholestasis
- Acute illnesses such as acute pancreatitis in the last 8 weeks
- Previous history of renal calcium oxalate stones
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 56 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 8 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.