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NCT05763953
NODE-202 is a Phase 2, multicenter, multinational, single dose, open-label, 2-part, sequential design study in pediatric patients with an established diagnosis of paroxysmal supraventricular tachycardia (PSVT) presenting with a symptomatic episode of PSVT. In Part 1, at least 30 patients aged 12 to \<18 years will be enrolled and treated with etripamil nasal spray (NS). Efficacy, safety, tolerability and PK (for at least 12 patients) will be assessed after administration of 70 mg etripamil NS (Part 1A). At least 18 subsequent patients will be enrolled and treated with the etripamil NS with the dose determined by the Pharmacokinetic (PK) analysis and will undergo efficacy and safety/tolerability assessments (Part 1B). In Part 2, at least 30 patients aged 6 to \<12 years will be enrolled and treated with etripamil NS at a dose selected based on appropriate body size-based modeling, as well as efficacy, safety/tolerability, and PK data collected in Part 1. Efficacy, safety, tolerability and PK (for at least 12 patients) will be assessed after administration of etripamil NS (Part 2A). At least 18 subsequent patients will be enrolled and treated with the etripamil NS with the dose determined by the PK analysis and will undergo efficacy and safety/tolerability assessments (Part 2B). The study will include the following visits: A Screening Visit, A Treatment Visit, , and A Follow-Up/End of Study Visit.
NCT03575351
The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a randomized, open-label, parallel-group, multi-center trial in adult subjects with Relapsed or refractory (R/R) aggressive Non-Hodgkin lymphoma (NHL) to compare safety and efficacy between the standard of care (SOC) strategy versus JCAR017 (also known as lisocabtagene maraleucel or liso-cel). Subjects will be randomized to either receive SOC (Arm A) or to receive JCAR017 (Arm B). All subjects randomized to Arm A will receive Standard of care (SOC) salvage therapy (R-DHAP, RICE or R-GDP) as per physician's choice before proceeding to High dose chemotherapy (HDCT) and Hematopoietic stem cell transplant (HSCT). Subjects from Arm A may be allowed to cross over and receive JCAR017 upon confirmation of an EFS event. Subjects randomized to Arm B will receive Lymphodepleting (LD) chemotherapy followed by JCAR017 infusion.
NCT02996305
The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult and adolescent subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
NCT06463340
The purpose of this study is to learn about the effects of a new study drug, called SGR-3515 that may be a treatment for advanced solid tumors.
NCT03176134
This study will evaluate the safety, tolerability, and efficacy of tedizolid phosphate (MK-1986) compared with comparator antibacterial agent in participants from birth to less than 12 years of age with acute bacterial skin and skin structure infections (ABSSSI).
NCT05505734
This is a US study comparing the efficacy and safety of BDA MDI \[Budesonide/Albuterol Sulfate (BDA) metered dose inhaler (MDI)\] with AS \[Albuterol Sulfate\] MDI, both are administered as needed for up to 12 months.
NCT06181604
The primary objective of this study is to establish an IL-6 concentration cutoff and optimal time point(s) for using Symphony IL-6 that predict 28-day mortality in patients who are admitted or are intended to be admitted to the intensive care unit (ICU) diagnosed with sepsis or septic shock.
NCT05369000
This is a phase 1, first-in-human study to evaluate Safety, Tolerability, PK, PD, Immunogenicity, and Antitumor Activity of LAVA-1207 alone or with low dose interleukin-2 or Pembrolizumab, in patients with therapy refractory metastatic castration resistant prostate cancer.
NCT03535740
The primary purpose of this study is to determine the efficacy of brigatinib by confirmed objective response rate (ORR) by response evaluation criteria in solid tumors (Response Evaluation Criteria in Solid Tumors \[RECIST\]), in participants with ALK+ locally advanced or metastatic NSCLC whose disease has progressed on therapy with alectinib or ceritinib.
NCT05480306
This is a Phase 2 randomized, open-label, two-part, multicenter study with a safety run-in to evaluate efficacy and safety of DKN-01 plus FOLFIRI/FOLFOX and bevacizumab versus standard of care (SOC) \[FOLFIRI/FOLFOX and bevacizumab\] as second-line treatment of advanced CRC patients.
NCT03649724
The LUCINDA Trial is a three-site, phase II, randomized, double-blind, placebo-controlled study of leuprolide acetate (Eligard) in women with Mild Cognitive Impairment or Alzheimer's Disease taking a stable dose of a cholinesterase inhibitor medication like donepezil. Its objective is to assess the efficacy of a 48-week regimen of leuprolide (22.5 mg per 12 weeks) compared to placebo on cognitive function, global function and plasma and neuroimaging biomarkers.
NCT05349721
This study will assess the efficacy and safety of PTC857 treatment in participants diagnosed with ALS.
NCT04565366
The overall goal of Transforming Research and Clinical Knowledge in Spinal Cord Injury (TRACK-SCI) study is to determine the relationships among the clinical, neuroimaging, cognitive, genetic and proteomic biomarker characteristics of acute traumatic spinal cord injury (SCI). TRACK-SCI seeks to combine high quality care variables with high density physiology data collection to better understand diagnose, characterize, and track the temporal profile of recovery for SCI patients. The Investigators are enrolling patients within 24 hours of injury who present to a TRACK-SCI site with a spinal cord injury that meets eligibility criteria.
NCT05403385
The study will first determine the optimal dose of inupadenant to be given in combination with carboplatin and pemetrexed to patients that progressed after receiving first line anti-PD(L)1 treatment for locally advanced or metastatic non-small cell lung cancer. The efficacy and safety of the combination is then compared to standard of care carboplatin and pemetrexed in the same populations.
NCT06285812
A pragmatic, mixed-method trial is to show the ability of OtoSight to change management of the pediatric patient presenting with ear pain in a way that improves patient outcomes and reduces costs.
NCT05150704
This is a phase III, multi-center international, single blind randomized controlled trial to test the efficacy of pulsed intravenous (IV) methylprednisolone versus standard therapy on top of maximal support in patients with Acute myocarditis (AM).
NCT05658497
The primary objectives of the study are to estimate the risk of major congenital malformations (MCMs) in infants born to women with multiple sclerosis (MS) who were exposed to diroximel fumarate (DRF) at any time from 2 weeks after the first day of their last menstrual period (LMP) up through the first trimester of pregnancy and to comparatively evaluate pregnancy outcomes with MCMs in women with MS who were exposed to DRF at any time from 2 weeks after the first day of their LMP through the first trimester of pregnancy with the following: i) women with MS who were unexposed to disease modifying therapies (DMTs) and, ii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries). The secondary objective of the study is to evaluate pregnancy outcomes in women with DRF exposure at any time from 2 weeks after the first day of their LMP through the end of pregnancy compared with the following: i) women with MS who were unexposed to DMTs, ii) women with dimethyl fumarate (DMF) exposure, iii) women with MS who were exposed to other DMTs (e.g., Avonex and Tysabri Pregnancy Registries), and iv) women without MS (e.g., women from external, general population comparators).
NCT06038578
This study will assess the efficacy, safety, optimal dose and ADA and NAbs development of TRK-950 at two separate dose levels in combination with ramucirumab and paclitaxel (RAM+PTX) as compared with RAM + PTX treatment alone in participants with gastric or gastro-esophageal junction (GEJ) adenocarcinoma.
NCT05176756
This is a two-arm, randomized, controlled trial to evaluate the effectiveness of using a behaviorally designed gamification intervention with social support compared to an attention control group to increase physical activity during a 6-month intervention with a 3-month follow-up period. We will enroll 150 Black or Hispanic breast and prostate cancer survivors who are at an especially high risk for developing major CVD from two U.S. cancer centers: the University of Pennsylvania Health System and City of Hope National Medical Center. All participants will receive a wearable activity tracker (Fitbit) and will be enrolled in the Way to Health system, a research information technology platform at the University of Pennsylvania. Within the Way to Health platform, patients will set a goal to increase daily step count from baseline, and will then be randomized to gamification plus social support or to attention control. The study will evaluate the effect of the gamification intervention on daily physical activity (as measured by daily steps and moderate to vigorous physical activity), physical function, fatigue, and health-related quality of life.
NCT06192927
This study is testing a drug called TenoMiR that is being developed for the treatment of tennis elbow (lateral epicondylitis). The study drug is a new compound that works by improving the quality of the collagen which helps repair damage to the elbow. The study drug is being developed in the hope of providing a more reliable treatment than those currently available and can be given at the time of first diagnosis, so that recovery can begin as soon as possible. The study drug has been tested in humans in an earlier study, and appeared to be safe and well-tolerated. This study aims to test the study drug in a larger number of participants.