Found 11072 clinical trials
Venglustat in Combination With Cerezyme in Adult and Pediatric Patients With Gaucher Disease Type 3
Cohort 3 Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing: CSF Lyso-GL1 levels Modified Friedreichs Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas Bone disease manifestations Evaluate safety …
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- 19 Feb, 2024
- 7 locations
Immunogenicity and Safety Study of a Quadrivalent Meningococcal Conjugate Vaccine When Co-administered With Routine Pediatric Vaccines in Healthy Infants and Toddlers in Europe
To describe the antibody responses against the antigens of the routine pediatric vaccines administered in a 3-dose series concomitantly with MenACYW conjugate vaccine or a licensed meningococcal vaccine. To describe the safety profile of MenACYW conjugate vaccine and a licensed meningococcal vaccine.
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- 19 Feb, 2024
- 29 locations
Immunogenicity and Safety Study of a Quadrivalent Meningococcal Conjugate Vaccine When Co-administered With Routine Pediatric Vaccines in Healthy Infants and Toddlers in Europe
To describe the antibody responses against the antigens of the routine pediatric vaccines administered in a 3-dose series concomitantly with MenACYW conjugate vaccine or a licensed meningococcal vaccine. To describe the safety profile of MenACYW conjugate vaccine and a licensed meningococcal vaccine.
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- 19 Feb, 2024
- 29 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objective: To characterize the safety and tolerability To characterize the pharmacokinetics (PK)
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- 19 Feb, 2024
- 11 locations
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objective: To characterize the safety and tolerability To characterize the pharmacokinetics (PK)
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- 19 Feb, 2024
- 6 locations
Study of a Pneumococcal Conjugate Vaccine When Administered Concomitantly With Routine Pediatric Vaccines in Healthy Toddlers and Infants
To assess the immune response (serotype specific IgG concentration) of the SP0202 formulations and Prevnar 13 1 month after the administration of one dose in toddlers (Groups 1-4) To assess the immune response (serotype specific IgG concentration) of the SP0202 formulations and Prevnar 13 1 month after the administration of …
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- 19 Feb, 2024
- 31 locations
Securing Access to Innovative Molecules in Oncology and Hematology for Children Adolescents and Young Adults
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (VIGINOM) and relying on the network of Interregional pediatric oncology appeal organizations (OIR) identified by INCa since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional …
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- 19 Feb, 2024
Analgetics vs Analgetics and Antibiotics for Pediatric Clinical and Sonographic Orchitis or Epididymitis
The study will compare clinical presentation between groups and will compare pediatric urologic outpatient clinic follow up findings.
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- 19 Feb, 2024
Communication Memory of Cancer Diagnosis Within the Pediatric Triangle
Present project aims to investigate memories related to a cancer communication diagnosis in pediatric oncology. It evaluates possible common elements and specificity between family and healthcare staff.
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- 19 Feb, 2024
Eosinophilic Esophagitis Steroid Safety Study
This observational study will follow pediatric patients with eosinophilic esophagitis who are scheduled to begin topical steroid treatment, from the initiation of treatment longitudinally to determine the safety profile of the drug.
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- 19 Feb, 2024