Found 11074 clinical trials
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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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
MEK Inhibitor and a PDL1 Inhibitor Patients With Locally Advanced and/or Metastatic Soft Tissue Sarcoma
The proposed study is a two-step trial with 1) a safety run in part conducted in pediatric patients and 2) a Phase II part in adult and pediatric patients aiming to evaluate the safety and clinical activity of atezolizumab + cobimetinib in advanced/metastatic soft tissue sarcomas.
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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
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
Gene Therapy for Fanconi Anemia Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …
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- 19 Feb, 2024