Sep 9th, 2022 - The hypothesis is that the addition of daratumumab administered SC to standard MM regimens will improve responses compared to response data observed in completed phase 3 studies without daratumumab. Disease evaluations will include measurements of myeloma proteins, bone marrow examinations, skeletal surveys, assessment of extramedullary plasmacytomas, and measurements of serum calcium corrected...

Sep 30th, 2022 - This study will confirm the dose and evaluate the safety, acceptability, tolerability, and pharmacokinetics (PK) of oral cabotegravir (CAB), long-acting injectable cabotegravir (CAB LA), and long-acting injectable rilpivirine (RPV LA) in virologically suppressed HIV-1 infected children and adolescents aged 12 to less than 18 years. The study will include two cohorts of participants and two step...

Sep 30th, 2022 - This study's objectives include: 1) develop a better understanding of the composition and structures of breast milk over the early stages of lactation with specific emphasis on the glycans and lipids; 2) how glycan diversity relates to maternal and infant gut microbiota with an emphasis on bifidobacteria species; 3)how milk composition and structure relate to maternal health and diet. These sam...

Sep 30th, 2022 - This is an open-label, multicenter Phase 1 study to determine the safety, pharmacokinetics (PK), and antitumor activity of JCAR017 in adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), follicular lymphoma Grade 3B, and mantle cell lymphoma (MCL). This study will evaluate and refine the dose and schedule of JCAR017 to op...

Sep 30th, 2022 - Background: Neurofibromatosis type 1 (NF1) is a genetic disorder that affects approximately 1 in 3,500 individuals and is associated with a broad variety of symptoms and physical findings. Plexiform neurofibromas (PN) are histologically benign tumors which occur in 25-50% of patients with NF1 and can lead to significant morbidity. Oral therapeutic options for the treatment of plexiform neurofib...

Sep 30th, 2022 - The overarching goal of this study is to develop a comprehensive understanding of the biology and therapeutic potential of sodium-free chloride supplementation. While sodium homeostasis has been the focus of substantial investigation, very little research has been devoted to understanding chloride homeostasis. Thus, this proposal is designed to obtain the full spectrum of information pertaining...

Sep 30th, 2022 - The Johns Hopkins Global mHealth Initiative, Oxford Policy Management, and University of Cape Town proposes supporting BBC Media Action and the Ministry of Health and Family Welfare (MOHFW) to conduct an external evaluation of Mobile Academy (MA) and Kilkari. In the below text, we outline 6 proposed activities which comprise the evaluation, which are planned from mid 2018 to April 2020. These s...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) of venetoclax, ponatinib, and dexamethasone in patients with relapsed/refractory Philadelphia positive (Ph+) acute lymphoblastic leukemia (ALL) or lymphoid blastic phase (BP)-chronic myelogenous leukemia (CML). (Phase I) II. To determine the efficacy of the regimen, as defined by the rate of complete remission (CR) or CR with ...

Sep 30th, 2022 - The registry intends to provide real world data on patient management and quality of care for patients with T2DM, heat failure and chronic kidney disease in clinical practice in many countries. To bridge this gap an observational voluntary registry is set up to capture real world data on patient characteristics, disease management, healthcare utilization, and outcomes in patients with type 2 di...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. To determine the therapeutic efficacy (as measured by response rate) of abatacept + ixazomib citrate (ixazomib) + dexamethasone in multiple myeloma patients who have relapsed (or who are primary refractory) following treatment with their first proteasome inhibitor-containing regimen (excluding ixazomib), compared to historical controls of ixazomib + dexamethasone. SECONDA...

Sep 30th, 2022 - This is a non-randomized, non-inferiority trial that has been created to compare results of achalasia procedures including but not limited to: POEM + Partial Fundoplication (PF), POEM and Laparoscopic Modified Heller Myotomy + PF and POEM + Peroral Plication of the esophagus. Using a set of consistent evaluation tools for patients undergoing treatment for achalasia allows a standardized evaluat...

Sep 30th, 2022 - This is a pilot study of 120 pregnant women and their infants conducted at hospitals in the Mount Sinai Health System in New York, NY. Eighty women will deliver via elective, unlabored C-section, and 40 will undergo spontaneous vaginal delivery. The 80 women undergoing C-section will be randomized in a masked (blinded) 1:1 fashion to have their neonates undergo vaginal seeding or placebo seedin...

Sep 30th, 2022 - Despite the growing importance of maternal immunization in the control of infectious pathogens in early life, the impact of pregnancy on vaccine immunogenicity remains poorly understood. Evidence suggests that pregnancy may influence the quality of the antibody response to vaccines. Pregnancy is associated with modifications in the glycosylation profile of immunoglobulins G (IgG). Different pat...

Sep 30th, 2022 - This is an open-label, Phase I, FTIH, multiple-dose, dose-escalation and cohort expansion multi-center study conducted in three parts to identify a safe and pharmacologically active dose and regimen for VMD-928 monotherapy, which can be implemented in Phase 2 studies (the RP2D). The regimen will be identified using an adaptive design, multiple-ascending dose study in cancer patients. To conserv...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. Establish the feasibility and safety of priming CMV immunity in donors by Triplex vaccination prior to peripheral blood stem cell (PBSC) harvest. SECONDARY OBJECTIVES: I. Examine if Triplex vaccination of hematopoietic stem cell transplant (HCT) donors has an impact on CMV events. OUTLINE: Donors receive multi-peptide CMV-modified vaccinia Ankara vaccine injection between...

Sep 30th, 2022 - The objective of this study is to evaluate the efficacy and safety of oral cabozantinib at a 60 mg dose compared with a 140 mg dose in subjects with progressive, metastatic MTC. It will test if the lower dose results in similar progression free survival (PFS) and overall response rate (ORR) with fewer adverse events compared to the PFS, ORR and adverse events found in previous clinical trials o...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. To determine the sensitivity and specificity of HPV testing using different methods of detection, including HPV Hybrid Capture 2 (HC2), HPV messenger ribonucleic acid (mRNA) assays (APTIMA) and OncoHealth HPV E6/E7 oncoprotein assay and whether they improve the screening performance of routine anal cytology for the detection of anal high-grade squamous intraepithelial les...

Sep 30th, 2022 - Background: Recent advances and insights into the molecular pathogenesis of cancer have led to the development of novel molecular and biologic targeted therapies for the treatment of advanced cancer patients. A critical challenge in extending these studies involves the identification and validation of new therapeutic targets for future cancer therapies. As Surgical Oncologists at the NCI, we ha...

Sep 30th, 2022 - Objective The primary objective of this study is to characterize the natural history of disease in patients who carry a repeat expansion in the C9ORF72 gene, which causes amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The secondary objective is to assess whether candidate biomarkers correlate with disease progression. Study population 62 persons with a documented repeat ...

Sep 30th, 2022 - Over the past two decades, the number of subjects with cardiometabolic diseases (CMD) such as atherosclerotic cardiovascular disease (CVD), dyslipidemia, insulin resistance and diabetes have been rising. Characterizing these disease states reveals that inflammation is a common feature of CMD; however, mechanistic links between inflammation and these disease states in humans remain poorly unders...