Clinical Research Directory

Effect of a Brief Educational Intervention on Endotracheal Tube Cuff Pressure

Endotracheal tube cuff pressure outside the recommended range of 20-30 cmH₂O is common in routine anesthesia practice. Over-inflation can injure the tracheal mucosa, while under-inflation increases the risk of aspiration and ventilator-associated pneumonia. This prospective, single-center, uncontrolled before-and-after study evaluates whether a brief educational intervention improves cuff pressure management in the operating room. Adult patients undergoing general anesthesia with endotracheal intubation at Marmara University Pendik Training and Research Hospital are enrolled in two consecutive periods. In both periods, cuff pressure is measured with a calibrated manometer shortly after intubation, together with the professional role and clinical experience of the provider who inflated the cuff. Between the two periods, a two-minute educational video explaining the importance of optimal cuff pressure and demonstrating manometer use is distributed to all operating room staff involved in cuff inflation, and reminder posters are placed in every operating room. The primary outcome is the proportion of cuff pressure measurements within the recommended range before versus after the intervention. The secondary outcome is the association between the cuff-inflating provider's role and experience and cuff pressure appropriateness. No change to patient care beyond the educational intervention is made.

A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)

The purpose of this trial is to evaluate whether treatment with pelabresib in combination with ruxolitinib leads to improved clinical outcomes compared to ruxolitinib alone in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not previously received Janus kinase (JAK) inhibitor therapy.

Adrenal Function in Critical Illness

An appropriate hypothalamic-pituitary-adrenal (HPA) axis response is required to survive critical illness. Primary adrenal insufficiency, relative adrenal insufficiency, tissue resistance to glucocorticoids, ACTH deficiency and immune-mediated inhibition of the HPA axis may impair the secretion or action of glucocorticoids in critically ill patients. Adrenal insufficiency is estimated to occur in up to 77% of critically ill patients, but currently, there is no consensus on the diagnostic criteria for adrenal insufficiency in this setting, and standard testing does not discriminate among the aforementioned factors. We will study the incidence and natural history of adrenal insufficiency in critically ill patients to further define adrenal insufficiency and provide data to develop diagnostic tests. Clinical features and outcomes will be correlated with laboratory measurements of hormones, cytokines and glucocorticoid action. Healthy volunteers will undergo cortrosyn tests with measurement of free cortisol levels to develop a normative range for this endpoint. \<TAB\> Previous glucocorticoid use, if prolonged and supraphysiologic, also inhibits the HPA and can result in adrenal insufficiency. Patients with short intermittent courses of glucocorticoid administration have not been studied well, and may also be at risk. To gain further information about this group, patients receiving pulse glucocorticoid doses as part of bone marrow transplant regimens at the Clinical Center will also be studied.

Biorepository in Participants Who Undergo OTC for Gonadotoxic Therapy

Background: Medical advances have improved survival rates for many cancers and other illnesses. This means that more people are coping with the long-term effects of these treatments. Some treatments can cause female infertility. Ovarian tissue cryopreservation (OTC) may help. Before undergoing a treatment that may damage their fertility, patients may opt to freeze a sample of ovarian tissue. The tissue contains immature egg cells. When thawed, the tissue can be reimplanted. This procedure can help women become pregnant. Objective: This natural history study will create a databank of ovarian tissue. The NIH will provide OTC as a clinical service. The NIH will also request a portion of the tissue to use for research. Eligibility: Females aged 4 to 35 who opt to have OTC before receiving cancer treatment. Design: Participants will be screened. Their existing medical records will be reviewed. They will be asked if they want to donate a portion of their ovarian tissue for research. No more than 20% of the tissue collected will be taken for research. Some other tissues that would otherwise be discarded will also be kept. Medical data from each participant may also be collected and stored in the database. This data may include results of routine blood tests, imaging tests, and other information. The data will be coded for privacy. Participants will answer a questionnaire. They will be asked about their fertility treatment and general health. The survey takes about 30 minutes. They will repeat the questionnaire once a year for 30 years.

Deep Brain Stimulation Surgery for Movement Disorders

Background: \- Deep brain stimulation (DBS) is an approved surgery for certain movement disorders, like Parkinson's disease, that do not respond well to other treatments. DBS uses a battery-powered device called a neurostimulator (like a pacemaker) that is placed under the skin in the chest. It is used to stimulate the areas of the brain that affect movement. Stimulating these areas helps to block the nerve signals that cause abnormal movements. Researchers also want to record the brain function of people with movement disorders during the surgery. Objectives: * To study how DBS surgery affects Parkinson s disease, dystonia, and tremor. * To obtain information on brain and nerve cell function during DBS surgery. Eligibility: \- People at least 18 years of age who have movement disorders, like Parkinson's disease, essential tremor, and dystonia. Design: * Researchers will screen patients with physical and neurological exams to decide whether they can have the surgery. Patients will also have a medical history, blood tests, imaging studies, and other tests. Before the surgery, participants will practice movement and memory tests. * During surgery, the stimulator will be placed to provide the right amount of stimulation for the brain. Patients will perform the movement and memory tests that they practiced earlier. * After surgery, participants will recover in the hospital. They will have a followup visit within 4 weeks to turn on and adjust the stimulator. The stimulator has to be programmed and adjusted over weeks to months to find the best settings. * Participants will return for followup visits at 1, 2, and 3 months after surgery. Researchers will test their movement, memory, and general quality of life. Each visit will last about 2 hours.

A Study to Investigate Mocertatug Rezetecan Compared With Chemotherapy in Participants With Endometrial Cancer After Platinum-based Chemotherapy and Immunotherapy (BEHOLD-Endometrial01)

This study specifically aims to evaluate how well mocertatug rezetecan (Mo-Rez) works in treating Endometrial Cancer (EC) compared to standard of care. The study also assesses whether Mo-Rez is safe and tolerated well by participants in comparison to standard of care and will help provide a better understanding of the main side effects of the drugs.

Comparative Effectiveness and Safety of Two Hyaluronic Acid Products in Knee Osteoarthritis

Knee osteoarthritis is a condition that causes pain, stiffness, and difficulty moving the knee. Some people continue to have symptoms despite receiving usual treatments, such as pain medications, exercise, and physical therapy. One treatment option is viscosupplementation, which involves injecting a gel-like substance called hyaluronic acid into the knee joint. Different products are available, but it is not clear whether they provide similar benefits, have similar side effects, or differ in cost. The goal of this study is to compare two hyaluronic acid products, Mesoheal and Synvisc, in adults with knee osteoarthritis. The researchers want to find out: * How well each product reduces knee pain. * How much each product improves knee movement and daily activities. * What side effects may occur. * How the costs of the two treatments compare with their benefits. The study will include approximately 160 adults who need viscosupplementation as part of their usual medical care. Each person's doctor will choose the treatment based on their medical needs and routine clinical practice. The researchers will not assign treatments. Participants will be followed for six months. During this time, researchers will assess changes in pain, knee function, and any side effects that may occur. The results may help doctors and healthcare providers better understand the benefits, safety, and costs of these treatments in routine clinical care.

Intermittent Fasting and Anthropometric Parameters

This study examined whether intermittent fasting helps people lose weight and body fat, and whether it affects muscle mass. The researchers compared intermittent fasting with a diet that reduced daily energy intake without limiting meal times. The study included healthy adult volunteers aged over 18 years. Researchers randomly assigned 95 participants to one of three groups. The dietary intervention lasted three weeks, and 75 participants completed it. Participants followed one of these eating plans: Intermittent fasting with reduced energy intake: Participants ate 25% less energy than their estimated daily needs. They ate all their meals within a six-hour period each day. Reduced energy intake without intermittent fasting: Participants ate 25% less energy than their estimated daily needs. They could eat throughout the day without a fixed eating window. Intermittent fasting without reduced energy intake: Participants ate enough energy to meet their estimated daily needs. They ate all their meals within a six-hour period each day. Researchers measured body weight and body composition before and after the intervention. They used a body composition analyzer to estimate body fat, muscle mass, and other body tissues. They also contacted participants weekly to monitor their progress and any problems related to the diet. After three weeks, participants in the first group lost an average of 2.3 kilograms of body weight. Those in the second group lost 2.2 kilograms. The difference between these groups was not statistically significant. Participants in the third group lost an average of 1.1 kilograms, which was significantly less than in the other two groups. All three groups lost some body fat. However, the differences in body fat loss between the groups were not statistically significant. The group combining intermittent fasting with reduced energy intake showed a greater loss of fat-free mass than the group practicing intermittent fasting without reducing energy intake. The results suggest that reducing daily energy intake was more important for short-term weight loss than limiting meal times alone. Longer studies are needed to determine the lasting effects of these eating patterns on body weight and body composition.

A Study of Opsynvi in Adults Participants With Pulmonary Arterial Hypertension in Korea

The purpose of this study is to evaluate how safe Opsynvi (macitentan/tadalafil fixed-dose combination) is in adult participants with pulmonary arterial hypertension under routine clinical practice in Korea. Pulmonary arterial hypertension is a condition where the blood vessels that carry blood from the heart to the lungs become narrowed or stiff.

Perceived Social Support in Adult Stroke

Given the limited evidence in the literature regarding the effect of perceived social support on life satisfaction and social participation in adults with stroke, this study aimed to investigate the effect of perceived social support on these outcomes.

A Study to Investigate the Safety and Efficacy of KQB365 as Monotherapy and in Combination in Participants With Advanced Solid Malignancies

The goal of this clinical trial is to learn if KQB365 works to treat advanced solid tumor cancer in adults. It will also learn about the safety of KQB365. The main questions it aims to answer are: * What is the safe dose of KQB365 by itself, in combination with cetuximab, or in combination with KQB198? * Does KQB365 alone, in combination with cetuximab, or in combination with KQB198 decrease the size of the tumor? * What happens to KQB365 in the body? Participants will: * Receive KQB365 infusion weekly alone, in combination with cetuximab, or in combination with oral KQB198. * Visit the clinic about 9 times in the first 6 weeks, and then once every week after that.

Follow-Up Evaluation for Gene-Therapy-Related Delayed Adverse Events After Participation in Pediatric Oncology Branch Clinical Trials

Background: \- Gene therapy involves changing the genes inside the body s cells to stop disease. It is very closely regulated. People who have had this therapy may have problems months or even years later. Researchers do not know the long-term side effects, so they want to study people who have had the therapy. They want the study to continue over the next 15 years. Objective: \- To study over time the negative side effects from genetically engineered cellular therapy. This will be studied in people who have been in Pediatric Oncology Branch (POB) gene therapy trials. Eligibility: \- People who are currently or were previously in a research study with gene therapy in the National Cancer Institute POB. Design: * Participants blood will be tested right before they get the genetically changed cells. They will get the cells as part of another study. * For the next year, they will come back to the clinic or see their doctor at home at least every 3 months. They will answer questions about their health and blood will be drawn. * For the next 5 years, they will go to the clinic or see their own doctor once a year. They will have physical exam and blood will be drawn. * For 10 years after that, they will be asked every year for health information. * Participants will keep their contact information up to date with researchers. They may be phoned for more health information. * If the participant was under 18 years old when given the gene therapy and turns 18 during this follow-up, they will be asked to sign a new consent form when they turn 18.

Lysergic Acid Diethylamide (LSD) as Treatment for Cluster Headache

Background: After no official research in humans in the last 40 years, research and therapeutic uses of the serotonergic psychedelic lysergic acid diethylamide (LSD) are now re-recognized and include its use in brain research, alcoholism, anxiety associated with terminal illness, and treatment of headache disorders. Specifically, LSD has been reported to abort attacks, to decrease frequency and intensity of attacks, and to induce remission in patients suffering from cluster headache (CH). Objective: To investigate the effects of an oral LSD pulse regimen (3 x 100 µg LSD in three weeks) in patients suffering from CH compared with placebo. Design: Double-blind, randomized, placebo-controlled two-phase cross-over study design. Participants: 30 patients aged ≥ 25 and ≤ 75 years with chronic or episodic CH with predictable periods lasting approximately 2 months and attacks responding to oxygen. Main outcome measures: Changes in frequency and intensity of CH attacks assessed with a standardized headache diary Significance: CH is often rated as the most painful of all primary headaches, which not only causes significant disability, but is also associated with enormous personal, economic, and psychiatric burden. At the moment, there is no specific treatment available for CH, but serotonergic compounds represent an important drug class, especially in the abortive management of cluster attacks. However, there is a need for new treatment approaches, as CH is also often insufficiently managed with available medication. This study will evaluate the potential benefit and safety of a treatment with LSD for patients with CH.

Transcranial Direct Current Stimulation for Chemotherapy-Related Chronic Pain in Cancer Survivors

The goal of this clinical trial is to learn if transcranial direct current stimulation (tDCS) combined with a working-memory task can treat chronic pain and related emotional and cognitive problems after chemotherapy in adult cancer survivors. tDCS is a non-invasive and painless technique that uses a very weak electrical current, applied through two small electrodes on the head, to modulate the activity of certain brain areas. The main questions it aims to answer are: * Does tDCS reduce chronic pain intensity after chemotherapy in cancer survivors? * Does tDCS also reduce anxiety, depression, and cognitive problems, and improve the regulation of cortisol, a stress-related hormone? * Are the effects on pain, anxiety, depression, cognitive problems, and cortisol sustained when treatment continues with a maintenance phase of up to 2 months? Researchers will compare real tDCS to sham (placebo) tDCS, a look-alike procedure that does not deliver effective stimulation, to see if real tDCS works to reduce pain and improve these symptoms. Participants will: * Receive real or sham tDCS (assigned by chance) in 5 daily sessions over 1 week. Each session lasts 20 minutes: 10 minutes at rest and 10 minutes while doing a working-memory task * If they did not respond to sham tDCS, be offered 5 additional daily sessions of real tDCS * If they respond to treatment, continue with real tDCS twice a week for up to 2 months * Complete assessments of pain, mood, memory, attention, and quality of life before treatment, after each phase, and during and at the end of the maintenance phase * Collect saliva samples to measure cortisol

A Study of Novel Agents or Combinations as Perioperative Treatment in Participants With Locally Advanced Resectable Gastroesophageal Adenocarcinoma

GEMINI-PeriOp GC study will assess the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of novel agents or novel combinations as perioperative treatment in participants with locally advanced resectable gastric, gastroesophageal junction (GEJ), or esophageal adenocarcinoma who have not received previous treatment for the disease.

Efficacy and Safety Study of Dovramilast in People With Leprosy Type 2 Reaction

Dovramilast has not been approved for leprosy type 2 reaction (erythema nodosum leprosum, ENL) or any other disease anywhere in the world. In this study, an experimental drug called dovramilast is being tested to see how it compares to current treatments for leprosy type 2 reaction. Specifically, this study aims to assess the efficacy of 100mg or 150 mg dovramilast compared with standard treatments (also known as standard of care). This study also aims to assess the safety of two strengths in adults with leprosy type 2 reaction.

Measuring Brain Changes Following Cognitive Intervention in Pediatric Patients With Brain Tumors

The goal of this study is to learn if an online computerized training program improves processing speed, attention, and working memory in children with a primary brain tumor during and after active cancer treatment. The main questions it aims to answer are: 1. Is the cognitive intervention feasible to administer to children with brain tumors during and after cancer treatment?, 2. Is the cognitive intervention acceptable to children and caregivers?, and 3. Does the cognitive intervention improve neuropsychological functioning? There is no comparison group for this study. Participants will: 1. Complete an online computerized cognitive training program for approximately 15 minutes per day, 5 days a week, for 8 weeks; 2. Have weekly check-ins with the cognitive coach, 3. Complete a survey at the end of the cognitive training program, and 4. Complete pre- and post- neuropsychological testing.

Massive Online Open Validation and Evaluation of Large Language Models for Resource Constrained Settings - a Study of Contextual Awareness, Safety, and Accuracy

Healthcare settings in low and middle income countries, clinicians are frequently required to manage a wide range of acute conditions under the constraints of time pressure, limited diagnostic resources, and variable availability of up-to-date clinical guidelines. Large language models (LLMs), which can synthesize and generate medical information in a natural language, have been proposed as potential clinical decision support tools. Most evaluations of LLMs in medicine have relied on standardized benchmark examinations or hypothetical case vignettes, often derived from high-income country contexts. MOOVE (Massive online open validation and evaluation) framework was developed to address the need for rigorous and context-sensitive evaluation of LLMs through a staged approach. Study is a non-interventional, prospective study designed to test LLMs safely in real clinical settings, evaluate their performance against human care, and explore how feasible their future implementation in healthcare might be.

Early Feasibility Study of NeoChord Transcatheter Mitral Repair System

Safety and performance evaluation of Transcatheter Mitral Repair System in subjects with moderate to severe (\>3+) degenerative mitral regurgitation.

Intra-Nasal Mechanical Stimulation (INMEST) as a Potential Preventative Treatment for Migraine

This is a prospective, randomized, open-label, delayed-start clinical investigation evaluating the safety and efficacy of the Walther System, a Class IIa investigational medical device delivering intra-nasal mechanical stimulation (INMEST), as a preventative treatment for migraine. A total of 110 adults (18-65 years) with a history of migraine will be randomized 1:1 into two groups: Group A (early treatment) and Group B (delayed treatment). The study includes baseline assessments, in-clinic device training, and home-based self-administered treatments every second day for six weeks. The primary outcome is the change in monthly migraine days during the primary treatment comparison period. Secondary outcomes include headache days, migraine intensity, duration, rescue medication use, patient-reported outcomes, device compliance, and safety. The study will be conducted at one site in Sweden.

Evaluating the Safety and Tolerability of Baricitinib in Patients With Job Syndrome With Lupus-Like Disease and/or Atopic Dermatitis

Background: Autosomal dominant hyper-IgE syndrome (HIES), also called Job syndrome, is a genetic disorder that affects the immune system. It can cause skin and lung infections and problems with blood vessels, connective tissues, and bones. People with HIES often have lupus-like disease or atopic dermatitis (skin rash). Researchers want to know if a drug approved to treat other immune system diseases (baricitinib) can help people with HIES. Objective: To test baricitinib in people with HIES with lupus-like disease or skin rash. Eligibility: People aged 12 years and older with HIES with lupus-like disease or skin rash. Design: Participants will have 5 clinic visits, 4 remote visits, and 2 phone visits in 9 months. Participants will be screened. They will have a physical exam with blood and urine tests. They will have tests of the speed and pressure of blood flow through their body: Blood pressure cuffs will be placed on each arm and leg; electrodes will be placed on the wrists and a microphone on the chest. The study has a 3-month lead-in period. Participants will not take the study drug during this time. They will continue with their usual medical care. They will have 2 phone calls with the study team. Baricitinib is a tablet taken by mouth. Participants will take 1 or 2 tablets by mouth every day for 6 months. They will start with a low dose and may increase to a higher dose. Blood and urine tests will be repeated during each study visit. Other tests may also be repeated during some visits. A skin sample may also be taken....

Evaluation, Treatment and Monitoring of Patients With a Known or Suspected Parasitic Infection

The purpose of this study is to evaluate, treat and follow patients with parasitic infections. People with a known or suspected parasitic infection who are at least 1 year old may be enrolled. This study does not involve any experimental treatments. Participants will have a physical examination and laboratory tests on blood, stool, or urine. Blood samples may be collected at regular intervals, but no more than 450 ml (15 ounces) of blood will be drawn from adults, and no more than 7 ml (1-1/2 teaspoons) per kg (2.2 pounds) of body weight from children, in any 6-week period. Other tests may include x-rays, electrocardiogram (EKG), or tissue biopsy (surgical removal of a small tissue sample), depending on the individual s condition. Patients may be offered treatment or may be referred to another study that is more appropriate for the problem. Any treatment provided in this study will be according to standard medical practice for the patient s specific medical problem. Patients responses to treatment will be evaluated at regularly scheduled clinic visits. The length of time between visits and the total duration of the study for a given individual will be determined by the study doctor, based on that person s medical condition.

A Phase II Study Evaluating the Efficacy and Safety of Inavolisib Plus Ribociclib Plus Fulvestrant Versus Placebo Plus Ribociclib Plus Fulvestrant in Participants With Advanced Breast Cancer

A study to evaluate the efficacy and safety of triplet combination of inavolisib plus ribociclib and fulvestrant versus placebo plus ribociclib and fulvestrant in the first-line setting in participants with endocrine-therapy-resistant hormone receptor (HR)-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer (ABC).

A Study of Apalutamide in Participants With High-Risk, Localized or Locally Advanced Prostate Cancer Who Are Candidates for Radical Prostatectomy

The purpose of this study is to determine if treatment with apalutamide plus androgen deprivation therapy (ADT) before and after radical prostatectomy (RP) with pelvic lymph node dissection (pLND) in participants with high-risk localized or locally advanced prostate cancer results in an improvement in pathological complete response (pCR) rate and metastasis-free survival (MFS) as compared to placebo plus ADT.