Clinical trials: Birth Defect
Hyperkyphosis, Adolescent, Muscle Activation
Schroth and Scapular Muscle Activation in Hyperkyphosis
This randomized controlled trial aims to investigate the effects of Schroth exercises on scapular muscle activation in children with thoracic hyperkyphosis.
- Conditions
- Hyperkyphosis, Adolescent, Muscle Activation, Scapula; Deformity, Acquired, Exercise Therapy
- Trial name
- NCT06907914
- Testing
- Schroth
- Where
- Turkey (Türkiye)
- Volunteers
- 56 people
- Sponsor
- Istanbul University - Cerrahpasa
Cystic Fibrosis
Comparison of the Airway Clearance Efficacy of Electro Flo 5000 and the G5 Flimm-Fighter in Patients With CF
It is the goal of the proposed study to compare the efficacy, as assessed primarily by sputum weight, of these two different devices (the Electroflo 500 and the G5 Flimm-Fighter) for airway clearance (AC) in CF patients with mild…
- Trial name
- NCT02423447
- Testing
- Electro-Flo Intervention, G5 Intervention
- Volunteers
- 25 people
- Sponsor
- Stanford University
Wilms Tumour
Collaborative Wilms Tumour Africa Project
Significant progress has been made in the treatment of Wilms tumor in high income countries, where survival is now around 85% - 90%.
- Trial name
- NCT01991652
- Where
- 3 sites in Cameroon, Ghana and 1 more
- Volunteers
- 400 people
- Sponsor
- Amsterdam UMC, location VUmc
Sickle Cell Disease, Beta Thalassemia
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease.
- Trial name
- NCT00408447
- Testing
- Busulfan, Fludarabine, Alemtuzumab
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Columbia University
Cystic Fibrosis, Covid19
The Effects of Telerehabilitation on Muscle Function, Physical Activity and Sleep in Cystic Fibrosis During Pandemic
The aim of this study is to evaluate the peripheral muscle function, sleep disorders and physical activity level in children with cystic fibrosis who are physically inactive at home due to social isolation and to examine the…
- Conditions
- Cystic Fibrosis, Covid19, Social Isolation
- Trial name
- NCT04742049
- Testing
- Telerehabilitation-based, Follow-up based
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Haemophilia
Effectiveness of the uPatient Platform in Prophylactic Treatment in Hemophilia
Research project whose main objective is to evaluate the use of a technology platform (uPatient) to register the prophylactic treatment of patients with hemophilia treated in different Hematology and Hemotherapy services of…
- Trial name
- uPatient (NCT02581735)
- Testing
- Patients with haemophilia
- Volunteers
- 40 people
- Sponsor
- Real Fundación Victoria Eugenia
Retinopathy of Prematurity (ROP)
Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care
Retinopathy of prematurity (ROP) is a disorder of development of the retina and its vasculature that can impact vision in vulnerable preterm neonates for a lifetime.
- Trial name
- (BabySTEPS3) (NCT07738874)
- Testing
- Ultra-widefield Optical Coherence Tomography (UWF-OCT), Wide-field ophthalmic imaging system
- Where
- 2 sites in United States
- Volunteers
- 175 people
- Sponsor
- Duke University
Cockayne Syndrome
Metabolic Study of Cockayne Syndrome
Cockayne syndrome (CS) is related to defective DNA transcription and/or repair and belongs to the family of Nucleotide Excision Repair.
- Trial name
- METABO-CS (NCT03044210)
- Testing
- Metabolic evaluation
- Where
- France
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Neurofibromatosis 1
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
Background: People with Neurofibromatosis type 1 (NF1) have an increased risk of developing plexiform neurofibromas (PNs). PNs are tumors that form in the tissue. They can form anywhere in the body.
- Trial name
- NCT04879160
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Adrenoleukodystrophy
Newborn Screening for Adrenoleukodystrophy
To test if the routine newborn screening dried blood spots can be used to test if elevation of C26:0 lysophosphatidylcholine (C26:0-lyso-PC), a status indicating adrenoleukodystrophy (ALD)
- Trial name
- NCT02952482
- Testing
- newborns testing for ALD
- Where
- Taiwan
- Volunteers
- 45,796 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
Single-ventricle, Posttraumatic Stress Disorder
The PaTHS Descriptive Correlational Longitudinal Study
The primary objective of this study is to measure parent's post-traumatic stress before and after their infant's second palliative heart surgery for single ventricle congenital heart disease.
- Conditions
- Single-ventricle, Posttraumatic Stress Disorder, Psychosocial Impairment
- Trial name
- PaTHS (NCT04785482)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Children's Mercy Hospital Kansas City
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
Chuvash Polycythemia, Cerebrovascular Disease
Pulmonary Hypertension, Hypoxia and Sickle Cell Disease
The study will look at the risk factors for pulmonary hypertension (high blood pressure in the lungs) in children and adolescents with sickle cell anemia (SCA) and examine the role of hypoxia (oxygen shortage) in the disease.
- Conditions
- Chuvash Polycythemia, Cerebrovascular Disease, Pulmonary Hypertension, Sickle Cell Anemia
- Trial name
- NCT00495638
- Where
- 7 sites in United States, Russia
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Jaundice, Neonatal, Neonatal Hyperbilirubinemia
Comparison of Phototherapy Using Neolight Skylife Versus Standardized Phototherapy for Hyperbilirubinemia in Newborns
Phototherapy has served as a primary treatment for hyperbilirubinemia in newborn populations. The light emitted through phototherapy interacts with bilirubin at the skin level to transform it into water-soluble products…
- Conditions
- Jaundice, Neonatal, Neonatal Hyperbilirubinemia, Neonatal Disorder
- Trial name
- NCT03599258
- Testing
- Skylife, Standard Therapy
- Where
- 2 sites in United States
- Volunteers
- 72 people
- Sponsor
- HonorHealth Research Institute
Muscular Dystrophies, Muscle Myopathies
Genetic and Physical Study of Childhood Nerve and Muscle Disorders
Background: Some nerve and muscle disorders that start early in life (before age 25), like some forms of muscular dystrophy, can run in families. However, the genetic causes of these disorders are not known.
- Conditions
- Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies
- Trial name
- NCT01568658
- Where
- United States
- Volunteers
- 2,586 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Atopic Dermatitis
Texting Atopic Dermatitis Patients to Optimize Learning and EASI Scores
The purpose of this study is to determine if sending text messages with treatment reminders and facts regarding atopic dermatitis (AD) to caretakers of children with AD will have a positive effect on the disease severity.
- Trial name
- TADPOLES (NCT02779426)
- Testing
- Text messages
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- Columbia University
Charcot Marie Tooth Disease (CMT), Pes Cavovarus
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study
The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.
- Conditions
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery, Exercise Training
- Trial name
- CMT-WALK (NCT07432035)
- Testing
- Functional Surgery plus Rehabilitation, Physical Exercise
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Methicillin-Resistant Staphylococcus Aureus, Atopic Dermatitis
The Underlying Mechanisms For S. Aureus Infection And Colonization Of Skin in People With Atopic Dermatitis With And Without Eczema Herpeticum (MRSA)
Staphylococcus aureus (S.aureus) is a bacterium that causes many painful skin and soft tissue conditions, such as scalded-skin syndrome, boils, or impetigo.
- Conditions
- Methicillin-Resistant Staphylococcus Aureus, Atopic Dermatitis, Eczema Herpeticum, MRSA
- Trial name
- MRSA (NCT00822276)
- Where
- 2 sites in United States
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Beta-Thalassemia
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with…
- Trial name
- NCT03381833
- Testing
- LJPC-401
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 84 people
- Sponsor
- La Jolla Pharmaceutical Company
Molar Incisor Hypomineralization
HYPERSENSITIVITY IN MOLAR INCISOR HYPOMINERALISATION USING LOW LEVEL LASER IN CHILDREN
Molar Incisor Hypomineralisation (MIH) is a qualitative defect of enamel that shows asymmetrically involvement of one to four on first permanent molars (FPM) with or without incisor teeth involvement.
- Trial name
- NCT06154733
- Testing
- Photobiomodulation usinf low level laser therapy, Resin-based sealant, Glass ionomer sealant
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Alexandria University
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