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The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.

This study is designed as a single-center, phase II exploratory clinical trial to evaluate the pathological response rate and safety of sintilimab (PD-1 inhibitor) combined with XELOX regimen (oxaliplatin + capecitabine) for neoadjuvant treatment of LARC. The study focuses on the…

A medicine that is FDA-approved for bone marrow stimulation (called sargramostim) will be tested for its safety and efficacy in individuals with mild-to-moderate Alzheimer's disease over a six month treatment period.

Acute respiratory distress syndrome (ARDS) is a severe form of acute lung injury and a major cause of Intensive Care Unit (ICU) admission worldwide. Despite a large number of randomized clinical trials, a specific and effective pharmacological approach for patients with ARDS is s…

Diversion colitis (DC) is a common inflammatory complication in patients with temporary ileostomy after rectal cancer surgery, and no standardized medical treatment exists. This prospective, assessor-blinded, parallel-group, randomized controlled trial evaluated whether autologou…

In this multiple-center phase II study, the aim is to evaluate the clinical outcome of reduced intensity conditioning regimen with fludarabine (150mg/m2), busulfan (6.4mg/kg) and melphalan (100mg/m2) in patients with myeloid malignancies including AML, MDS and CMML \>=55 years.

This study will compare the new medicine IcoSema, which is a combination of insulin icodec and semaglutide, taken once a week, to insulin icodec taken once a week in people with type 2 diabetes. The study will look at how well IcoSema controls blood sugar level in people with ty…

The primary objective is to define the safety and tolerability of AB8939 in patients with AML by determining the dose-limiting toxicities, the maximum tolerated dose, and the recommended dose for dose expansion study.

69 subjects with ALS will be enrolled in the study and randomized at a 2:1 ratio to receive the study drug or placebo tablets. Randomization sequences will be in random block sizes and stratified for ENCALS risk category \[high risk ≥ -4.5 vs. low risk \< -4.5\], and for backgrou…

This is a multicenter, single-blinded, randomized controlled clinical trial evaluating the safety and efficacy of the AGN1 LOEP SV Kit for the treatment of painful vertebral compression fragility fractures (VCFs). The objective of this study is to demonstrate non-inferiority of t…

This study will investigate the anti-anhedonic efficacy of a novel neurostimulation strategy termed accelerated intermittent theta burst stimulation (aiTBS) in participants with treatment resistant depression (TRD).

This is a multicentre, open label, two-part study to determine whether the focal adhesion kinase (FAK) inhibitor AMP945, when given prior to dosing with gemcitabine and nab-paclitaxel, improves response to therapy in first-line patients with unresectable or metastatic pancreatic …

The purpose of this study is to compare mepolizumab-based regimen to conventional therapeutic strategy for remission induction in patients with Eosinophilic Granulomatosis with Polyangiitis.

The incidence of type 2 diabetes worldwide is growing rapidly, being one of the fastest growing global health emergencies of the 21st century according to the International Diabetes Federation. In MicrobiAr the investigators seek to achieve type 2 diabetes remission through a pla…

The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered orally to pediatric participants with moderate to severe active ulcerative colitis (UC) who have had an inade…

Introduction: The increasing prevalence of obesity is particularly pronounced among adolescents. Currently available treatment options consist of structured lifestyle interventions. However, 25 % of adolescents do not respond to lifestyle treatment, why new effective treatment s…

DMX-200 (repagermanium) is a C-C chemokine receptor type 2 (CCR2) inhibitor that, when administered concurrently with an ARB, is designed to inhibit recruitment of monocytes implicated in the inflammatory chemokine environment of chronic disease. The purpose of this pivotal rando…

The goal of this clinical study is to learn more about the study drug, sacituzumab govitecan-hziy (SG), and its dosing in combination with pembrolizumab or pembrolizumab and a platinum agent (carboplatin or cisplatin), in participants with advanced or metastatic (cancer that has …

A Phase 2, Multi-Center, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of Lorlatinib Monotherapy in Crizotinib and Platinum-based Chemotherapy Treated Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer (NSCLC)Subjects in China

This product will be used as a diagnostic test to screen for the likelihood of endometriosis in menstruating adults with symptoms suggestive of endometriosis. The purpose of the test will be to guide clinical decisions regarding: 1) whether to undergo diagnostic laparoscopy to co…