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Heart failure (HF) is a severe disease, burdened with a poor prognosis (30% mortality at 2 years, 30% of rehospitalization within 1 month). It is also a major cause of health burden representing between 1.5 and 2 billions euros per year in France. Approximately 75% of these costs…

This is a study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and anti-tumor activity of DS-1001b in patients with gliomas that harbor IDH1-R132 mutations.

The goal of this clinical research study is to learn whether delivering spine radiosurgery in a single large dose is better than delivering spine radiosurgery over 3 smaller doses. Researchers also want to learn about the effects of a single dose on participant's symptoms, pain,…

The purpose of the study is to see if daily transdermal nicotine is able to produce a significant cognitive, clinical and functional improvement in participants with MCI. Neuronal nicotinic receptors have long been known to play a critical role in memory function in preclinical s…

This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in participants with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.

This hypothalamic obesity is associated with serious metabolic and psychosocial consequences. The purpose of the study is to compare the change of body weight after 6 months treatment with a lifestyle intervention + exenatide compare to the one after the same lifestyle intervent…

Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes. While recent work has demonstrated that whole genome sequencing (WGS), ha…

Part 1: Biomarker evaluation/screening phase Primary Objectives: * Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease Type 3 (GD3) participants that distinguish GD3 from adult Gaucher disease Type 1 (GD1) participants * Screen adult GD3 participants who qual…

The ultimate aim of this study is to identify a biomarker of suicide risk in MDD by measuring the "hedonic spectrum" (pain and reward responsivity), attention and its associated brain structures using brain scans (fMRI and DTI), as well as the stability of markers over time.

Post-Traumatic Stress Disorder (PTSD) is an anxiety disorder that can develop after exposure to a terrifying event or ordeal in which there was the potential for or actual occurrence of grave physical harm. Traumatic events that may trigger PTSD include violent personal assaults,…

To investigate the effectiveness of the JAK 1/3 inhibitor tofacitinib in treating Blau syndrome and explore the association between various clinical and genetic features and therapeutic responses within the cohort.

Parkinson's disease (PD) is the second most common neurodegenerative disorder causing motor and non-motor symptoms. PD is characterized by death of dopaminergic (DA) neurons in the substantia nigra (SN) pars compacta and formation of inclusions known as Lewy bodies (LBs) that pri…

This study evaluates the efficacy and the safety of the HIFU for the treatment of breast fibroadenoma.

The purpose of this phase 2 multicenter, randomized, double-blind, placebo-controlled, study is to assess the safety and efficacy of ifetroban in patients with diffuse cutaneous systemic SSc (dcSSc) or SSc-associated pulmonary arterial hypertension (SSc-PAH).

To improve upon the non-invasive detection of BCa by further validating a multiplex ELISA assay directed at a BCa-associated diagnostic signature in voided urine samples of patients with gross hematuria.

This is an open label, multi-drug, biomarker-directed, multi-centre, multi-arm, Phase 1b study in patients with muscle invasive bladder cancer (MIBC) (urothelial) who have progressed on prior treatment. This study is modular in design, allowing evaluation of the safety, tolerabil…

This is a Phase 2a, multicenter, randomized, subject-blind, investigator-blind, study to investigate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of bimekizumab in adult subjects with moderate to severe chronic plaque psoriasis

The purpose of this study is to evaluate the safety and tolerability of itacitinib in combination with osimertinib in subjects with locally advanced or metastatic non-small cell lung cancer (NSCLC).

Treatment of regionally-advanced head and neck squamous cell carcinoma (HNSCC) requires a multidisciplinary approach with a combination of surgery, radiotherapy (RT) and chemotherapy. Due to these aggressive combined modalities, patients undergoing treatment and many survivors de…

This research is being done to evaluate multimodality imaging, including magnetic resonance imaging-guided therapy (MRT), as a possible treatment for gynecologic cancers. The therapy takes place in the Department of Radiation Oncology at the Johns Hopkins SKCCC. The purpose of th…