Clinical trials: Functional Deformity
Dilated Cardiomyopathy
Simvastatin Therapy in Patients With Dilated Cardiomyopathy.
Dilated cardiomyopathy (DCM) is the most common childhood cardiomyopathy and is associated with significant early morbidity and mortality. About half of patients die or require heart transplantation within 5 years of diagnosis.
- Trial name
- SavDCM (NCT03775070)
- Testing
- Simvastatin
- Where
- Taiwan
- Volunteers
- 8 people
- Sponsor
- National Taiwan University Hospital
Premature Baby 25 to 32 Weeks
Cerebral Abnormalities Detected by MRI, Realized at the Age of Term and the Emergence of Executive Functions
The survival of premature babies increased during the last decades. However, the neurocognitive long term development of these children is worrying, as demonstrated by EPIPAGE 1 study.
- Trial name
- EPIRMEX (NCT02083822)
- Testing
- term MRI, executive EF functions testing
- Where
- 15 sites in France
- Volunteers
- 600 people
- Sponsor
- University Hospital, Tours
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Hirschsprung's Disease (HD)
Neuropsychological Development and Functional Outcome Sin Children With Hirschsprung Disease at School Age
Hirschsprung's disease (HD) is a rare congenital disease (1:5000) characterized by neonatal functional low bowel obstruction that is caused by aganglionosis of the distal bowel.
- Trial name
- Hirschsprung (NCT03406741)
- Testing
- psychometric evaluation, neuropsychological evaluation
- Where
- France
- Volunteers
- 2 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Cystic Fibrosis
Exocrine Pancreatic Function Testing in Cystic Fibrosis
Purpose The purpose of this study is to develop and validate multimodal testing of exocrine pancreatic function (EPF). The investigators will be testing exocrine pancreatic function in patients with cystic fibrosis (CF).
- Trial name
- NCT01446861
- Where
- Norway
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Haukeland University Hospital
Cleft Lip and Palate
Evaluation of the Effect of Prosthetic Management for Congenital Cleft Lip and Palate Infants on the Middle Ear Function
Aim of the study was to evaluate the effect of feeding appliance for congenital cleft lip and palate infants on the middle ear function
- Trial name
- NCT06075069
- Testing
- Feeding appliance
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Alexandria University
Von Hippel-Lindau Disease, Central Nervous System Hemangioblastoma
Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas
Von Hippel-Lindau (VHL) disease is a rare hereditary cancer predisposition syndrome associated with the development of central nervous system hemangioblastomas from childhood.
- Trial name
- VHL-PED-CHB (NCT07705529)
- Where
- 3 sites in France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cystic Fibrosis
Transition From Pediatric to Adult Cystic Fibrosis Care Center
The main objective of register-SAFETIM is to assess the impact of the transition from pediatric to adult cystic fibrosis care center on changes in lung function and nutritional status of patients.
- Trial name
- SAFETIM RNM (NCT02790879)
- Testing
- Transition pediatric - adult
- Where
- France
- Volunteers
- 300 people
- Sponsor
- Hospices Civils de Lyon
Congenital Adrenal Hyperplasia
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02349503
- Testing
- NBI-77860
- Where
- 2 sites in United States
- Sponsor
- Neurocrine Biosciences
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Thalassemia, Iron Overload
Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox
Thalassemia Major patients developed Iron Overload due to blood transfusions and intestinal iron absorption. Renal function caused by Iron overload was studied in a previous study and shows principally tubular disfunction.
- Trial name
- NCT01905774
- Testing
- Deferasirox
- Where
- Israel
- Volunteers
- 36 people
- Sponsor
- HaEmek Medical Center, Israel
Down Syndrome, Trisomy 21
Executive Function Play Opportunities for Children With Down Syndrome
The Executive Function Play Opportunities (EXPO) study is a sequential, multiple assignment, randomized trial (SMART) evaluating adaptive caregiver-mediated intervention pathways for improving executive function (EF) in children…
- Trial name
- EXPO (NCT07792382)
- Testing
- EXPO, EXPO Extended, EXPO Foundations
- Where
- 2 sites in United States, Italy
- Volunteers
- 150 people
- Sponsor
- Colorado State University
Duchenne Muscular Dystrophy (DMD)
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration.
- Trial name
- Acti-nRoll (NCT07664124)
- Testing
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength
- Where
- Belgium
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Liege
Down Syndrome
Hippotherapy Versus Balance Therapy on Funvtional Mobility in Down Syndrome
Down Syndrome is a genetic condition caused by having an extra copy of chromosome 21 and 47 chromosomes instead of 46. A complete or partial extra copy of chromosome 21 is responsible for the anomaly and related structural and…
- Trial name
- NCT06502652
- Testing
- hippotherapy, balance training
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- Riphah International University
Asthma, Allergic Rhinitis
Fractional Exhaled Nitric Oxide (FeNO) Result Prediction Factors in Children With Allergic Diseases
In about 800 children with allergic diseases (asthma, allergic rhinitis, atopic dermatitis) the investigators will retrospectively and cross-sectionally analyze the influence/correlation of diagnosis, treatment methods, allergy…
- Conditions
- Asthma, Allergic Rhinitis, Atopic Dermatitis
- Trial name
- NCT00815984
- Testing
- measurements of lung function tests
- Where
- Poland
- Volunteers
- 800 people
- Sponsor
- Medical University of Lodz
Cystic Fibrosis
Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
- Trial name
- NCT00179998
- Testing
- Recombinant Human DNase (Pulmozyme)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Nationwide Children's Hospital
Primary Ciliary Dyskinesia, Kartagener Syndrome
Physical Fitness, Exercise Capacity and Activities of Daily Living in Primary Ciliary Dyskinesia: a Retrospective Study
Decreased pulmonary function, peripheral muscle strength, and exercise capacity were reported in primary ciliary dyskinesia (PCD) in recent studies.
- Conditions
- Primary Ciliary Dyskinesia, Kartagener Syndrome, Immotile Cilia Syndrome
- Trial name
- NCT05040607
- Testing
- Data Collection
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Cystic Fibrosis
Early Assessment of Respiratory Function, Inflammation and Bronchial Reshuffle Among Newborns Screened for Cystic Fibrosis
The main objective of this study is to show that there is a concordance between lung disease at 13 months and the existing 9 weeks in newborn babies with cystic fibrosis asymptomatically.
- Trial name
- NCT02883816
- Testing
- assessment of lung function
- Where
- France
- Volunteers
- 52 people
- Sponsor
- University Hospital, Montpellier
Duchenne Muscular Dystrophy
Relationship Between PFTs and Pdi in DMD
A cross-sectional study to explore the relationship between clinically assessed pulmonary function test (PFT) measures and transdiaphragmatic (Pdi) measures in Duchenne muscular dystrophy (DMD) as well as to explore the…
- Trial name
- NCT03335384
- Testing
- Esophageal Balloon, Gastric Balloon, Nasal Pressure Transducer
- Sponsor
- University of Minnesota
Asthma, Allergic Rhinitis
PF and FeNO and Clinical Characteristics in Children Being Diagnosed Due to Suspicion of Allergic Diseases
The aim of this study is to assess the influence of kind of allergic disease, allergic profile, allergen exposure, treatment method on lung function parameters, fractional exhaled nitric oxide level in children suspected and…
- Conditions
- Asthma, Allergic Rhinitis, Atopic Dermatitis, Urticaria
- Trial name
- NCT01805635
- Testing
- No intervention
- Where
- Poland
- Volunteers
- 5,500 people, healthy volunteers welcome
- Sponsor
- Medical University of Lodz