This single-center randomized controlled trial at Mount Sinai West will enroll 80 patients undergoing percutaneous nephrolithotomy for calcium oxalate stones. Participants will be randomized to receive either empiric therapy or selective therapy guided by 24-hour urine evaluation. The primary outcome is change in calcium oxalate supersaturation at 4 weeks, aiming to determine whether empiric therapy can provide outcomes comparable to selective therapy while simplifying access to prevention.

Type: Interventional

Start Date: Oct 2025

open study

INHALE-1st is a Phase 2, single-arm, multi-center, clinical study evaluating the safety and efficacy of Afrezza in combination with subcutaneously-injected basal insulin (BI) for youth 6 to <18 years old with newly diagnosed stage 3 type 1 diabetes (T1D). The study will also evaluate the effect of an Afrezza plus BI reigmen on participant and parent/legally authorized representative satisfaction. Participants will be followed for 13 weeks during the main phase followed by an optional Extension Phase for participants continuing to use Afrezza in combination with BI for up to 26 weeks.

Type: Interventional

Start Date: Feb 2026

open study

The current standard of care treatment for adult patients with acute myeloid leukemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation. Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die. The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes. This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given. After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared. 875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.

Type: Interventional

Start Date: Mar 2026

open study

The goal of this clinical trial is to learn whether a postpartum chatbot powered by generative artificial intelligence (genAI) can help new mothers get better pelvic floor health information and feel less lonely after childbirth. The main questions this study aims to answer are: - Does using the chatbot improve postpartum pelvic floor health knowledge? - Does using the chatbot help reduce feelings of loneliness during the postpartum period? - Does using the chatbot impact pelvic floor symptoms? Researchers will compare standard postpartum care to standard care plus the chatbot. Participants will: Be assigned by chance (like flipping a coin) to standard postpartum care with or without access to the chatbot. If in the chatbot group, participants will receive education and support via the chatbot over a 4-week period. Both groups will complete questionnaires to measure their pelvic floor knowledge, pelvic floor symptoms, feelings of loneliness, depression, infant bonding, perceived social support, adverse childhood experiences, and peri-traumatic distress. The chatbot was created by urogynecology experts in collaboration with UC San Diego computer science and biomedical informatics researchers. The chatbot is designed to give new mothers personalized, evidence-based information and support in real time.

Type: Interventional

Start Date: Jan 2026

open study

In patients with prostate cancer (PC), cardiovascular disease (CVD) causes significant morbidity and is the second leading cause of death. Both pre-existing CVD and the use of androgen deprivation therapy (ADT)-a key cornerstone of treatment for men with locally advanced or metastatic PC1,2 contribute to increased CV risk. ADT has been associated with adverse metabolic effects, including increased central adiposity, elevated low-density lipoprotein (LDL) levels, impaired glycemic control, and arterial wall remodeling and endothelial dysfunction The data demonstrates that for most patients, the status quo is insufficient6 and there remains a critical gap in the early identification of high CV-risk PC patients who may benefit most from aggressive risk mitigation strategies. Mitigation strategies, like the addition of statins as primary prevention, have shown decrease in MI/CHD death across thousands of patients. Age-related expansion of hematopoietic clones carrying recurrent somatic mutations, termed clonal hematopoiesis of indeterminate potential (CHIP) has recently been identified as a significant driver of atherosclerosis, doubling the risk of coronary heart disease. Notably, while CHIP is detectable in ~10% of persons over 70 years old, it is enriched in patients with solid malignancies, and radiotherapy exposure is among the most decisive risk factors for developing CHIP12-15. The inflammation-related metabolic signals are activated androgen signaling and exacerbated in patients with CHIP. However, the mechanistic link and clinical consequence are less understood. Therefore, it is critical to study the CV impact of CHIP and metabolic perturbations in patients with PC treated with ARSI therapy. We plan to address these critical gaps by testing our innovative hypothesis that early cardio-oncology intervention with aggressive guidelines-based CV optimization during ARPI therapy will reduce CV risk and that CHIP and metabolomics will help identify adverse metabolic remodeling to improve CV risk prediction. Robust epidemiological and clinical trial data consistently demonstrate that patients with PC are poorly optimized from a CV risk modification perspective, and existing CV risk models do not perform well in patients with cancer. The data demonstrates that for most patients, the status quo is insufficient and there remains a critical gap in the early identification of high CV-risk PC patients who may benefit most from aggressive risk mitigation strategies.

Type: Interventional

Start Date: Aug 2026

open study

The purpose of this study is to: - Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed - Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels

Type: Interventional

Start Date: Dec 2025

open study

The goal of this clinical trial is to test whether replacing sugary sodas with unsweetened, flavored sparkling waters can reduce added sugar intake and improve health in Black/African American and Latine adolescents with obesity who prefer sweet-tasting beverages. The main questions it aims to answer are: - Does replacing sugary sodas with water change liking for sugary drinks, and water? - Do shifts in liking for sweetness lead to improved diet quality and cardiometabolic health? Researchers will compare replacing sugary sodas with one of three alternative beverages: unsweetened sparkling water, plain water, and beverages with gradually reduced sugar to determine which strategy is most effective. Participants will: - Replace sugary sodas with study drinks for 4 weeks - Complete taste tests to measure their liking for and sensory experience of sweetness over 8-weeks - Provide dietary recalls, body measurements, and blood samples over 8-weeks

Type: Interventional

Start Date: Jul 2025

open study

The purpose of this study is to determine safe and effective dose (recommended phase 2 doses [RP2Ds]) of JNJ-89862175 in Part 1 (dose escalation), and to further evaluate how safe JNJ-89862175 is at the RP2Ds in Part 2 (dose expansion) in participants with advanced stage solid tumors.

Type: Interventional

Start Date: Oct 2025

open study

In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein. In this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. The main goal of this study is to see if starting salanersen before signs or symptoms appear can prevent signs or symptoms of SMA or make them less severe. Researchers will use different tests to learn if motor symptoms are changing, including the World Health Organization (WHO) motor milestones. The main questions researchers want to answer in this study are: - How many participants with 2 copies of the SMN2 gene can sit without support at 12 months? - How many participants with 3 copies of the SMN2 gene can walk alone at 18 months? Researchers will also learn more about: - The effects on participants' motor symptoms and how many new movement milestones participants achieve. - How many participants stay free of SMA symptoms - How much salanersen gets into the fluid surrounding the brain and spinal cord. - How much salanersen gets into the blood. - How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug. This study will be done as follows: - First, participants will be screened to check if they can join the study. The screening period will be up to 28 days. - This is an "open label" study. This is a study in which the participants, study doctor, and site staff know which study drug participants are receiving. In this study, all participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord. - There will be 2 parts in this study. During Part 1, participants will receive 2 doses of salanersen, about 12 months apart from each other. Part 1 will last up to 25 months. - During Part 2, participants will continue to receive salanersen. They will receive up to 3 doses, 12 months apart from each other. Part 2 will last up to 36 months. - During Part 1, participants will have up to 11 clinic visits and up to 3 phone calls. During Part 2, participants will have up to 7 clinic visits and up to 12 phone calls.

Type: Interventional

Start Date: Apr 2026

open study

This research study is being conducted to better understand the impact of the PPAL assisted toileting device. The PPAL consists of a powered, height-adjustable commode chair with integrated transfer boards. The system is designed to allow users to assist with their own transfers to and from the bed or chair to the PPAL for toileting, with minimal manual assistance from caregivers. Users who require assistance with transfers, and their caregivers, have a high likelihood of experiencing injury during transfers. The purpose of this study is to evaluate the PPAL bedside commode in a home setting to better understand the impact of the device during a six-week at-home trial. Participants will be asked to schedule a home visit with the research team to screen their home, answer questions, and observe the participant's current transfer methods to their toilet or commode to ensure that both the participant and caregiver are appropriate candidates for the device. Once approved, a delivery date will be arranged to deliver the PPAL to the participant's home. At the end of the six-week trial, the study team will arrange a final visit to retrieve the PPAL and again ask questions and observe a toilet transfer using both the PPAL and the customary commode. Finally, the investigators will interview the participant and caregiver to assess their satisfaction with the device. This interview will be audio recorded. This study involves minimal risks, which are expected to occur infrequently. There is a possibility that the participant could experience a scrape, bruise, cut, or pinch injury while using a device with which they are not familiar. There is also the possibility that the participant could fall when attempting to transfer to or from the device; however, the investigators will thoroughly demonstrate the device and monitor its use to minimize this risk. There is also a risk of breach of confidentiality, as text messages and emails may not be encrypted or secure during transmission or storage, and it is possible they could be intercepted or accessed by individuals not associated with this study. There will be no direct benefit to the participant from participating in the study, but participation will help developers of the PPAL better understand the opinions, needs, and abilities of caregivers and patients who may use the device.

Type: Interventional

Start Date: Jan 2026

open study

A pivotal, randomized, double-blind, placebo-controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of CACNA1A. The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.

Type: Interventional

Start Date: Aug 2026

open study

The purpose of this study is to evaluate the safety and efficacy of Pumitamig in combination with chemotherapy versus Nivolumab in combination with chemotherapy in participants with previously untreated advanced or metastatic gastric, gastroesophageal junction, or esophageal adenocarcinoma

Type: Interventional

Start Date: Mar 2026

open study

This clinical trial is designed to assess the safety, preliminary efficacy, and pharmacokinetics (PK) of DS3790a monotherapy and combination regimens in participants with hematological malignancies.

Type: Interventional

Start Date: Jan 2026

open study

This study will test how different doses of study medicine (Inno8) work in the bodies of people with haemophilia A. The purpose of the study is to see if Inno8 is safe to use for people with haemophilia A. The study medicine is a new medicine that cannot yet be prescribed by doctors. The study will last for about 11 weeks.

Type: Interventional

Start Date: Oct 2025

open study

The purpose of this study is to see if brenipatide when compared to a placebo works and is safe for participants with moderate-to-severe Alcohol Use Disorder (AUD). Participation in this study will last approximately 56 weeks.

Type: Interventional

Start Date: Oct 2025

open study

The purpose of this study is to see if brenipatide when compared to a placebo works and is safe for participants with Alcohol Use Disorder (AUD) and hazardous alcohol use. Participation in this study will last approximately 56 weeks.

Type: Interventional

Start Date: Oct 2025

open study

In this study, researchers will learn more about a drug called felzartamab in people who have received a kidney transplant and later developed a condition called microvascular inflammation (MVI). MVI is a type of injury to small blood vessels in the transplanted kidney and may be a sign of rejection by the body. It can lead to serious kidney problems over time. In many cases, MVI is caused by antibodies that attack the transplanted kidney. But in some people, MVI happens without these antibodies. This type of MVI is called isolated MVI. There are currently no approved treatments for isolated MVI. The main goal of the study is to learn about the effect felzartamab has on inflammation in the transplanted kidney. The main question researchers want to answer is: • How many participants have no signs of active inflammation in the transplanted kidney after 24 weeks of treatment with felzartamab? Researchers will also study how felzartamab affects kidney function, immune activity, and overall health. They will monitor safety through kidney biopsies, lab tests, and by recording adverse events throughout the study. Adverse events are health problems that may or may not be caused by the study drug. The study will be done in 2 parts as follows: - Participants will be randomly assigned to receive either felzartamab or a placebo. A placebo looks like the study drug but contains no real medicine. - In Part A, participants will receive their assigned drug for 24 weeks. Neither the researchers nor the participants will know who is receiving felzartamab or placebo. - Part B will last another 28 weeks. All participants will receive felzartamab and both participants and researchers will know this. - All treatments will be given by intravenous (IV) infusion at the study site. - Participants will have kidney biopsies at the start of the study, at Week 24, and at Week 52 to help measure changes in inflammation. - Participants will stay in the study for about 1 year.

Type: Interventional

Start Date: Jan 2026

open study

This clinical trial aims to test the effectiveness and safety of semaglutide, a GLP-1 receptor agonist, in treating moderate to severe alcohol use disorder (AUD) in Veterans. Participants who qualify will be randomly assigned to receive either semaglutide injections or placebo injections over a 24-week period, followed by a 4-week post-treatment safety assessment period. Participants receiving semaglutide will start with a low dose, gradually increasing to a maximum of 2.4 milligrams (mg) per week, depending on their tolerance. The primary measure of success will be a reduction in risky drinking, assessed through a reliable calendar-based interview method called the Timeline Follow-Back (TLFB), a well-validated calendar-based interview technique for recording daily alcohol consumption. The purpose of this research is to gather information on the safety and effectiveness of semaglutide for treating AUD, potentially offering a new and more appealing treatment option.

Type: Interventional

Start Date: Jul 2026

open study

The purpose of this study is to assess how well nipocalimab works when compared to efgartigimod in participants with generalized myasthenia gravis (a condition in which body's immune system mistakenly attacks and damages the connection between nerves and muscles causing muscle weakness).

Type: Interventional

Start Date: Jan 2026

open study

The goal of this study is to evaluate the safety and effectiveness of EVOLVE104 in participants with advanced urothelial and squamous cell carcinomas who have previously taken standard treatment options, have declined or have been ineligible for treatment with these medications. Participants with advanced or metastatic cancer who meet all eligibility criteria may be eligible to participate in the study.

Type: Interventional

Start Date: Nov 2025

open study

Researchers are looking for new ways to treat metastatic cervical cancer. Cervical cancer is cancer in the cervix, the lower part of the uterus (womb). Metastatic means the cancer has spread to other parts of the body. Researchers want to learn about giving the study medicine sacituzumab tirumotecan (also called sac-TMT or MK-2870) along with pembrolizumab and bevacizumab treatments. Sac-TMT is an antibody drug conjugate, which is a type of medicine that attaches to specific targets on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn: - About the safety of sac-TMT with pembrolizumab and bevacizumab, and if people tolerate them when given together, and - If people who receive sac-TMT and pembrolizumab, with or without bevacizumab, live longer overall or without their cancer getting worse as compared to those who receive standard treatment

Type: Interventional

Start Date: Jan 2026

open study

This study is being done to understand the effect of aripiprazole on adults who stutter. Stuttering is a disorder that affects speech fluency. This study aims to understand sensorimotor pathways of stuttering and possible interventions.

Type: Interventional

Start Date: Sep 2025

open study

The purpose of this study is to evaluate a questionnaire called PRO-AYA: Patient-Reported Outcomes for Adolescents and Young Adults.

Type: Observational

Start Date: Sep 2025

open study

Alport syndrome (AS) is a rare genetic condition that causes kidney disease, hearing loss, and eye abnormalities that occur due to changes in specific genes (COL4A3, COL4A4, and COL4A5). These genes help in producing an important protein called collagen. People with AS have a high risk of developing chronic kidney disease (CKD), a condition in which there is progressive loss in kidney function over time. The kidneys soon lose their ability to remove waste products from the body properly, resulting in end-stage kidney disease. A common sign of decreasing kidney function is the presence of excess protein in the urine that is not usually found with healthy kidneys. This condition is known as proteinuria. The study drug, BAY 3401016 (a monoclonal antibody), is a type of medicine that blocks a protein called Semaphorin 3A (Sema3A), which is thought to be involved in causing kidney damage in AS. By blocking the action of the Sema3A protein, BAY 3401016 may prevent proteinuria and slow down the loss in kidney function due to AS. The main purpose of this study is to learn more about how well BAY 3401016 works in slowing down the loss in kidney function in adults with a rapidly progressing AS.

Type: Interventional

Start Date: Nov 2025

open study

During heart surgery images of the heart are taken with transesophageal echocardiography. The images track how the heart is doing during surgery. It is normal practice to place an oral gastric tube in the stomach during heart surgery. The oral gastric tube is used to suction out stomach contents to avoid potential aspiration and avoid stomach acid build up. The purpose of this research study is to see if the images of the heart have better quality after your stomach has been emptied. This project hopes to help determine the best method to obtain the better quality images of the heart during cardiac surgery.

Type: Observational

Start Date: Oct 2025

open study