Found 11072 clinical trials
Precision Guided Tacrolimus Dosing in Pediatric Heart Transplant
The goal of this research proposal is to clinically validate an innovative precision medicine strategy for dosing the immunosuppressant tacrolimus in pediatric heart transplant, which will in turn lead to improvements in long-term transplant survival outcomes. The strategy and techniques used in this project can be extended to improve drug …
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- 19 Feb, 2024
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects 2 and 50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.
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- 19 Feb, 2024
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome
This is a Phase 1/2, open-label, multiple-dose, dose-escalating study to evaluate the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients with Angelman syndrome. Approximately 20 patients (male and female) 4 and 17 years of age with a genetically confirmed diagnosis of full maternal UBE3A …
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- 19 Feb, 2024
A Study of Subcutaneous Risankizumab Injection for Pediatric Participants With Moderate to Severe Plaque Psoriasis to Assess Change in Disease Symptoms
Psoriasis is a chronic, systemic, inflammatory disease in which skin cells build up and develop thick, red and white scaly patches on the skin. There is an unmet medical need for effective treatment in pediatric patients and this study is being done to evaluate risankizumab in pediatric participants with moderate …
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- 19 Feb, 2024
A Study of Lenvatinib (MK-7902) in Pediatric Participants With Relapsed or Refractory Solid Malignancies (MK-7902-013/E7080)
The main purpose of this study is to evaluate the antitumor activity and safety of Lenvatinib (MK-7902/E7080) in children, adolescents, and young adults with relapsed or refractory solid malignancies after administration. Participants will be enrolled into initial tumor-specific cohorts which will be expanded based on observed response.
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- 19 Feb, 2024
Phase 1/2 Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of SEL-302 in Pediatric Subjects With MMA (reiMMAgine)
Detailed Description:MMA is a rare inborn error of branched chain amino acid metabolism. Despite strict dietary adherence and vigilant monitoring and care, affected individuals have recurrent episodes of severe illness and develop complications from different organ systems that can be life-threatening. Liver transplants can help, but gene transfer therapy could …
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- 19 Feb, 2024
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CCCG Relapsed Acute Lymphoblastic Leukemia 2017 Study in Children
Study of adding anti-CD20 antibody (rituximab) with chemotherapy: CD20 is found to be expressed in 40-50% of B-lineage ALL, and rituximab has been studied in adult ALL with superior survival (75% vs 47%,). There is little experience of using rituximab in pediatric ALL thus a CCCG Relapsed ALL 2017 Study …
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- 19 Feb, 2024
Study on Clostridium Difficile Infection in Infants
This study aims to investigate the epidemiological characteristics and biomarkers of Clostridium difficile infection in infants . By analyzing historical medical data and patient questionnaires, this retrospective cohort study will identify potential high-risk factors and establish baseline biomarkers to improve diagnosis and treatment for affected patients.
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- 03 Mar, 2025
Efficacy of Hydroxychloroquine Prophylaxis for Health Care Workers at High Risk for COVID-19
The HCW Prophylaxis (HCWP) Study, single, open and off label intervention study. Up to 350 participants will be assigned to group that takes HCQ or group that opts to not take study medication. Participants will be UNM HEALTH SYSTEM HCW at high risk for occupational exposure to SARSCoV- 2. Study …
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- 19 Feb, 2024
Study to Evaluate the Safety Tolerability Pharmacokinetics and Efficacy of Remdesivir (GS-5734 ) in Participants From Birth to < 18 Years of Age With Coronavirus Disease 2019 (COVID-19)
The primary objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of remdesivir (RDV) in participants with laboratory-confirmed coronavirus disease 2019 (COVID-19) aged 0 days to < 18 years.
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- 19 Feb, 2024