Clinical Research Directory

Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

Background: \- Partial 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 age 6 months and older with partial lipodystrophy who * have received metreleptin through NIH studies and shown improvement AND * cannot get metreleptin other ways. Design: * Participants will come to NIH approximately every 6 months during year one, then every 1 2 years. Financial assistance may be available for travel within the U.S. * At visits, participants will get a supply of metreleptin to take home for daily injections, or it can be shipped to them inside the U.S. They will have: * plastic catheter placed in an arm vein. * blood tests, urine collection, and physical exam. * oral glucose tolerance test, drinking a sweet liquid. * ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the skin and pictures are taken of the organs. * echocardiogram, which takes pictures of the heart with sound waves. * Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they breathe is measured. * Participants will have up to 3 DEXA scan x-rays per year. * Participants may have: * annual bone x-rays. * liver biopsies every few years. A needle will be inserted into the liver to obtain a small piece. Participants will sign a separate consent for this. * Participants must be seen regularly by their local doctors and have blood tests at least every 3-6 months at home.

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 who: * have received metreleptin through NIH studies AND * cannot get it through approved or compassionate use mechanisms in their home country. Design: * Participants will come to NIH approximately every 6 months during year one, then every 1 2 years. Financial assistance may be available for travel within the U.S. * At visits, participants will get a supply of metreleptin to take home for daily injections. They will have: * plastic catheter placed in an arm vein. * blood tests, urine collection, and physical exam. * oral glucose tolerance test, drinking a sweet liquid. * ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the skin and pictures are taken of the organs. * echocardiogram, which takes pictures of the heart with sound waves. * Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they breathe is measured. * Participants will have up to 3 DEXA scan x-rays per year. * Participants may have: * annual bone x-rays. * liver biopsies every few years. A needle will be inserted into the liver to obtain a small piece. Participants will sign a separate consent for this. * Participants must be seen regularly by their local doctors and have blood tests at least every 3 6 months at home.

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 6 months? Participants in the interventional group receive: * Pharmacist contact * Repeat lipid requisitions * Review of medications, adherence, and side effects * Treatment escalation according to national dyslipidemia guidelines

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 decrease other adverse events in patients taking oral statins? Participants will: * Take Atorvastatin combined with UDCA or a placebo daily for 6 months * Have follow-up visits on day 40, day 110, and day 180 Have their examination indicators recorded.

Management Strategy of 1L Lorlatinib With Hyperlipidemia in Stage IIIB-IV ALK Positive NSCLC

For Patients and Families Brief Title: Management of 1L Lorlatinib with Hyperlipidemia in ALK+ Advanced NSCLC What is this study about? This study is for people with ALK-positive non-small cell lung cancer (NSCLC) who are taking lorlatinib (Lorbrena®) as their first treatment and have developed high cholesterol (hyperlipidemia) as a side effect. Why is this study needed? Lorlatinib is a highly effective targeted therapy, but it frequently causes elevated cholesterol and triglycerides. There is currently no standard guideline on how to best manage this side effect. This study aims to find the best approach to control lipid levels while on lorlatinib treatment. What will happen in this study? The study has two parts: * Part A (Observational) : About 100 participants. Doctors manage hyperlipidemia according to routine clinical practice. Researchers simply observe and record which lipid-lowering treatments are used and how well they work. * Part B (Randomized Controlled Trial) : 60 participants with high-risk factors are randomly assigned to either: * Intensive treatment: rosuvastatin + ezetimibe + evolocumab * Standard treatment: rosuvastatin + ezetimibe What tests are involved? * Blood tests for lipid levels at baseline, Weeks 4, 8, 20, and 24 * Routine CT or MRI scans for tumor assessment * Some participants in Part B may have a non-invasive vascular ultrasound (FMD) test * Total participation per patient: up to 7 months Is this study safe? * ✅ Approved by the Ethics Committee of Sun Yat-sen University Cancer Center * ✅ All drugs used (lorlatinib, statins, ezetimibe, evolocumab) are already approved and widely used * ✅ An independent Data Monitoring Committee (DMC) monitors safety throughout the study * ✅ Participants may withdraw at any time without affecting their regular care For Healthcare Providers Study Title: Management strategy of 1L Lorlatinib with Hyperlipidemia in Stage IIIB-IV ALK positive NSCLC: A multi-center prospective study in China Sponsor / Investigators: Sun Yat-sen University Cancer Center (PI: Prof. Zhang Li) Study Type: * Part A: Observational, prospective, real-world cohort study * Part B: Prospective, randomized controlled trial (RCT) Estimated Enrollment: 160 participants (Part A: \~100, Part B: 60) Study Duration: Approximately 4 years (anticipated completion: December 2029) Key Inclusion Criteria: * Stage IIIB-IV ALK+ NSCLC (confirmed by IHC, FISH, PCR, NGS, or ctDNA) * No prior systemic therapy for advanced/metastatic disease * ECOG PS 0-2 * Age ≥ 18 years * Hyperlipidemia (ULN ≤ TC \< 12.93 mmol/L, Grade 1-3) while on first-line lorlatinib * At least one measurable lesion per RECIST v1.1 * Life expectancy ≥ 6 months Primary Endpoints: * Part A: Describe real-world treatment patterns for hyperlipidemia management * Part B: Percentage change in LDL-C from baseline to Week 12 Oversight: * Independent Data Monitoring Committee (DMC) * Trial Management Committee * Ethics Committee of Sun Yat-sen University Cancer Center (Approval No. B2026-159-01) Participating Centers: 8 sites across China

A Clinical Study of Enlicitide in Participants With High Cholesterol (MK-0616-037)

Researchers designed a study medicine called enlicitide to lower low-density lipoprotein cholesterol (LDL-C). In this study, researchers want to learn about giving enlicitide with another medicine called rosuvastatin. Rosuvastatin is a standard (usual) treatment to lower LDL-C. The goal of this study is to learn if enlicitide given with rosuvastatin works better than placebo on lowering LDL-C in a person's blood. A placebo looks like the study medicine but has no study medicine in it. Using a placebo helps researchers better understand the effects of a study medicine.

Assessment of the Effects and Safety of Spermidine Supplementation on Blood Lipids and Body Weight in Overweight or Obese Individuals With Hyperlipidemia

Evaluate the effect of spermidine supplementation on lipid profile changes in individuals with overweight or obesity accompanied by hyperlipidemia. Analyze the impact of spermidine on metabolic parameters and assess the safety of spermidine supplementation, including liver and kidney function, gastrointestinal reactions, and other adverse effects.

A Clinical Study Evaluating the Efficacy and Safety of HRS-7249 in Treating Patients With Hyperlipidemia

The study is being conducted to evaluate the efficacy and safety of HRS-7249 for patients with hyperlipidemia, and to explore the reasonable dosage of HRS-7249 for patients with hyperlipidemia.

Lipoprotein Metabolism in Normal Volunteers and Patients With High Levels of Lipoproteins

Researchers plan to study the fat-rich particles, called lipoproteins, which circulate in the blood. This study is designed to improve understanding of normal, as well as abnormal, lipoprotein metabolism and the role it plays in the development of hardening of the arteries (atherosclerosis). Patients participating in this study will receive injections of lipoproteins or apolipoproteins (the protein component of lipoproteins) that have been isolated and purified. These lipoproteins will be labeled with small amounts of radioactive material and sterilized before they are injected into the patient. Patients participating in the study will be required to have blood samples taken, and provide urine samples throughout the course of the study. In addition, patient will be required to follow a specially formulated diet. Patients will be weighed throughout the course of the study.

Regional Innovation in Chronic Disease and Elderly Care

This project aims to develop a community-based health promotion intervention model for populations at potential risk of metabolic syndrome. Through exercise training and lifestyle modifications, the project seeks to improve cardiopulmonary function and disease control, align with current public health policies, and establish an evidence-based model with strong potential for broader implementation.

A Trial to Evaluate the Efficacy and Safety of SHR-1918 in Patients With Hyperlipidemia

The study is being conducted to evaluate the efficacy, and safety of SHR-1918 in reducing serum low-density lipoprotein cholesterol (LDL-C) and total triglycerides (TG) in patients with hyperlipidemia compared with placebo, and also includes To evaluate the effectiveness of SHR-1918 in reducing other lipid indicators in patients with mixed hyperlipidemia and to assess the safety of SHR-1918 in patients with mixed hyperlipidemia.

Changes in Plaque Characteristics After Short-term Statin Therapy as Assessed With Coronary CT

INTENSE Trial is a prospective, double-blind, randomized, placebo-controlled, single-center study with two arms (40 mg intensified statin therapy vs matching placebo for rosuvastatin) among statin-naive patients referred to coronary CT angiography due to stable chest pain, followed for 24 months by using a photon-counting detector CT (PCD-CT). INTENSE Trial aims 1) to assess the effect of short-term intensified statin therapy on coronary anatomy and physiology using PCD-CT and 2) to determine the impact of short-term, intensified statin therapy on coronary plaque morphology and hemodynamics to identify statin responder and non-responder patients in addition to testing the hypothesis of "plaque memory" after the 24-month follow-up period.

Effect of Smartphone Application (MED-AD) on Medication Adherence Among Patients With Cardiovascular Disease in Oman

This study aims to: 1. Investigate the impact of MED-AD intervention on medication adherence among CVD patients, compared to patients receiving text message reminders or routine care alone at baseline, 6 months, and 12 months later. 2. Evaluate the effectiveness of MED-AD intervention on clinical outcomes among patients with CVD, compared to the outcomes of patients receiving text or routine care alone at baseline, 6 months, and 12 months later. This study is a prospective randomized controlled clinical trial (RCT) with three arms: 1. MED-AD intervention: MED-AD is a smartphone application that will use the following strategies (pill reminders, Medication Adherence Report, medication intake confirmation, Medical Appointment Reminder, and educational notifications) to enhance medication adherence 2. Text message reminders: Participants will receive short text message (SMS) reminders to remind them to take medications as prescribed once daily 3. Routine care only: Participants will receive routine care that does not include text messages or reminders and education through an application.

Development and Clinical Validation of Key Technologies for Non-invasive Breath Metabolic Fingerprinting for Large-scale Population Lipid Screening

This study collects exhaled breath condensate samples from healthy populations and hyperlipidemic populations, analyzes the characteristics of the exhaled metabolic lipid profile for early identification of people with abnormal blood lipids, verifies the feasibility and accuracy of a new gas detection method for screening abnormal blood lipids, and explores a simple, fast, and non-invasive method for screening abnormal blood lipids.

Chronic Kidney Disease and Metabolic Disorders in the Elderly

The goal of this observational study is to investigate the impact of metabolic comorbidities on outcomes in elderly Chinese adults, through two parallel sub-cohorts: one comprising elderly individuals without chronic kidney disease (CKD) at baseline, and one comprising elderly patients with established CKD. The main questions it aims to answer are: Do metabolic diseases (diabetes, hypertension, hyperlipidemia, hyperuricemia, calcium-phosphorus disorders) increase the risk of incident CKD, major adverse cardiovascular events (MACE), and all-cause mortality in elderly individuals without CKD at baseline? What are the optimal diagnostic thresholds and criteria for CKD in the elderly population? How do metabolic comorbidities affect long-term outcomes - including all-cause mortality, end-stage renal disease (ESRD), and cardiovascular events - in elderly patients with established CKD? What is the comorbidity profile and disease burden of elderly CKD patients in China? Participants in both sub-cohorts will: Undergo baseline assessments including laboratory tests and clinical evaluations. Provide longitudinal follow-up data through scheduled clinic visits and medical record linkage. Contribute to a large-scale cohort of 100,000 elderly participants across multiple centers in China.

Evaluating the Effectiveness of a Multi-level Health-Related Social Needs Initiative

The goal of this study is to evaluate the effectiveness of a multi-level health-related social needs (HRSN) initiative among adult patients diagnosed with type 2 diabetes, plus hypertension or hyperlipidemia. The multi-level initiative includes HRSN data collection, training and tools for health care professionals, care coordinator support, and community resources. Patients with type 2 diabetes plus hypertension or hyperlipidemia will be screened for HRSN as part of the primary care clinic intake process to assess if patients have any social needs (like difficulty with getting food, housing, or transportation). If patients screen positive for having social needs, then patients will be offered support, which can include help from primary care providers with adjusting a patient's disease management plan, referrals to care coordinators to provide additional assistance in addressing social needs, and information about community organizations that offer resources for social needs. The initiative will be integrated as a system change across VUMC adult primary care practices. The investigators will use a prospective, single-arm clinical trial to evaluate effects on clinical outcomes for 12 months. Data will be extracted from the EHR on adult patients diagnosed with type 2 diabetes, and either hypertension or hyperlipidemia. The investigators hypothesize that the initiative will reduce the impact of HRSN on clinical outcomes over the study period. The investigators will also administer surveys to a subgroup of patients to examine trends in self-reported psychosocial and behavioral measures over the course of the initiative.

Duke Cardiometabolic Prevention Clinic's Impact on High-risk Cardiovascular Patients With Uncontrolled Risk Factors

This project is studying whether a team-based specialty clinic can help people with type 2 diabetes and heart disease better manage their blood pressure and cholesterol. The clinic includes coordinated care from heart doctors, kidney doctors, diabetes specialists, and liver doctors. The study will compare two groups of patients: one receiving usual care from their primary care provider, and one referred to the Duke Cardiometabolic Prevention Clinic for multidisciplinary care. The main goals are to find out if this clinic improves blood pressure and cholesterol control over 12 months, increases use of recommended heart medications, and reduces hospital visits and other healthcare use. Participants will be randomly assigned to one of the two groups. Those referred to the clinic will: 1) Meet with a cardiologist for an initial evaluation. 2) Be referred to other specialists (such as endocrinology, nephrology, or hepatology) based on their needs. 3) Receive ongoing, coordinated care from a team of specialists working together to improve their heart and metabolic health.

Prospective Exploratory Study on the Comprehensive Application Effectiveness of Exercise Prescription Decision Support Tools in the Management of Patients With "Four Highs" (Hypertension, Hyperglycemia, Hyperlipidemia, Hyperuricemia)

This study conducted a six-month exploratory clinical trial to evaluate the impact of an exercise prescription mini-program, based on the "Exercise Guidelines for the 'Four Highs'", on the physical activity levels and related health indicators of patients with hypertension, hyperglycemia, hyperlipidemia, and hyperuricemia in primary healthcare settings in China.

A Study to Evaluate COR-1004 in Adult Volunteers.

This research study is being done to see if a new, injectable drug called COR-1004 is safe for people to take. It will also help doctors find the right dose of the drug. The study will look at how different dose levels of COR-1004 affect the levels of LDL cholesterol (often called "bad" cholesterol) in the blood. The study will enroll otherwise healthy volunteers who have LDL cholesterol of 100 mg/dl or higher but are not currently taking any cholesterol-lowering medicines. The main purpose of this study is to test a new drug, COR-1004. Researchers want to find out: * If the drug is safe and if people have any side effects. * The highest dose that can be given without causing serious side effects. * How the body processes the drug. * How the drug affects the amount of "bad" cholesterol in the blood. This is an early-phase study. The information learned will help decide if COR-1004 should be tested in larger studies in the future. To be in this study, you must: * Be generally healthy. * Have an LDL ("bad") cholesterol level of 100 mg/dL or higher. * Not currently be taking any medications to lower your cholesterol. If you decide to join the study, you will be placed into a group by chance, like flipping a coin. This is called "randomization." In each group, most people (8 out of 10) will get a single injection of the study drug (COR-1004). The other people (2 out of 10) will get a placebo. A placebo looks just like the study drug but has no active medicine in it. Using a placebo helps researchers understand the true effects of the real drug. This is a "double-blind" study, which means that neither you nor the study doctor will know if you are getting the study drug or the placebo. The study will test different dose levels of COR-1004. It will start with a low dose and, only if it is proven to be safe, will a new group of participants be given a slightly higher dose. To be extra safe, the first two people in each new dose group will be watched for 24 hours before the other eight people in that group get their injection. After the injection, you will need to visit the study clinic several times over the next year for follow-up. These visits will include: * Answering questions about your health. * Physical exams. * Blood tests to check your safety and your cholesterol levels. A special group of experts, called a Safety Review Committee, will carefully watch over the study to protect all participants. Your active participation in the study, including follow-up visits, will last for about 12 months after you receive the single injection. Because COR-1004 is a new drug, there may be risks that are not yet known. The study staff will explain all the known risks to you before you agree to participate. Known risks may include discomfort at the injection site, like redness, swelling, or pain. The study team will monitor you very closely for any side effects throughout the study.

Implementation of an Algorithm- and Multi-professional Team-supported Strategy to Improve Lipid Management of Patients With Atherosclerotic Cardiovascular Disease

This is a randomized, controlled, collaborative, multicenter, pilot implementation science study evaluating the hypothesis that algorithm- and multi-professional team-supported lipid management reduces low density lipoprotein-cholesterol (LDL-C) compared with usual care in patients with atherosclerotic cardiovascular disease (ASCVD).

Rapid Escalation of LipiD Lowering Therapy to Reduce RecUrrent Cardiovascular Event

Hyperlipidaemia especially elevation of low-density lipoprotein cholesterol (LDL-C) is known to be the main contributor to the development of atherosclerotic cardiovascular disease (ASCVD). Lowering LDL-C have been shown to reduce the risk of ASCVD2,3. Both the American Heart Association (AHA) and the European Society of Cardiology (ESC) advocated aggressive LDL-C target with statin being the first-line lipid-lowering therapy (LLT). However, a significant portion of patients did not attain their LDL-C goal in our locality. Statin no adherence, low uptake of adjuvant non-statin LLT, and therapeutic inertia are few potential causes for not achieving the LDL-C target. ESC recommends repeating blood test in 6-8 weeks after addition or alternation of LLT, but this recommendation was not routinely followed in current local practice due to resources constrain. This registry therefore aims to investigate the strategy of frequent monitoring of LDL-C and titration of LLT in achieving LDL-C treatment target at a dedicated specialist clinic.

A Study to Evaluate the Pharmacokinetic Interaction and the Safety of AD-231A and AD-231B and AD-231C

To evaluate the pharmacokinetic interactions of AD-231A, AD-231B, and AD-231C in healthy adult volunteers.

Inspire HER: Inspiring the Heart and Emotions for Radical Health

Poor heart health puts Black women at risk for a shorter life with more illness than people of any other non-indigenous racial group. We will refine and conduct a randomized, wait-list controlled trial of Inspire HER (a heart health lifestyle intervention for Black women that provides health education, physical activity, and social resources). We will also compare Inspire HER and Black Impact (a heart health intervention for Black men) data to study how women and men respond differently to stress. This trial aligns with American Heart Association's pledge to address heart health equity through new interventions that consider the person and the circumstances with which they live.

"Smart Family Doctor" Assisted Comprehensive Management of Secondary Prevention Among Post Revascularization Patients

This study aims to evaluate the effect of an AI-assisted "Smart family doctor" digital health management tool on improving the control rates of hypertension, diabetes, and dyslipidemia in post-revascularization patients. A randomized controlled trial design will be used, involving approximately 10-20 hospitals and 951 participants. Eligible participants are adults aged between 18 and 80 years, post-CABG or post-PCI patients with hypertension, diabetes, and dyslipidemia.