Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and < 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.

Type: Interventional

Start Date: Dec 2025

open study

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.

Type: Interventional

Start Date: Oct 2025

open study

The purpose of this research is to address the challenges of correctly monitoring, managing, and diagnosing epilepsy in participants whose seizures are not well captured by standard electroencephalography (EEG) tests and who cannot use or are not able to use more standard monitoring techniques. This research is being done to understand how the Minder System helps physicians make decisions about participant's epilepsy treatment after an actionable event. The Minder System was granted De Novo classification by the U.S. Food and Drug Administration (FDA) and is not investigational. Participants that have completed the DETECT study and received the Minder System previously will consent to join this long-term follow-up observational study. The study will collect information about general wellbeing, use of healthcare services, and experience using the Minder data over time to support long-term epilepsy care. All participants will continue to be followed by their treating physician and undergo assessments and visits every six (6) months until two (2) years after receiving the Minder device.

Type: Observational

Start Date: Jun 2026

open study

OSANOVA is a non-randomized clinical trial which aims to compare outcomes of mandibular advancement device (MAD) and hypoglossal nerve stimulation (HGNS) therapies in moderate-to-severe OSA patients who fail, decline, or are intolerant to positive airway pressure (PAP) therapy (referred to as PAP-failing patients). The primary aim of the study is to compare the outcomes between PAP-failing moderate-to-severe OSA patients receiving MAD and those receiving HGNS therapy. Primary Outcome measures include changes in Pittsburgh Sleep Quality Index (PSQI) scores. Secondary aims will help us describe the outcomes between PAP-failing moderate-to-severe OSA patients receiving MAD and those receiving HGNS therapy. Secondary outcome measures include: - adverse events, - Epworth Sleepiness Scale (ESS), - Symptoms of Nocturnal Obstruction and Related Events (SNORE-25), - patient-reported satisfaction, - CGI-Improvement, - the rate of subjects re-selecting the treatment, and - the rate of subjects recommending the treatment. and - changes in sleep study metrics (i.e., AHI, ODI, mean arterial saturation, and Time<90%),

Type: Interventional

Start Date: Jun 2025

open study

Multiple myeloma is a cancer of the plasma cells in the bone marrow. The main aim of this study is to learn how well the Immune Globulin Infusion (human), 10 percentage (%) (IGI, 10%) can help prevent infections in participants with multiple myeloma receiving B-cell maturation antigen (BCMA) x cluster of differentiation 3 (CD3) directed bispecific antibody therapy. Participants will be randomly assigned to one of two groups: 1. Primary infection prevention group: They will receive IGI, 10% for 12 months. 2. Secondary infection prevention group: They will only receive IGI, 10% if they develop a serious infection during the 12 months study period. During the study, participants will visit their study clinic 15 times (for 4-week dosing interval) or 19 times (for 3-week dosing interval) and their total participation duration will be up to 14 months (including screening period of up to 8 weeks).

Type: Interventional

Start Date: Jan 2026

open study

Phase I: Characterize safety and tolerability of ECI830 as a single agent and in combination with ribociclib and fulvestrant. Identify dose range for optimization/recommended dose for future studies. Phase II: Assess the anti-tumor activity of ECI830 in combination with ribociclib and fulvestrant in patients with hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) advanced breast cancer.

Type: Interventional

Start Date: Apr 2025

open study

This phase III trial compares standard therapy given after surgery (adjuvant) to standard therapy given before and after surgery (perioperative) in treating patients with stage II-IIIB non-small cell lung cancer (NSCLC) that can be removed by surgery (resectable). The usual approach for patients with resectable NSCLC is chemotherapy and/or immunotherapy before surgery, after surgery, or both before and after surgery. This study is being done to find out which approach is better at treating patients with lung cancer. Treatment will be administered according to the current standard of care at the time of enrollment. Chemotherapy options may include cisplatin, carboplatin, pemetrexed, gemcitabine, docetaxel, and vinorelbine at standard doses according to the treating physician. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of tumor cells. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of tumor cells. Pemetrexed is in a class of medications called antifolate antineoplastic agents. It works by stopping cells from using folic acid to make deoxyribonucleic acid (DNA) and may kill tumor cells. Gemcitabine is a chemotherapy drug that blocks the cells from making DNA and may kill tumor cells. Docetaxel is in a class of medications called taxanes. It stops tumor cells from growing and dividing and may kill them. Other chemotherapy drugs, such as vinorelbine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading . Immunotherapy with monoclonal antibodies, such as nivolumab, pembrolizumab, and atezolizumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread. Starting treatment with chemotherapy and immunotherapy prior to surgery and continuing treatment after surgery may be a more effective treatment option than adjuvant therapy alone in patients with stage II-IIIB resectable NSCLC.

Type: Interventional

Start Date: Dec 2024

open study

This is a Phase 1b study to assess the safety, tolerability, PK, and PD of investigational phage therapy (IP) in adults with SCI and bladder colonization (ASB). It is a single-center, randomized, double-blind, placebo-controlled study in adults with SCI with neurogenic bladders and bacteriuria who use indwelling catheters, or who require intermittent catheterization for bladder drainage.

Type: Interventional

Start Date: Feb 2025

open study

This phase III trial compares proton craniospinal irradiation (pCSI) to involved-field radiation therapy (IFRT) for the treatment of breast or non-small cell lung cancer that has spread from where it first started to the cerebrospinal fluid filled space that surrounds the brain and spinal cord (leptomeningeal metastasis). Patients with leptomeningeal metastasis (LM) may develop multiple areas of nervous system (neurologic) impairment that can be life-threatening. Radiation therapy (RT) effectively relieves local symptoms due to LM. RT uses high energy radiography (x-rays), particles, or radioactive seeds to kill cancer cells and shrink tumors. IFRT is commonly used to treat symptoms of LM. IFRT is radiation treatment that uses x-rays to treat specific areas of LM and to relieve and/or prevent symptoms. pCSI uses protons that can be directed with more accuracy than x-rays which allows treatment of the entire central nervous system space containing the cerebrospinal fluid (CSF), brain, and spinal cord. The pCSI treatment could delay the worsening of LM. Giving pCSI may be better than IFRT in treating LM in patients with breast or non-small cell lung cancer.

Type: Interventional

Start Date: Mar 2025

open study

The purpose of this study is to see if taking the study drug, Belumosudil, for 52 weeks in addition to your usual care and medication, will prevent Chronic Lung Allograft Dysfunction (CLAD) in participants who have a lung biopsy that shows evidence of rejection or inflammation to the transplanted lung(s). For this study, biopsies that show evidence of Acute Rejection (AR), Lymphocytic Bronchiolitis (LB), Organizing Pneumonia (OP) or Acute Lung Injury (ALI) are referred to as "Qualifying Biopsies"; patients who had evidence of one or more of these conditions on a recent biopsy are eligible for enrollment in this study. Belumosudil is an investigational drug that blocks a molecule in the body that reduces inflammation and scarring and may play a role in the development and progression of CLAD. Belumosudil is a drug approved by the FDA to treat adults and children 12 years and older with chronic graft-versus-host disease (cGVHD), a condition with some similarities to CLAD. The primary objective it to determine the efficacy of treatment with Belumosudil + maintenance immunosuppression (IS) versus placebo + maintenance IS on preventing the subsequent development of probable or definite CLAD, lung retransplant, or death.

Type: Interventional

Start Date: Mar 2025

open study

The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).

Type: Observational

Start Date: Dec 2023

open study

To define the frequency of monoclonal-X and polyclonal-X tumors in PHPT participants having parathyroidectomy (PTX) and to define the relationship between parathyroid tumor clonal status and multiple gland neoplasia (MGN), we will compare surgical and pathologic outcomes to tumor clonal status in a multicenter cohort of patients having bilateral neck exploration (BNE) and PTX (primary objectives).

Type: Observational

Start Date: Jul 2023

open study

Use of vaping products (e.g., electronic nicotine delivery systems, e-cigarettes) has been increasing rapidly, particularly among teens and young adults. With limited information on the long-term effects of vaping products, health information about vaping has been somewhat unclear in regards to associated health risks. Teens and young adults may be reluctant to disclose their use of vaping products to parents or health providers and instead turn to social media to share and seek out information regarding vaping risks and cessation supports. Thus, our current proposal outlines the use of social media to identify teens and young adults socially networking about vaping, the use of an online chatbot screen to evaluate individual cessation support needs, and the use of a digital intervention system to support vaping cessation. The mobile intervention used in this study is based on a widely-used evidence-based mobile intervention for combustible smoking (i.e., quitSTART) and has been adapted for vaping and young adults to include an in-app chatbot to guide users to tailored content and to motivate and encourage their cessation efforts. We aim to integrate our social media recruitment and online screening approach to connect individuals with this mobile app intervention, and will conduct a randomized controlled trial to evaluate user engagement with and preliminary efficacy of the digital intervention on reducing vaping behaviors among teens and young adults.

Type: Interventional

Start Date: Jun 2025

open study

This phase II/III trial examines whether patients who have undergone surgical removal of bladder, kidney, ureter or urethra, but require an additional treatment called immunotherapy to help prevent their urinary tract (urothelial) cancer from coming back, can be identified by a blood test. Many types of tumors tend to lose cells or release different types of cellular products including their DNA which is referred to as circulating tumor DNA (ctDNA) into the bloodstream before changes can be seen on scans. Health care providers can measure the level of ctDNA in blood or other bodily fluids to determine which patients are at higher risk for disease progression or relapse. In this study, a blood test is used to measure ctDNA and see if there is still cancer somewhere in the body after surgery and if giving a treatment will help eliminate the cancer. Immunotherapy with monoclonal antibodies, such as nivolumab and relatlimab, can help the body's immune system to attack the cancer, and can interfere with the ability of tumor cells to grow and spread. This trial may help doctors determine if ctDNA measurement in blood can better identify patients that need additional treatment, if treatment with nivolumab prolongs patients' life and whether the additional immunotherapy treatment with relatlimab extends time without disease progression or prolongs life of urothelial cancer patients who have undergone surgical removal of their bladder, kidney, ureter or urethra.

Type: Interventional

Start Date: Feb 2024

open study

This is a Phase 1/2, multicenter, open-label (unless otherwise specified in a combination-specific module) study of inlexisertib in combination with anticancer therapies. Modules within the master protocol are defined according to different combinations of inlexisertib with other anticancer agents.

Type: Interventional

Start Date: Sep 2023

open study

This Phase III Trial will determine whether adjuvant chemotherapy (ACT) added to ovarian function suppression (OFS) plus endocrine therapy (ET) is superior to OFS plus ET in improving invasive breast cancer-free survival (IBCFS) among premenopausal, early- stage breast cancer (EBC) patients with estrogen receptor (ER)-positive, HER2-negative tumors and 21-gene recurrence score (RS) between 16-25 (for pN0 patients) and 0-25 (for pN1 patients).

Type: Interventional

Start Date: Aug 2023

open study

This study aims to utilize a novel biomarker-driven approach to guide neoadjuvant treatment selection. It is the hypothesis that this will improve clinical response for postmenopausal women with clinical stage II/III ER-positive, HER2-negative breast cancer and identify those who may not require neoadjuvant chemotherapy, with a primary focus on outcomes in Black patients.

Type: Interventional

Start Date: May 2024

open study

The phase III trial compares the effect of pembrolizumab to observation for the treatment of patients with early-stage triple-negative breast cancer who achieved a pathologic complete response after preoperative chemotherapy in combination with pembrolizumab. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. This trial may help researchers determine if observation will result in the same risk of cancer coming back as pembrolizumab after surgery in triple-negative breast cancer patients who achieve pathologic complete response after preoperative chemotherapy with pembrolizumab.

Type: Interventional

Start Date: Jun 2023

open study

This trial studies how well proton beam radiation therapy compared with intensity modulated photon radiotherapy works in treating patients with stage I-IVA esophageal cancer. Proton beam radiation therapy uses a beam of protons (rather than x-rays) to send radiation inside the body to the tumor without damaging much of the healthy tissue around it. Intensity modulated photon radiotherapy uses high-energy x-rays to deliver radiation directly to the tumor without damaging much of the healthy tissue around it. It is not yet known whether proton beam therapy or intensity modulated photon radiotherapy will work better in treating patients with esophageal cancer.

Type: Interventional

Start Date: Jun 2019

open study

The goal of this clinical trial is to compare two different skin incisions used in surgery to treat trigger finger in adult patients. Trigger finger occurs when a finger gets stuck or locks because of swelling of the sheath around the tendons the help move the fingers. The main questions it aims to answer are (1) Does the type of skin cut affect how well the hand works 6 weeks after surgery? (2) Does the type of skin cut affect pain levels, scar healing, and how quickly people return to their normal activities? Orthopaedic surgeons will compare a straight incision along the finger (longitudinal incision) to a incision across the crease in the palm (transverse incision) to see if one type of incision results in better recovery, less pain, a better scar, and higher patient satisfaction with the procedure. Participants who have trigger finger and are indicated for surgery will have surgery to release the pulley in their finger using one of the two types of incisions (assigned by chance). Participants will fill out questionnaires about their hand function and pain before surgery and at follow-up visits. Participants will have their scar checked and rated at about 2, 6, and 12 weeks after surgery, and report on their pain for the first few days after surgery and when they can return to work and move their finger without pain.

Type: Interventional

Start Date: Apr 2026

open study

In this study, the investigators will evaluate the feasibility of the Wellness Program, including patient recruitment, screening and outcome measures, and feasibility of adapting the intervention with older Black surgical patients who endorse clinically significant symptoms of depression and/or anxiety.

Type: Interventional

Start Date: Jan 2026

open study

This is an open-label, three-part study to assess the pharmacokinetics (PK) and pharmacodynamics (PD) of epinephrine administered as a single dose of L-dipivefrin (IN-001) sublingual spray or drop in healthy adults. For all parts of the study, participants will undergo at least 10 hours of fasting prior to dosing. Part 1 of the study focuses on IN-001 dose/formulation exploration in a small number of participants (N=6); Part 2 of the study involving up to 12 participants will test a dose selected from Part 1 delivered as both a sublingual spray and a sublingual drop. In Part 3, one delivery method will be tested. Total number of participants is 30.

Type: Interventional

Start Date: Oct 2025

open study

The purpose of the present Phase 3 trial is to confirm the efficacy and safety of glepaglutide 10 mg twice weekly in a patient population with SBS-IF and generate additional long-term safety data. Glepaglutide is the International Nonproprietary Name and United States Adopted Name (USAN) for ZP1848.

Type: Interventional

Start Date: Feb 2026

open study

The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986368 in participants with Multiple Sclerosis Spasticity

Type: Interventional

Start Date: Jun 2025

open study

While tremendous progress has been made against HIV, both in preventing the infection and in treating AIDS, the disease it causes, AIDS-related malignancies like Kaposi sarcoma (KS) remain a significant health burden, in both the U.S. and especially the developing world. In many cases, multiple KS lesions develop simultaneously, and may progress and regress independently. Photographs are an essential part of the evaluation for KS, as reflected in their formal usage described in the KS Tumor Assessment Manual of Procedures. However, acquiring a clear, informative photo is not trivial, since anatomy is 3D and conventional imaging is 2D. The importance of accurate, quantitative 3D information is especially pronounced for the treatment of KS because when a tumor responds positively to treatment, the initial change is usually a flattening of the lesion, without any significant change in the projected 2D area. To evaluate the vertical space, along with other characteristics of a KS lesion, we have created an innovative imaging system, SkinScan3D, utilizing new commercial liquid lens technologies and AI based image analysis software, with strategies borrowed from astronomical imaging techniques previously used on NASA space telescopes. In this study, the investigators will develop and demonstrate a protocol for recording measurable 3D parameters, which may be used in a longitudinal study to rigorously monitor therapeutic responses of KS and statistically compare with that of the conventional AMC criteria.

Type: Observational

Start Date: Feb 2025

open study