Clinical Trials for Lipid Metabolism Disorders

Source: ClinicalTrials.gov — U.S. National Library of Medicine — A database of privately and publicly funded clinical studies conducted around the world (U.S. National Library of Medicine, public domain).

This page lists clinical trials related to Lipid Metabolism Disorders from the ClinicalTrials.gov database.

Trial Summary

• Total studies: 50
• Recruiting: 17
• Active, not recruiting: 11
• Completed: 13
• Other: 9

Results are refreshed periodically and may not reflect the most current information available from ClinicalTrials.gov.

Recruiting Trials

NCT00027274 — Cancer in Inherited Bone Marrow Failure Syndromes

Background:

A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.

Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic cancers.

Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with IBMFS who do not develop can…

NCT00029965 — Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Study description:

This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis. Patients may be evaluated every 6 months for infantile onset disease, yearly for juvenile onset and approximately every two years for adult-onset disease as long as they are clinically stable to travel. Data will be evaluated…

NCT00353782 — Causes and Natural History of Dyslipidemias

This study will evaluate people with dyslipidemias - disorders that affect the fat content in the blood. Fats, or lipids, such as cholesterol and triglycerides, are carried in the blood in particles called lipoproteins. These particles are involved in causing blood vessel diseases that can lead to conditions like atherosclerosis (hardening of the arteries) or heart attack. Participants will und…

NCT00344331 — Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick Disease, Type C (NPC), a genetic disorder that results in progressive loss of nervous system function. The study will: 1) look for a clinical or biochemical marker that can be used as a measure of response to treatment, and 2) define the …

NCT05047354 — Biochemical and Phenotypical Aspects of Smith-Lemli-Opitz Syndrome and Related Disorders of Cholesterol Metabolism

Background:

Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder. It can cause birth defects and developmental delays. There is no cure for SLOS or other inherited diseases related to cholesterol production or storage. The data gained in this study may help researchers find ways to measure how well future treatments work.

Objective:

To learn more about SLOS and related disorders and how t…

NCT05588167 — Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Background:

Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But symptoms can vary among different people with the disease. Some may have se…

NCT05705804 — Effects of Pitavastatin or Combination of Pitavastatin and Ezetimibe on Glucose Metabolism Compared to AtoRvastatin in atheroscLerotic Cardiovascular Disease Patients With Metabolic Syndrome: The EZ-PEARL Randomized Trial

The purpose of this study was to investigate the effect of pitavastatin or pitavastatin and ezetimibe combination therapy on glucose metabolism compared to atorvastatin in patients with atherosclerotic cardiovascular disease with metabolic syndrome.

NCT06884358 — Functional Capacity in Anderson-Fabry Disease Patients

The goal of this observational study is to observe the relation between excercise parameters - assessed by CPET - and rest/stress hemodynamic parameters - assessed by echocardiogram and CMR - in patients with a genetic diagnosis of Anderson-Fabry Disease.

Participants will undergo:

• baseline evaluation: clinical evaluation, disease staging with FASTEX and MSSI, KCCQ for quality of life asses…

NCT06684106 — Ursodeoxycholic Acid Attenuates Statin-Induced Impaired Glucose Tolerance

The purpose of this clinical trial is to understand whether the drug Ursodeoxycholic acid (UDCA) can prevent glucose intolerance in participants with hyperlipidemia who are taking statins. It will also assess the safety of UDCA. The primary questions it aims to answer are:

• Will UDCA reduce the incidence of glucose intolerance in participants taking oral statins?
• Will the use of UDCA decrea…

NCT06909565 — Inclisiran Versus Placebo for the Prevention of Major Adverse Cardiovascular and Limb Events in Patients Undergoing Percutaneous Coronary Intervention or Peripheral Endovascular Intervention

V-INTERVENTION will evaluate the effectiveness of inclisiran in preventing major cardiovascular and limb events in patients receiving percutaneous coronary or peripheral arterial revascularization. Inclisiran is a subcutaneous, twice-yearly injection that is FDA-approved as an adjunct with statin therapy and on the market to lower LDL-C in high-risk populations.

NCT07269210 — A Study of Solbinsiran (LY3561774) in Participants With Severe Hypertriglyceridemia

The purpose of this study is to evaluate the safety and efficacy of solbinsiran in lowering triglycerides and other lipid measures compared to placebo in participants with severe hypertriglyceridemia. Participants will receive two subcutaneous injections.

NCT07428473 — A Study of STX-1150 in Participants With Elevated Low-Density Lipoprotein Cholesterol (LDL-C)

STX-1150 is an investigational therapy designed to lower LDL-C by silencing a gene called PCSK9 in the liver. STX-1150 does not edit or permanently change the gene. STX-1150 comprises an mRNA and guide RNA (gRNA) delivered via lipid nanoparticles (LNP) for intravenous infusion. The mRNA produces a protein that switches off the PCSK9 gene expression without altering the DNA sequence. This proces…

NCT07624500 — Food and Metabolism Study

In this study, Investigators are interested in looking at the influence of eating avocados regularly, which are rich in healthy fats, fibers, and unique carbohydrates, on triglyceride and glucose metabolism in people with prediabetes and insulin resistance.

NCT07109375 — Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting

PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.

NCT07477704 — A Study to See How Safe and Effective Alirocumab is When Given Weekly to Adult Participants Who Have Hypercholesterolemia

This study is researching an experimental drug called alirocumab, referred to as “study drug”. In the United States, alirocumab is approved for the treatment of hypercholesterolemia (high blood cholesterol levels).

The aim of the study is to see how safe and effective alirocumab is when given weekly to adult participants who have hypercholesterolemia.

The study is looking at several other res…

NCT07682948 — A Randomized, Open-label, Two-arm, Two-period, Multiple-dose, Study to Evaluate Pharmacokinetic Drug-drug Interaction, Safety and Tolerability of UIC202601 and UIC202602 in Healthy Volunteers

A Randomized, Open-label, Two-arm, Two-period, Multiple-dose, Study to Evaluate Pharmacokinetic Drug-drug Interaction, Safety and Tolerability of UIC202601 and UIC202602 in Healthy Volunteers

NCT07191561 — Hepatic Lipid Metabolism-Alcohol Use Disorder

Patients with hepatic steatosis due to alcohol will be offered liver biopsies when they enter a detoxification program. The first biopsy will occur in the first week of admission and the second in the fourth week when the steatosis has resolved. The hepatic transcriptome will be compared,

Active, Not Recruiting

NCT02262832 — Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy

Background:

- Generalized lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin improves health in people with this disease.

Objective:

- To test the safety and effectiveness of metreleptin.

Eligibility:

• People ages 6 months and older with generalized lipodystrophy w…

NCT03655223 — Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health condi…

NCT03737214 — A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.

NCT04476862 — Cerliponase Alfa Observational Study in the US

This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa. Patients receiving or expected to receive cerliponase alfa within 60 days of signing the informed consent form (ICF) may be eligible to enroll in the study…

NCT04713475 — Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis

NCT04692441 — Apolipoprotein E (APOE) Genotype Effects on Triglycerides and Blood Flow in the Human Brain

High fat feeding (HFF) increases the risk of Alzheimer’s disease (AD) but individuals who carry the AD risk gene E4 paradoxically improve after acute HFF. The investigators propose to further study this phenomenon with a clinical study to assess cerebral blood flow which can be measured by a technique called arterial spin labeling (ASL) on an MRI and is tightly related to brain metabolism.

NCT04020055 — A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)

NCT05739383 — A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.

CKJX839D12302 is a pivotal Phase III study designed to test the hypothesis that treatment with inclisiran sodium 300 milligram (mg) subcutaneous (s.c.) administered on Day 1, Day 90, and every 6 months thereafter in patients at high cardiovascular (CV) risk without a prior major atherosclerotic cardiovascular disease (ASCVD) event will significantly reduce the risk of 4-Point-Major Adverse Card…

NCT06275724 — Specified Drug-use Survey of Leqvio for s.c. Injection.

The objective of this study is to evaluate the long-term safety of Leqvio in patients with familial hypercholesterolaemia or hypercholesterolaemia in post-marketing clinical practice

NCT06094491 — Virtual Diabetes Group Visits Across Health Systems

The purpose of this project is to evaluate the effectiveness of a virtual diabetes group visits on patients with type 2 diabetes mellitus (T2DM).

NCT05774665 — Specialized Pro-resolving Lipid Mediators and Treatment Resistant Depression

The goal of this clinical trial is to determine the impact of omega-3 fatty acids on the production of anti-inflammatory effects and clinical improvement in people with depression who have not responded well to standard antidepressant treatment. The main questions it seeks to answer are:

1. Do omega-3 fatty acids added to ineffective antidepressant treatment increase production of compounds th…

Not Yet Recruiting

NCT07684469 — Comparative Assessment of Inclisiran on Top of Standard of Care Versus Standard of Care Alone for LDL-C Evaluation in Acute Coronary Syndrome Patients in Saudi Arabia

This study aims to evaluate the effectiveness and safety of implementing a systematic augmented low-density lipoprotein cholesterol (LDL-C) management care pathway initiated in hospitals for patients with Acute Coronary Syndrome. This pathway includes treatment with inclisiran on top of the standard of care (SOC), which consists of statins with or without ezetimibe, compared to a high-intensity…

NCT07679828 — Polypills Approach for Multiple Cardiovascular Risk Factors

The Polypill Approach for Multiple Cardiovascular Risk Factors (PACIF) trial is a multicenter randomized controlled trial that will test the effectiveness and safety of a fixed-dose combination strategy for integrated management of hypertension, dyslipidemia, and diabetes among adults aged 50 to 75 years in China. The trial will evaluate whether a simplified regimen combining blood pressure-low…

NCT07313787 — Effects of Meal Macronutrients on Postprandial Lipids

Background:

Abnormal fats in the blood can lead to many problems, including heart disease. Researchers want to learn more about how eating meals with different levels of nutrients affects fats in the blood. Specifically, they want to study people with too much body fat, too little body fat, and a kidney problem called nephrotic syndrome.

Objective:

To learn more about how different types of …

NCT07545954 — DSP-0390 in Combination With Atezolizumab for Small Cell Lung Cancer

This is a single center pilot, phase Ib study with a safety lead-in evaluating the safety and preliminary efficacy of the EBP inhibitor DSP-0390 in combination with atezolizumab in patients with extensive stage small cell lung cancer (ES-SCLC) whose disease has not progressed after initial induction therapy with platinum-based chemotherapy and anti-PD-L1 immunotherapy (atezolizumab or durvaluma…

NCT07685639 — Electronic Health Record Augmented LDL-Cholesterol Optimization and Control Via Active Lipid Engagement (EHR-LOCALE): A Quality Improvement Implementation Study

The purpose of this study is to determine whether a proactive, EHR-guided approach helps more individuals reach safe cholesterol levels, improves medication use, and reduces future heart attacks and strokes, compared to usual care.

The main question it aims to answer is:

• Does the EHR-LOCALE pathway increases the proportion of patients who reach the LDL-cholesterol target of <1.8 mmol/L at …

NCT07680829 — A Phase 3 Trial of DR10624 Versus Placebo in Patients With Severe Hypertriglyceridemia

This is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial for adults with severe hypertriglyceridemia whose lipid levels remain uncontrolled under standard Icosapent Ethyl background treatment plus lifestyle management. Eligible participants complete a screening run-in period, then randomize equally to three treatment groups: two active DR10624 subcutaneous injection gro…

Enrolling by Invitation

NCT00001215 — Genetic Studies of Lysosomal Storage Disorders

The purpose of this study is to identify genetic, biochemical, and clinical factors that are associated with disease severity in people with Gaucher disease and other lysosomal storage disorders.

There is a vast spectrum of clinical manifestations in people with Gaucher disease as well as other lysosomal storage disorders. This study will evaluate patients with lysosomal disorders on an outpat…

Completed Trials

NCT00001204 — Cardiovascular Evaluation of Patients With High Cholesterol and Normal Volunteers

Homozygous familial hypercholesterolemia is a rare inherited disease of metabolism. It occurs in less than 1 in 1 million people within the United States. Patients with the disease are typically children and young adults who develop heart disease early in life. Children less than age 5 years with this disease have suffered heart attacks and death.

The normal process that removes cholesterol pa…

NCT00001721 — Study of Smith-Lemli-Opitz Syndrome

Smith-Lemli-Opitz Syndrome (SLOS) is a genetic disorder (autosomal recessive) caused by an abnormality in the production of cholesterol. The disorder can occur in both a “mild” or “severe” form. SLOS is associated with multiple birth defects and mental retardation. Some of the birth defects include; abnormal facial features, poor muscle tone, poor growth, shortened life span, and abnormalities …

NCT04737720 — Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

Background:

• High density lipoprotein (HDL), or good cholesterol, moves cholesterol from the artery walls back to the liver. A blood enzyme known as LCAT maintains HDL levels and helps it remove cholesterol from the body. Familial LCAT deficiency (FLD) is a genetic disease that results in low levels or total absence of LCAT. People with FLD often have kidney problems, and may develop kidney f…

NCT02311335 — Genes Involved in Lipid Disorders

Background:

- Genes are the instructions our body uses to function. Researchers can look for changes, or variants, in the genes. The goal of this study is to find new gene changes that lead to lipid disorders. Older research methods looked at one or a few genes at a time. Genomic sequencing looks at most of the genes at once. Genomic sequencing may find the cause researchers haven t been able…

NCT03140605 — Greek Registry - Familial Hypercholesterolaemia

Familial hypercholesterolemia (FH) [heterozygous (heFH) or homozygous FH (hoFH)] is a common genetic disorder, characterized by elevated plasma low density lipoprotein (LDL) cholesterol concentration leading (if untreated) to cholesterol deposits in the corneas, eyelids and extensor tendons, rapidly progressing vascular disease, and aortic valve disease.

NCT05075902 — Effect of Aerobic Training on the Health Parameters of Postmenopausal Women With Multimorbidity

People affected by multiple chronic diseases have a greater chance of hospitalization, longer hospital stays, worse general health, worse physical and mental function and lower functional capacity, with an average risk of 50% of functional decline with each additional condition. The frequency of multimorbidity is higher in older, inactive women, who live in urban areas in low- and middle-income…

NCT05852431 — To Evaluate the Efficacy and Safety of Pegozafermin in Subjects With Severe Hypertriglyceridemia

To determine the effect of Pegozafermin on fasting serum triglyceride levels in subjects with Severe Hypertriglyceridemia (TG ≥500 to ≤2000 mg/dL) after 26 weeks of treatment.

NCT07674875 — Meal Frequency and Timing on Metabolic Health in Patients With Type 2 Diabetes

The goal of this clinical trial is to learn if changing when and how often people eat works to improve health in adults with Type 2 diabetes. The main questions it aims to answer are:

How do these eating patterns change body weight and body fat?

Does changing meal timing and frequency lower blood sugar levels (HbA1c) over 12 weeks?

Does it lower blood fat levels, such as cholesterol and trig…

NCT07682285 — Taiwanese Green Propolis for Lipid Profiles, Fatigue, and Quality of Life in Oral Cavity Cancer Survivors

This study examined whether Taiwanese Green Propolis (TGP), a natural bee-derived supplement, can improve blood fat levels, reduce tiredness (fatigue), and improve quality of life in people who have completed treatment for oral cavity (mouth) cancer.

People who had finished treatment for oral cavity squamous cell carcinoma (a type of mouth cancer) and were in the follow-up period were invited …

NCT06640972 — Effects of RDX-002 on Postprandial Triglycerides in Patients Discontinuing GLP-1 Agonists

The goal of this clinical trial is to learn if drug RDX-002 works to treat high levels of fat (known as triglycerides, or TGs) in the blood in adults. It will also learn about the safety of drug RDX-002. The main question it aims to answer is if treatment with RDX-002 will lower triglycerides after a high-fat meal in patients who have recently stopped treatment with semaglutide or tirzepatide f…

NCT05988866 — Effects of a Digital Health Application (Lipodia) on LDL-cholesterol Levels

The goal of this clinical trial is to find out if lipodia, a digital health intervention, can help lower cholesterol levels and improve other health measures in adults with high cholesterol.

The main questions it aims to answer are:

• Does lipodia, together with regular treatment, lower LDL cholesterol (bad cholesterol) better than regular treatment alone?
• Does lipodia help improve other he…

NCT07691268 — Study to Evaluate the Safety and the Pharmacokinetics of “UI111” and “UIC202006” Under Fed Conditions

Phase 1 study to evaluate the safety and the pharmacokinetics of “UI111” and “UIC202006” in healthy adult volunteers under fed state conditions

- Open-label, randomized, single-dose, 2-sequence, 4-period, crossover design

NCT07423598 — A Study to Investigate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics With AZD0780 Following Repeated Dose Administration in Healthy Adults With Elevated LDL-C Levels

The purpose of this study is to evaluate pharmacodynamics (PD) of AZD0780 compared with placebo by assessment of low-density lipoprotein-cholesterol (LDL-C) levels following repeated oral dosing in healthy adults with elevated LDL-C levels.

Other (Suspended)

NCT02506946 — NAFLD in Adolescents and Young Adults With PCOS

This project focuses on an at-risk adolescent and young adult population who may gain long-term health benefits from detection of risk factors at a young age. The primary aims of this proposal are: 1) To observe whether adolescents and young adults with Polycystic Ovary Syndrome (PCOS) are more likely to have elevated liver fat (>/=4.8%) than controls by studying liver fat deposition measured …

Other (Terminated)

NCT07455864 — Lysosomal Acid Lipase Deficiency in Risk Groups

A multicenter real-world observational study of the prevalence, diagnostic pathways, and clinical characteristics of lysosomal acid lipase deficiency in pediatric and adolescent risk groups in the Russian Federation (HELIOS)