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Last Updated: 01/06/2023

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19 Clinical Trials
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Etiologic Investigation of Cancer Susceptibility in Inherited Bone Marrow Failure Syndromes: A Natural History Study

Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with IBMFS who do not develop cancer. These cancer-...

Etiology, Pathogenesis, and Natural History of Idiopathic CD4+ Lymphocytopenia

Background: Idiopathic CD4+ lymphocytopenia (ICL) is a condition in which there is a decreased level of CD4+ lymphocytes (a type of white blood cell), which can lead to opportunistic infections or autoimmune disorders and diseases.

A Pilot Study of NT-I7, a Long-Acting Recombinant IL-7 Molecule, as an Immune Reconstitution Strategy for Lymphopenia in Patients With Progressive Multifocal Leukoencephalopathy

Background: Progressive multifocal leukoencephalopathy (PML) is a brain infection. It is caused by a virus. PML can happen in people with a weakened immune system. PML is associated with cognitive and visual impairment as well as motor and speech disturbances. There is no treatment for PML. Researchers want to see if a new drug can help.

Bilateral Orthotopic Lung Transplant in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant From Partially HLA-Matched Cadaveric Donors

Summary: The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched hematopoietic stem cell transplantation (HSCT) is safe and effective for patients aged 5-45 years with primary immunodeficiency (PID) and end-stage lung disease.

Evaluation of Serum Clusterin and Serum PTX3 Assay During Febrile Aplasia in Children Treated in Pediatric Oncology

Summary: Febrile aplasia is a common occurrence in children/adults treated with chemotherapy for malignant blood diseases or solid cancers. This acquired deficiency of immunity mainly causes susceptibility to bacterial and fungal infections, pathogens normally recognized by specific receptors of innate immunity (Pattern Recognition Receptor, PRR). Thus, the febrile episodes in the context of post-chemother...

Global Mitochondrial Registry to Define Natural History and Outcome Measures to Achieve Definite Trial Readiness for Mitochondrial Disorders

Summary: The main goal of the project is provison of a global registry for mitochondrial disorders to harmonize previous national registries, enable world-wide participation and facilitate natural history studies, definition of outcome measures and conduction of clinical trials.

The Effect of Cold Steam Application on Body Temperature in Fever Management in Children With Febrile Neutropenia

Summary: The aim of this study is to determine the effect of the cold steam application on body temperature in combination with the treatment algorithm in fever management in children with febrile neutropenia.

DEXamethasone EARLY Administration in Hospitalized Patients With Covid-19 Pneumonia and High Risk of Developing Acute Respiratory Distress Syndrome

Summary: The aim of this study is to evaluate the efficacy of dexamethasone in hospitalized adults with COVID-19 pneumonia who do not require supplementary oxygen on admission, but have high risk of developing acute respiratory distress syndrome (ARDS). This is a prospective, multicenter, phase 4, parallel-group, randomized and controlled trial that is open-label to investigators, participants and clinical...

Safety and Efficacy of Tenofovir Alafenamide to Prevent Mother-to-child Transmission of Hepatitis B Virus in Middle/Late Pregnancies With High Hepatitis B Virus DNA Load: A Prospective Multicenter Cohort Study

Summary: Hepatitis B virus (HBV) infection is still a major public health problem worldwide. There are 260 million people with chronic HBV infection in the world, and 890,000 people die of HBV-related diseases annually. Mother-to-child transmission (MTCT) is still the main transmission route of HBV in high-endemic areas, such as China, sub-Saharan Africa, etc. Fortunately, the morbidity of HBV infection is...

A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime

Summary: This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders. A regimen that maximizes host immune suppression is expected to reduce graft rejection and optimize donor cell engraftment.

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Summary: This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

Risk Factors & Outcomes for Clinically Documented Infections in Pediatric Cancer Patients With Fever & Neutropenia

Summary: The aim of the study is to identify clinical and laboratory parameters, present at the time of initial evaluation that could help predict which children with cancer, fever, and neutropenia will be at risk for developing clinically documented infections and/ or complications.

Showing 1-12 of 19

Last Updated: 01/06/2023