Clinical trials: Pediatric Disease
Graft-versus-host Disease, Acute-graft-versus-host Disease
An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)
This is a study comparing the defibrotide prophylaxis arm vs standard of care arm for the prevention of aGvHD.
- Trial name
- NCT03339297
- Testing
- Defibrotide, Standard of Care
- Where
- 62 sites in United States, Austria and 12 more
- Volunteers
- 152 people
- Sponsor
- Jazz Pharmaceuticals
Learning Disorder, Specific, Learning Disability
Examining the Medium-term Effect on Non Inferiority of the Previous Training With the DMD Poppins Clinical for Cognitive, Musical Training in Addition to Bi-monthly Speech and Reading Therapy on Reading Writing Abilities of Pediatric Patients With SLD Reading VS Control Group
Poppins-02-b is an observational follow-up study examining the medium-term effect on non-inferiority of the previous training with the digital medical device (DMN) Poppins Clinical for cognitive and musical training in addition…
- Conditions
- Learning Disorder, Specific, Learning Disability, Specific Learning Disorder, With Impairment in Reading, Dyslexia
- Trial name
- POPPINS-02-B (NCT06902610)
- Testing
- Poppins Clinical
- Where
- 2 sites in France
- Volunteers
- 306 people
- Sponsor
- Poppins
COVID-19
EPIC-Peds: A Study to Learn About the Study Medicine Called PF-07321332 (Nirmatrelvir)/Ritonavir in Patients Under 18 Years of Age With COVID-19 That Are Not Hospitalized But Are at Risk for Severe Disease
The purpose of this clinical trial is to learn about the safety, pharmacokinetics (pharmacokinetics helps us understand how the drug is changed and eliminated from your body after you take it), and efficacy (how well a study…
- Trial name
- NCT05261139
- Testing
- nirmatrelvir, ritonavir
- Where
- 73 sites in United States, Bulgaria and 5 more
- Volunteers
- 160 people
- Sponsor
- Pfizer
Pharyngitis
Pharyngitis,Group A Streptococcal Infection,Rapid Streptococcal Test,Throat Swab Culture
Comparison of Immunochromatographic Test for rapid detection of group A streptococcal antigen with culture in Pediatric patients.
- Trial name
- NCT02494219
- Testing
- Rapid Immunochromatographic streptococcal test
- Where
- Qatar
- Volunteers
- 906 people
- Sponsor
- Hamad Medical Corporation
Psoriasis
An Efficacy, Safety, and Pharmacokinetics Study of Subcutaneously Administered Ustekinumab in the Treatment of Moderate to Severe Chronic Plaque Psoriasis in Pediatric Participants Greater Than or Equal to 6 to Less Than 12 Years of Age
The purpose of this study is to evaluate the efficacy and safety of ustekinumab in pediatric participants aged greater than or equal to (>=) 6 through less than (<) 12 years with moderate to severe chronic plaque psoriasis
- Trial name
- CADMUS Jr (NCT02698475)
- Testing
- Ustekinumab 0.75 mg/kg, Ustekinumab 45 mg, Ustekinumab 90 mg
- Where
- 31 sites in United States, Belgium and 7 more
- Volunteers
- 44 people
- Sponsor
- Janssen Research & Development, LLC
Tumor Lysis Syndrome
Febuxostat for Tumor Lysis Syndrome Prevention in Hematological Malignancies of Paediatric Patients and Adults
The purpose of this study is to compare the exposure of febuxostat in pediatric patients (≥6<18 years of age) and in adults suffering from hematological malignancies at intermediate to high risk of TLS and to compare the effect…
- Trial name
- FLORET (NCT03605212)
- Testing
- Febuxostat
- Where
- 17 sites in Bulgaria, Hungary and 2 more
- Volunteers
- 30 people
- Sponsor
- Menarini Group
Depression, Bipolar I Disorder
A Study to Assess Change in Disease Activity and Adverse Events (AEs) With Cariprazine in the Treatment of Depressive Episodes in Pediatric Participants Participants (10 to 17 Years of Age) With Bipolar I Disorder.
Bipolar disorder is a severe chronic mood disorder that affects up to 4% of the adult population and 1.8% of the pediatric population in the United States.
- Trial name
- 3112 Ped BPD (NCT04777357)
- Testing
- Cariprazine
- Where
- 81 sites in United States, Puerto Rico and 1 more
- Volunteers
- 380 people
- Sponsor
- AbbVie
Arthritis, Juvenile
A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy
The purpose of this study is to evaluate the pharmacokinetics (the study of the way a drug enters and leaves the blood and tissues over time) of golimumab administered intravenously (IV) to pediatric participants with…
- Trial name
- GO-VIVA (NCT02277444)
- Testing
- Golimumab, Methotrexate
- Where
- 38 sites in United States, Argentina and 7 more
- Volunteers
- 130 people
- Sponsor
- Janssen Research & Development, LLC
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
CTX
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
The study is made up of two cohorts: a randomized double-blind crossover (placebo withdrawal with rescue) study among patients ≥ 16 years of age (adult cohort) and an open-label dose titration study among pediatric patients ≥1…
- Trial name
- NCT04270682
- Testing
- Blinded CDCA 250 mg TID, Open-Label CDCA 250 mg TID, Rescue Medication CDCA 250 mg TID
- Where
- 12 sites in United States, Brazil
- Volunteers
- 19 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Psoriasis
Maximal Usage Pharmacokinetics and Safety Study of ARQ-154 Foam 0.3% in Pediatric Participants With Scalp and Body Psoriasis
This study will evaluate the systemic exposure and characterize the plasma pharmacokinetic (PK) profile and assess the safety and tolerability of ARQ-154 foam 0.3%.
- Trial name
- NCT07340216
- Testing
- ARQ-154 Foam 0.3%
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Arcutis Biotherapeutics, Inc.
Familial Hypercholesterolemia - Homozygous
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to <12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein…
- Trial name
- ORION-19 (NCT06597006)
- Testing
- Inclisiran
- Where
- 19 sites in United States, Austria and 9 more
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency
Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
This study will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population
- Conditions
- Parenteral Nutrition Associated Liver Disease (PNALD), Essential Fatty Acid Deficiency, Malnutrition, Pediatric ALL
- Trial name
- NCT06274788
- Testing
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Where
- 10 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fresenius Kabi
Ear Infection
Application of the Belle.AI Comparative Image Reference System for Describing Chronic Ear Infections in Pediatric Patients From Low-Resource Communities
The purpose of this research is to examine the clinical effectiveness of an investigational AI-powered smartphone app that describes the physical characteristics of an otoscopy image. It is not intended to make any diagnosis.
- Trial name
- Otoscopy AI (NCT07025473)
- Testing
- Smartphone-enabled otoscopy
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- BelleTorus Corporation
Autism, Autism Spectrum Disorder
Online Evaluation of the Diagnostic Accuracy of BlinkLab's Digital Assessments for Autism
This observational study aims to evaluate how patterns of behavioral and sensorimotor responses measured using the BlinkLab Dx1 smartphone application relate to autism diagnoses in children ages 2 to 11.
- Conditions
- Autism, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD), Neurodevelopmental Conditions
- Trial name
- NCT07590973
- Testing
- BlinkLab Dx1, Retrospective Neurodevelopmental Diagnostic Assessment
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Blinklab Limited
Diabetes Mellitus, Type 1
Glucose Response of G-Pen (Glucagon Injection) in Pediatric T1D Patients
This is a sequential efficacy and safety study in pediatric patients with type 1 diabetes. Subjects will be administered insulin to induce a low normal glycemic state and will then receive an age-appropriate dose of G-Pen…
- Trial name
- NCT03091673
- Testing
- Glucagon
- Where
- 7 sites in United States
- Volunteers
- 31 people
- Sponsor
- Xeris Pharmaceuticals
Systemic Juvenile Idiopathic Arthritis
An Open-label Extension Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis and Active Systemic Manifestations Manifestations and Response Characterization Study in Canakinumab Treatment-naïve Patients With Active SJIA With and Without Fever.
This open-label extension study will permit patients with Systemic Juvenile Idiopathic Arthritis (SJIA) who previously were responsive to treatment with canakinumab and canakinumab treatment-naïve patients with active SJIA with…
- Trial name
- β-SPECIFIC 3 (NCT00891046)
- Testing
- Canakinumab
- Where
- 73 sites in United States, Argentina and 19 more
- Volunteers
- 270 people
- Sponsor
- Novartis Pharmaceuticals
Psoriasis Vulgaris, Psoriatic Arthritis
Special Drug Use-results Survey to Evaluate Safety and Efficacy of Cosentyx in Pediatric Patients With PsV, PsA, or GPP
This was a multicenter, centrally registered observational study without a control group. This observational study was a specified drug use-results survey conducted under GPSP to collect information on safety and efficacy during…
- Conditions
- Psoriasis Vulgaris, Psoriatic Arthritis, Pustular Psoriasis
- Trial name
- NCT05215561
- Testing
- Cosentyx
- Where
- 21 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Rabies, Healthy Volunteers
Study of Purified Vero Rabies Vaccine Compared With Two Reference Rabies Vaccines, Given in a Pre-exposure Regimen to Children and Adults and as Single Booster Dose to a Subset of Adults
The primary objective of this study is: To demonstrate the VRVg-2 is non-inferior to Verorab and Imovax Rabies vaccines in each age group (pediatric and adult populations) when administered as a 3-dose PrEP regimen, in terms of…
- Trial name
- VRV12 (NCT04127786)
- Testing
- Purified vero rabies vaccine - serum free - VRVg-2, Purified inactivated rabies vaccine - Verorab®, Purified inactivated rabies vaccine - Imovax® Rabies
- Where
- 4 sites in Thailand
- Volunteers
- 1,708 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Neuroblastoma
Naxitamab for High-Risk Neuroblastoma Patients With Primary Refractory Disease or Incomplete Response to Salvage Treatment in Bone and/or Bone Marrow
Children and adults diagnosed with high-risk neuroblastoma patients with primary refractory disease or incomplete response to salvage treatment in bone and/or bone marrow will be treated for up to 101 weeks with naxitamab and…
- Trial name
- NCT03363373
- Testing
- GM-CSF + Naxitamab
- Where
- 20 sites in United States, Canada and 7 more
- Volunteers
- 122 people
- Sponsor
- Y-mAbs Therapeutics
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