Clinical trials: Functional Deformity
Down Syndrome, Aerobic Exercise
Effects of Aerobic-Based Virtual Reality Exercise Training and Traditional Aerobic Exercise Training on Physical Fitness, Functional Capacity and Cognitive Function in Individuals With Down Syndrome
This study aims to investigate the effectiveness of aerobic-based virtual reality exercise training and traditional aerobic exercise training on physical fitness, functional capacity, cognitive functions and quality of life in…
- Conditions
- Down Syndrome, Aerobic Exercise, Virtual Reality Based Therapy, Exergame
- Trial name
- NCT06617637
- Testing
- Aerobic Based Virtual Reality Exercise Training, Traditional Aerobic Exercise Training
- Where
- Turkey (Türkiye)
- Volunteers
- 44 people
- Sponsor
- Bezmialem Vakif University
Congenital Heart Disease
Hybrid Versus Norwood Management Strategies in Infants Undergoing Single Ventricle Palliation
The purpose of this trial is to determine, at 3 years of life, how the neurologic and functional outcomes in infants with single ventricles are different when comparing children treated with the Hybrid strategy to the Norwood…
- Trial name
- NCT01134302
- Testing
- Norwood management strategy, Hybrid Strategy
- Where
- Canada
- Volunteers
- 50 people
- Sponsor
- The Hospital for Sick Children
Cystic Fibrosis
Longitudinal Assessment of Exercise Capacity and Vascular Function in Patients With CF
This project is an attempt to understand how Orkambi treatment affects exercise capacity and the function of the arteries in CF patients who are homozygous F508del.
- Trial name
- NCT03338595
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Augusta University
Molar-Incisor Hypomineralization (MIH), Masticatory Performance
Evaluation of Occlusal Force and Masticatory Function in Children With Molar-Incisor Hypomineralization
This study aims to evaluate right-left chewing balance and masticatory function in children with Molar-Incisor Hypomineralization (MIH) before and after restorative treatment.
- Conditions
- Molar-Incisor Hypomineralization (MIH), Masticatory Performance, Occlusal Analysis, Chewing Function
- Trial name
- NCT07589179
- Testing
- Glass Hybrid Restorative Treatment
- Volunteers
- 68 people, healthy volunteers welcome
- Sponsor
- Marmara University
BPD - Bronchopulmonary Dysplasia, Apnea of Prematurity
Early Caffeine in Preterm Neonates
This is a clinical trial which will investigate whether administration of caffeine, a respiratory stimulant, to preterm babies soon after birth can prevent the need for a breathing tube, or intubation.
- Conditions
- BPD - Bronchopulmonary Dysplasia, Apnea of Prematurity, Hemodynamic Instability, Intubation
- Trial name
- NCT03086473
- Testing
- Caffeine Citrate
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Jennifer Shepherd
Spinal Muscular Atrophy
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)
To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥…
- Trial name
- SMART (NCT04851873)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 24 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
LBM & Lung Function in Adolescents With CF
In patients with cystic fibrosis (CF) the commonest cause of death is respiratory failure. Respiratory failure can have many causes.
- Trial name
- NCT02797912
- Testing
- No intervention
- Where
- United Kingdom
- Volunteers
- 29 people
- Sponsor
- King's College Hospital NHS Trust
Cystic Fibrosis, Homozygous F508del Mutation
Validation of Respiratory Epithelial Functional Assessment to Predict Clinical Efficacy of Orkambi®.
The purpose of the study is to investigate whether the correction of CFTR function by Lumacaftor/Ivacaftor in a patient-derived primary nasal cell model is a surrogate biomarker for respiratory improvement in Orkambi® treated…
- Trial name
- PREDICT-CF (NCT03894657)
- Testing
- Nasal brushing, Sputum sample, blood sample
- Where
- France
- Volunteers
- 91 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Glanzmann Thrombasthenia
The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders…
- Trial name
- NCT00230165
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Rockefeller University
Down Syndrome
Effects of Vojta Therapy on Functional Mobility and Gait Parameters in Children With Down Syndrome
This randomized clinical trial investigates the effects of Vojta therapy on functional mobility and gait parameters in children with down syndrome.
- Trial name
- NCT07248267
- Testing
- Vojta Therapy, repetitive sensorimotor exercises
- Where
- Pakistan
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Riphah International University
Duchenne Muscular Dystrophy
Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.
- Trial name
- NCT01359670
- Testing
- Tadalafil, Sildenafil
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Cedars-Sinai Medical Center
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors
Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder
Urea cycle disorders are inherited illnesses in which the body does not produce enough of the chemicals that remove ammonia, a byproduct of protein metabolism, from the blood stream.
- Conditions
- Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
- Trial name
- NCT00345605
- Testing
- Sodium Phenylbutyrate, Arginine
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Brendan Lee
Sanfilippo Syndrome, Mucopolysaccharidosis III
Study of Cannabidiol in Sanfilippo Syndrome
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in…
- Trial name
- NCT06333041
- Testing
- Epidiolex
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Esophageal Atresia, Oesophageal Atresia
Evaluation of the Respiratory Impact After Conventional or Minimally Invasive Esophageal Atresia Surgery
Right thoracotomy, conventional approach to esophageal atresia repair, leads to up to 60% radiological chest wall sequelae anomalies. The impact of these anomalies on the patient's respiratory function remains unknown.
- Conditions
- Esophageal Atresia, Oesophageal Atresia, Restrictive Lung Disease, Chest Wall Anomaly
- Trial name
- RestriMIS (NCT04136795)
- Where
- France
- Volunteers
- 500 people
- Sponsor
- University Hospital, Angers