Clinical trials: Functional Deformity
Hypoxia Ischemia, Cerebral, Hypoxia-Ischemia, Brain
Functional Imaging of Baby Brains
Infants are at risk of developing motor and cognitive neurodevelopmental disabilities as a sequelae to hypoxic-ischemic brain injury during the perinatal period.
- Conditions
- Hypoxia Ischemia, Cerebral, Hypoxia-Ischemia, Brain, Hypoxia Neonatal, Hypoxic-Ischemic Encephalopathy
- Trial name
- FIBB (NCT05514665)
- Testing
- Diffuse Optical Tomography
- Where
- Canada
- Volunteers
- 25 people
- Sponsor
- Hamilton Health Sciences Corporation
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
Pectus Excavatum, Pectus Carinatum
Chest Wall Deformities Beyond the Chest Wall
Pectus excavatum (PE) and pectus carinatum (PC) are chest wall deformities that may influence respiratory function, physical capacity, posture, and quality of life.
- Trial name
- BEYOND (NCT07844382)
- Where
- Turkey (Türkiye)
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- Eastern Mediterranean University
Fabry Disease
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients.
- Trial name
- LDN6702 (NCT01581424)
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- University of Minnesota
Friedreich Ataxia
Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients
Friedreich's Ataxia (FA) is an autosomal recessive disease the mutation of which leads to a deficiency of a protein called frataxin, which is responsible for the symptoms of the disease.
- Trial name
- CalcitriolFA (NCT04801303)
- Testing
- Calcitriol, Blood analysis for frataxin's level measurement, Blood analysis for hypercalcemia's control
- Where
- Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Berta Alemany
Friedreich's Ataxia, Motor Disorders
Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
Prospective, exploratory, multicenter pilot study investigating the structural and functional connectome in patients with Friedreich's Ataxia (FRDA) using high-density electroencephalogram (HD-EEG).
- Trial name
- CONNETTOMA (NCT07095062)
- Testing
- HD-EEG recordings, combined with cognitive and motor assessment
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Cystic Fibrosis, Inflammation
Connexin Genotypes in Cystic Fibrosis
Background: There is wide variety in lung disease phenotype for the delta F508 (homozygous) genotype. A leukocyte driven inflammation is most important for the pathogenesis of pulmonary disease in CF.
- Trial name
- NCT04242420
- Testing
- Genotyping, Lung function, Microbiology
- Where
- 3 sites in Germany, Netherlands
- Volunteers
- 300 people
- Sponsor
- University Childrens' Hospital (Zentrum für Kinderheilkunde des Universitätsklinikum Bonn)
Congenital Diaphragmatic Hernia
Microbiome and Volatile Organic Compounds in Patients With CDH
Despite improved prenatal diagnostics and therapeutic possibilities, congenital diaphragmatic hernia (CDH) represents a cross-disciplinary challenge.
- Trial name
- CDHVOCS (NCT03787160)
- Testing
- initial VOC, initial fecal microbiome, initial pulmonary microbiome
- Where
- Austria
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Congenital Diaphragmatic Hernia
Preliminary Study of Pediatric Diaphragmatic Elastography
This diaphragmatic elastography study is a preliminary pilot study to have first values of diaphragmatic stiffness in patients with a history of diaphragmatic hernia and to estimate the difference in stiffness compared to a group…
- Trial name
- ECHODIA (NCT06666140)
- Testing
- Ultrasonic elastography
- Where
- France
- Volunteers
- 80 people
- Sponsor
- University Hospital, Strasbourg, France
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Becker Muscular Dystrophy
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
- Trial name
- MESA (NCT06066580)
- Testing
- Sevasemten
- Where
- 48 sites in United States, Australia and 10 more
- Volunteers
- 260 people
- Sponsor
- Edgewise Therapeutics, Inc.
Fragile X Syndrome
Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals With Fragile-X Syndrome
This study focuses on the therapeutic relevance of the endocannabinoid (eCB) system for the treatment of Fragile-X syndrome (FXS), the primary hereditary cause of autism spectrum disorder (ASD).
- Trial name
- CANAX (NCT06261502)
- Testing
- CBD Oral Solution
- Where
- Canada
- Volunteers
- 40 people
- Sponsor
- Université de Sherbrooke
Hirschsprung Disease, Ganglion
The Association Between Calretinin and the Function of Ganglion Cells in Hirschsprung Disease
This study aims to compare the outcomes of patients with long segment Hirschsprung disease or total colonic aganglionosis who had negative calretinin staining and positive ganglion cells on the proximal resection margins to those…
- Conditions
- Hirschsprung Disease, Ganglion, Hirschsprung Disease, Long-Segment, Hypoganglionosis
- Trial name
- NCT04149093
- Testing
- Calretinin staining
- Where
- Canada
- Volunteers
- 50 people
- Sponsor
- St. Justine's Hospital
Leukemia, Down Syndrome
Neurocognitive and Psychosocial Outcomes in Survivors of Childhood Leukemia With Down Syndrome
Survivors of acute lymphoblastic leukemia (ALL) are at risk for neurocognitive deficits, reduced educational and psychosocial outcomes, and lower quality of life.
- Trial name
- NCT02859389
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- St. Jude Children's Research Hospital
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Thalassemia Majors (Beta-Thalassemia Major)
Effect of Omega 3 Fatty Acids on Heart and Liver in Beta Thalassemia Patients
the aim of this interventional study to show the effect of taking omega 3 for about 6 months for patients beta thalassemia major maintained on standard of care and receiving the iron chelator deferasirox on liver, cardiac…
- Trial name
- NCT07863791
- Testing
- Omega 3 Fish Oil supplements, Deferasirox
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Ain Shams University
Pes Planus, Pronated Foot
Effect of Trunk Stabilization on Pronated Feet
the purpose of this study is to investigate The effect Trunk stabilization on dynamic balance and foot function of pronated foot posture.
- Trial name
- NCT07734168
- Testing
- trunk stabilization exercise, pronated foot posture exercise
- Where
- Egypt
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Kafrelsheikh University
Cerebral Palsy
Soft Tissue Release for Elbow Pronation in Cerebral Palsy
Upper extremity deformities are highly common in individuals with cerebral palsy (CP) as a result of motor control impairment and muscle imbalance.
- Trial name
- NCT07601165
- Testing
- Upper extremity soft-tissue release
- Volunteers
- 28 people
- Sponsor
- Istanbul University - Cerrahpasa
Tetralogy of Fallot
Early Right Ventricular Function After Repair Of Tetralogy Of Fallot , An Evidence Based Study.
This study aims to assessing right ventricular function early after surgical repair of tetralogy of Fallot and identifying the risk factors associated with development of RV dysfunction.
- Trial name
- NCT03470064
- Where
- 2 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Assiut University
Cystic Fibrosis
Sleep in Patients With Cystic Fibrosis
The aim of the study is to identify the parameters that are associated with nocturnal hypoventilation in children and adults with cystic fibrosis.
- Trial name
- Sommeil (NCT00804661)
- Testing
- SENTEC device
- Where
- France
- Volunteers
- 76 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris