TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will determine if suppressing inflammatory responses with either corticosteroids or equine anti-thymocyte globulin therapy improves survival for children with this rare, life-threatening condition.

Type: Interventional

Start Date: Feb 2022

open study

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Type: Interventional

Start Date: Nov 2021

open study

The dose escalation phase of this trial identifies the safety, side effects and best dose of ceralasertib (AZD6738) when given in combination with trastuzumab deruxtecan (DS-8201a) in treating patients with solid tumors that have a change (mutation) in the HER2 gene or protein and have spread to other places in the body (advanced). The dose expansion phase (phase Ib) of this trial compares how colorectal and gastroesophageal cancers with HER2 mutation respond to treatment with a combination of ceralasertib and trastuzumab deruxtecan versus trastuzumab deruxtecan alone. Ceralasertib may stop the growth of tumor cells and may kill them by blocking some of the enzymes needed for cell growth. Trastuzumab deruxtecan is a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug, called deruxtecan. Trastuzumab attaches to HER2 positive cancer cells in a targeted way and delivers deruxtecan to kill them. Ceralasertib and trastuzumab deruxtecan may be safe, tolerable and effective in treating patients with advanced solid tumors expressing the HER2 protein or gene.

Type: Interventional

Start Date: Aug 2021

open study

This is an open-label, single center, one cohort, non-randomized, phase II study. The aim is to evaluate the efficacy and safety of the combination of hypofractionated radiotherapy (HRT) and pembrolizumab on local tumor control in mucosal melanoma patients. Treatment effect will be compared with historical radiation therapy-alone control data.

Type: Interventional

Start Date: May 2020

open study

This study is being conducted to validate if photoacoustic imaging potentially reduces benign surgeries without compromising cancer detection sensitivity. The study will also explore whether using the photoacoustic imaging/ultrasound technique has any potential with early ovarian cancer detection in a group of high risk patients.

Type: Interventional

Start Date: Feb 2020

open study

Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia. In Phase 2, participants will be enrolled in 3 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of revumenib.

Type: Interventional

Start Date: Nov 2019

open study

This study will demonstrate the impact of taVNS on reducing adverse events in NeuroICU patients, determine if taVNS reduces length of stay, and quantify the economic benefits of taVNS implementation in a broader neurocritical care population.

Type: Interventional

Start Date: Sep 2025

open study

The purpose of this study is to see: 1. If using these two drugs (carfilzomib and belatacept) together is safe 2. If the use of these two study drugs in addition to the usual immunosuppression for kidney transplant patients can improve your transplanted kidney function by lowering the antibodies you have against your transplanted kidney 3. If the study drugs effect the immune cells that were responding to your donor kidney. And, whether blood or urine tests can measure signs of inflammation and kidney cell injury 4. If using new computer techniques can help describe important changes seen on biopsy in your donated kidneys The primary objective is to assess the efficacy of carfilzomib and belatacept therapy when added to current treatment with steroids and maintenance immunosuppression, compared to conventional treatment alone, to improve the clinical outcome of renal transplant patients with active and chronic - active ABMR occurring more than 6 months after renal transplantation or less than 6 months post-transplant with persistent refractory Antibody-Mediated Rejection (ABMR)

Type: Interventional

Start Date: Oct 2025

open study

The purpose of this study is to determine the recommended Phase 2 dose of zanzalintinib when given in combination with paclitaxel in patients with recurrent high-grade uterine cancer. Other objectives include overall safety and tolerability as well as rates of response.

Type: Interventional

Start Date: Oct 2025

open study

This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, RLY-2608, in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.

Type: Interventional

Start Date: Jun 2025

open study

This phase I trial studies the side effects and best dose of CA-4948 when given together with fluorouracil, leucovorin, oxaliplatin (FOLFOX) plus bevacizumab in treating patients with colorectal cancer that has spread from where it first started (primary site) to other places in the body (metastatic). CA-4948 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. The chemotherapy drugs used in FOLOX, fluorouracil and oxaliplatin, 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. Leucovorin is used with fluorouracil to treat colorectal cancer. Bevacizumab is in a class of medications called anti-angiogenic agents. It works by stopping the formation of blood vessels that bring oxygen and nutrients to the tumor. This may slow the growth and spread of the tumor. Giving CA-4948 with FOLFOX plus bevacizumab may be safe, tolerable and/or effective in treating patients with metastatic colorectal cancer.

Type: Interventional

Start Date: Jun 2025

open study

BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.

Type: Interventional

Start Date: May 2025

open study

Early Intervention to Promote Cardiovascular Health of Mothers and Children (ENRICH) is a cluster randomized clinical trial, funded by the National Heart Lung & Blood Institute. The study is designed to test the effectiveness of home visiting intervention to promote cardiovascular health and reduce disparities in maternal and early childhood cardiovascular health. Sites, in partnership with evidence-based home visiting programs, are recruiting 6618 participants in total (i.e., 3309 mother-child dyads which includes 3309 mothers and 3309 children) from diverse community settings with a high burden of cardiovascular disease risk factors.

Type: Interventional

Start Date: Jun 2024

open study

The study is a prospective, longitudinal, non-interventional, multicenter study of participants with HNSCC who will have tissue and blood based molecular biomarker profiling during their standard of care treatment.

Type: Observational [Patient Registry]

Start Date: Aug 2024

open study

The focus on this application is low-income, rural patients, since cardiovascular disease (CVD) prevalence is 40% higher among rural than urban residents. Health behavior counseling and follow-up care are required for patients with an elevated body mass index who have increased risk for CVD. Counseling is most effective when developed with, and tailored to, the patient and offered with resources that support healthy food intake and physical activity. Resource referral and follow-up is particularly important in rural low income residents who often have more severe social needs that impede healthy behaviors. The proposed research will leverage the candidate's digital health tool (PREVENT) for healthcare teams to use within the clinic visit. PREVENT visually displays patient-reported and electronic health record (EHR) data to facilitate counseling and deliver tailored physical activity and healthy food intake goals and resources. PREVENT may improve the quality of required care and promote cardiovascular health equity. This research will: 1) collaborate with rural and clinic partners to modify and integrate the PREVENT tool for low-income, rural patients with obesity (Aim 1); and 2) conduct a pilot pragmatic clinical trial of PREVENT to optimize feasibility, acceptability, appropriateness, and potential health equity impact.

Type: Interventional

Start Date: Aug 2024

open study

This first-in-human (FIH) dose-escalation and dose-validation/expansion study will assess KO-2806, a farnesyl transferase inhibitor (FTI), as a monotherapy and in combination, in adult patients with advanced solid tumors.

Type: Interventional

Start Date: Oct 2023

open study

The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3

Type: Interventional

Start Date: Sep 2023

open study

The goal of this clinical trial is to compare buprenorphine patch for induction (starting) of buprenorphine in pregnant patients with opioid use disorder. The main questions it aims to answer are: 1. Is there a buprenorphine induction method that results in the least moderate-to-severe opioid withdrawal symptoms in pregnant patients with opioid use disorder? 2. Is there a buprenorphine induction method that results in a higher treatment success rate? Under normal circumstances, patients who are planning to start sublingual (under the tongue) buprenorphine for opioid use disorder must first go into withdrawal to start the medication safely. Study participants will be given a buprenorphine patch during the required withdrawal period before starting sublingual treatment, and be surveyed daily by phone to assess their withdrawal symptoms. They will also be followed at prenatal appointments to evaluate treatment success based on urine drug screen results. Researchers will compare patients receiving no buprenorphine patch according to the current standard care protocol.

Type: Interventional

Start Date: Nov 2023

open study

This clinical study to evaluate sonobiopsy is significant because sonobiopsy will fundamentally enhance the clinician's insight into the molecular features of an intracranial lesion to tailor treatment approaches and optimize outcomes. In addition to the standard diagnostics of anatomic imaging and surgical histology, sonobiopsy has the potential to become the third pillar for brain tumor management by radically advancing the ability to easily and regularly acquire tumor genetic and molecular signatures. This enhanced capability will have a dramatic impact on patient survival and quality of life.

Type: Interventional

Start Date: Apr 2022

open study

This is a first in human, open-label, multi-center Phase 1 / 2 study to evaluate the safety, tolerability, and initial efficacy of AU-007 in patients with advanced solid tumors. AU-007 will be administered either as a monotherapy, or in combination with a single loading dose of aldesleukin, or with both AU-007 and aldesleukin given every 2 weeks (Q2w). Once the recommended phase 2 dose (RP2D) of AU-007 plus aldesleukin was determined, (AU-007 Q2w plus a single loading dose of aldesleukin), AU-007 plus aldesleukin is also being administered with avelumab or nivolumab.

Type: Interventional

Start Date: Apr 2022

open study

The purpose of this study is to demonstrate the safety and effectiveness of the Cardioblate iRF and CryoFlex hand held devices for the treatment of non-paroxysmal atrial fibrillation (AF).

Type: Interventional

Start Date: Nov 2018

open study

The purpose of this study is to determine the clinical benefit and characterize the safety profile of tabelecleucel for the treatment of Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) in the setting of (1) solid organ transplant (SOT) after failure of rituximab (SOT-R) and rituximab plus chemotherapy (SOT-R+C) or (2) allogeneic hematopoietic cell transplant (HCT) after failure of rituximab.

Type: Interventional

Start Date: Dec 2017

open study

This study is a randomized controlled trial that will evaluate the effect of non-invasive auricular vagal nerve stimulation on inflammatory markers, glycemic control, postoperative pain, and inflammation-related clinical outcomes after long-segment spinal fusion surgeries when compared to current accepted management.

Type: Interventional

Start Date: Oct 2025

open study

AMD is a leading cause of blindness in individuals over 50 years old, with dry AMD being the most common form. Geographic atrophy (GA) is an advanced stage of dry AMD characterized by progressive retinal cell degeneration. The primary objectives of the study are to assess the safety, tolerability, and evidence of activity of SGLT2 inhibitors in subjects with Geographic Atrophy associated with AMD.

Type: Interventional

Start Date: Oct 2025

open study

This study will assess the safety and efficacy of VS-7375 alone and in combination in patients with advanced solid tumors harboring a KRAS G12D-mutation.

Type: Interventional

Start Date: Jun 2025

open study