Clinical trials: CHILD Syndrome
Marfan Syndrome
Adapted Physical Activity Program (APA) for Effort Rehabilitation of Children and Teenagers With Marfan Syndrome
This research study aims to evaluate the effect of a 6-month adapted physical activity program (APA) on the endurance capacities (evaluated as the maximum oxygen consumption [VO2 peak] on a cardiopulmonary exercise test) of…
- Trial name
- M&M's (NCT05838235)
- Testing
- Adapted Physical Activity program
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
Hunter Syndrome
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.
- Trial name
- NCT05058391
- Testing
- Elaprase
- Where
- 5 sites in India
- Volunteers
- 5 people
- Sponsor
- Takeda
Down Syndrome, Occupational Therapy
Occupational Therapy Home Programs in Children With Down Syndrome
Children with Down Syndrome frequently encounter difficulties with sensory functions and feeding, such as oral sensory processing issues and developmental disorders.
- Conditions
- Down Syndrome, Occupational Therapy, Feeding Problem
- Trial name
- NCT06244108
- Testing
- Occupational therapy home program
- Where
- Turkey (Türkiye)
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Bezmialem Vakif University
Chronic Otitis Media, Age Determined Bacteria
Bacterial Assessment of Middle Ear Fluid in Children Undergoing Ventilation Tube Placement Surgery
Children with chronic otitis media have more resistant bacteria that require surgical intervention to eradicate the infections. The study will evaluate the dominant bacteria and their sensitivities to antibiotics.
- Trial name
- NCT01157910
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Hillel Yaffe Medical Center
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Acute Respiratory Distress Syndrome
Assessment of Acute Respiratory Distress in Children Presenting to the Emergency Department: Clinical, Epidemiological, and Etiological Insights
This study investigates the clinical and epidemiological characteristics of children with acute respiratory distress presenting to the emergency unit at Assiut University Children Hospital.
- Trial name
- NCT07149389
- Volunteers
- 70 people
- Sponsor
- Assiut University
Nephrotic Syndrome in Children, Telitacicept
Study of the Telitacicept in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome
The main objective is to evaluate the effectiveness of telitacicept in pediatric patients with frequently relapsing or steroid dependent nephrotic syndrome within the 52-week follow-up.
- Trial name
- STERN (NCT06125405)
- Testing
- Telitacicept
- Where
- China
- Volunteers
- 20 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Tuberous Sclerosis Complex
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
- Trial name
- NCT05059327
- Where
- 27 sites in United States, India and 6 more
- Volunteers
- 61 people
- Sponsor
- Noema Pharma AG
Down Syndrome, Congenital Heart Disease
Congenital Heart Disease: Impact on Learning and Development in Down Syndrome (CHILD-DS)
The study objective is to compare neurodevelopmental (ND) and behavioral outcomes between children with Down syndrome (DS) who had complete atrioventricular septal defect (CAVSD) repair and children from the same clinical sites…
- Trial name
- CHILD-DS (NCT05312177)
- Testing
- Stanford Binet Intelligence Scales, Fifth Edition, Vineland Adaptive Behavior Scales Third Edition, Peabody Picture Vocabulary Test, Fifth Edition
- Where
- 14 sites in United States, Canada
- Volunteers
- 273 people, healthy volunteers welcome
- Sponsor
- Carelon Research
Myasthenia Gravis
A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis
The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to less than (<) 18 years of age (globally) and 8 to <18 years of age (for Unites Stated (US)…
- Trial name
- NCT05265273
- Testing
- Nipocalimab
- Where
- 19 sites in United States, Japan and 2 more
- Volunteers
- 12 people
- Sponsor
- Janssen Research & Development, LLC
Growth Hormone Deficiency, Turner Syndrome
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal…
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- LGS (NCT01604395)
- Where
- South Korea
- Volunteers
- 6,000 people
- Sponsor
- LG Chem
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Epilepsy, Epilepsy in Children
An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE
To assess the long-term safety and tolerability of XEN496 in pediatric subjects with KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE) who had participated in the primary study (XPF-009-301).
- Conditions
- Epilepsy, Epilepsy in Children, Epilepsy; Seizure, Disease
- Trial name
- EPIK-OLE (NCT04912856)
- Testing
- XEN496
- Where
- 4 sites in United States, Australia and 1 more
- Volunteers
- 8 people
- Sponsor
- Xenon Pharmaceuticals Inc.
Down Syndrome
Effects of Rhythmic Aerobic Exercises on Functional Balance in Children With Down Syndrome
Down syndrome (DS) is the most commonly occurring chromosomal condition and it affects more than 400,000 people in the United States. The chances of having a child with DS increases as the mother ages.
- Trial name
- NCT06196983
- Testing
- Rhythmic aerobic exercises, Control group, strength and balance exercises
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- Riphah International University
Shock Hypovolemic, Shock, Septic
Management of Shock in Children With SAM or Severe Underweight and Diarrhea
Diarrhea is one of the leading causes of under-five childhood mortality and accounts for 8% of 5.4 million global under-5 deaths.
- Conditions
- Shock Hypovolemic, Shock, Septic, Blood Transfusion, Adrenaline
- Trial name
- NCT04750070
- Testing
- Blood and Dopamine, Blood and adrenaline
- Where
- Bangladesh
- Volunteers
- 135 people
- Sponsor
- International Centre for Diarrhoeal Disease Research, Bangladesh
HIV Infections
Effect of Multiple Micronutrient Supplementation on Growth, Morbidity, and Mortality of HIV Infected Children in Uganda
Micronutrient deficiencies are common in HIV infected children and are aggravated by poor nutrition, especially in poor resource countries such as Uganda.
- Trial name
- MMS (NCT00122941)
- Testing
- multiple micronutrients
- Where
- 2 sites in Norway, Uganda
- Volunteers
- 860 people
- Sponsor
- Makerere University
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Euthyroid Sick Syndrome
Levothyroxine for Children With Euthyroid Sick Syndrome
The purpose of this study is to determine whether levothyroxine is effective and safe in the treatment of children with euthyroid sick syndrome, hospitalized in a pediatric intensive care unit.
- Trial name
- NCT01631305
- Testing
- Levothyroxine, Calcium magnesia
- Where
- Mexico
- Volunteers
- 80 people
- Sponsor
- Sinaloa Pediatric Hospital
Infant Apnea, Neonatal Abstinence Syndrome
Interventions to Help Infants Recover in the Hospital
This pilot project will evaluate independently two non-pharmacological interventions, 1) Neurosensory, Environmental Adaptive Technology (NEATCAP) and 2) Stochastic Vibrotactile Stimulation (SVS), as adjuvant non-pharmacological…
- Conditions
- Infant Apnea, Neonatal Abstinence Syndrome, Sleep
- Trial name
- NCT03881553
- Testing
- NEATCAP, SVS mattress
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Elisabeth Salisbury, PhD
Hemoglobinuria, Paroxysmal
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.
- Trial name
- NCT00867932
- Testing
- Eculizumab
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Alexion Pharmaceuticals, Inc.