Clinical trials: Newborn Diseases
Hypoxic Ischemic Encephalopathy of Newborn, Neonatal Encephalopathy
Investigating Cerebral Oxygenation in the Newborn
The goal of this single centre observational study is to use near-infrared spectroscopy (NIRS) monitoring to investigate cerebral oxygenation in two groups of newborn infants who are at high risk of brain injury.
- Conditions
- Hypoxic Ischemic Encephalopathy of Newborn, Neonatal Encephalopathy, Premature Infant Disease
- Trial name
- ICON (NCT05588960)
- Testing
- Near-infrared spectroscopy monitoring (Masimo O3 Regional Oximeter with Neonatal Sensor)
- Where
- United Kingdom
- Volunteers
- 99 people
- Sponsor
- University of Edinburgh
Chorioamnionitis
Selective Use of Antibiotics in Neonates Born to Mothers With Suspected Chorioamnionitis
Retrospective Chart Review to determine the selective use of antibiotics in neonates born to mothers with suspected chorioamnionitis
- Trial name
- Chorio (NCT03576560)
- Testing
- Retrospective Chart Review
- Where
- United States
- Volunteers
- 768 people
- Sponsor
- Pediatrix
Atopic Dermatitis
Long-term Follow-up Study to Evaluate the Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD Inj
A multi-center, open, long-term follow-up study to Evaluate the Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD inj. for Moderate to Severe Chronic Atopic Dermatitis: 5-year Results from the K0104 Extension Study
- Trial name
- NCT04730024
- Testing
- Not applicable(observational study)
- Where
- South Korea
- Volunteers
- 102 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Neurofibromatosis 1, Neurofibromatosis Type 1
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
Background: Plexiform neurofibromas are tumors that grow in and around nerves. The only way to treat them is with surgery. Some of these tumors cannot be completely removed.
- Conditions
- Neurofibromatosis 1, Neurofibromatosis Type 1, NF1, Neurofibroma, Plexiform
- Trial name
- NCT01362803
- Testing
- AZD6244
- Where
- 4 sites in United States
- Volunteers
- 99 people
- Sponsor
- National Cancer Institute (NCI)
Lysosomal Storage Diseases, Metachromatic Leukodystrophy
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human…
- Trial name
- NCT04283227
- Testing
- OTL-200
- Where
- Italy
- Volunteers
- 6 people
- Sponsor
- Orchard Therapeutics
Procedural Pain, Congenital Heart Disease
Procedural Pain Management by Multimodal Sedation Analgesia Combining Hypnosis in Children With Congenital Heart Disease
The aim of this prospective randomized controlled trial is to evaluate therapeutic hypnosis as a co-analgesia in thoracic drain removal in children with congenital heart disease.
- Conditions
- Procedural Pain, Congenital Heart Disease, Heart Surgery, Hypnosis
- Trial name
- PEACE-HYPNO (NCT06373627)
- Testing
- Hypnosis, Conventional medications used for sedation-analgesia, Medications used in addition to hypnosis
- Volunteers
- 104 people
- Sponsor
- Fondation Hôpital Saint-Joseph
Metabolic Disease, Propionic Acidemia
Natural History, Physiology, Microbiome and Biochemistry Studies of Propionic Acidemia
Background: People s bodies need to break down food into the chemicals. These chemicals are used for energy and growth. Some people cannot process all chemicals very well. Too much of some chemicals can cause diseases.
- Conditions
- Metabolic Disease, Propionic Acidemia, Organic Acidemia
- Trial name
- NCT02890342
- Where
- 2 sites in United States
- Volunteers
- 1,045 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Corneal Dystrophies, Hereditary
Descemet Stripping (Automated) Endothelial Keratoplasty (DSEK or DSAEK)
To evaluate the transplantation of a posterior corneal disc for the management of corneal endothelial disorders in Descemet Stripping Endothelial Keratoplasty (DSEK) or Descemet Stripping Automated Endothelial Keratoplasty…
- Trial name
- DSAEK (NCT00543660)
- Testing
- Descemet Stripping Automated Endothelial Keratoplasty (DSAEK)
- Where
- Netherlands
- Volunteers
- 20 people
- Sponsor
- Netherlands Institute for Innovative Ocular Surgery
Diabetic Peripheral Neuropathy Type 2
Concurrent Training vs Soleus Push-Ups on Neurogenesis-Related Biomarkers in Diabetic Neuropathy Patients
This study will examine how two types of exercise programs affect nerve health in people with diabetic peripheral neuropathy. Diabetic peripheral neuropathy is a common complication of type 2 diabetes that can cause numbness…
- Trial name
- NCT07675720
- Testing
- Concurrent Training, Soleus Push-ups
- Where
- Pakistan
- Volunteers
- 99 people
- Sponsor
- Riphah International University
Neuroendocrine Neoplasm, Neuroendocrine Neoplasm of Gastrointestinal Tract
Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients
Neuroendocrine neoplasms (NENs) are a heterogeneous group of lesions derived from cells with the ability to produce hormones that may arise from multiple different organs.
- Conditions
- Neuroendocrine Neoplasm, Neuroendocrine Neoplasm of Gastrointestinal Tract, Neuroendocrine Neoplasm of Lung, Thymic Neuroendocrine Neoplasm
- Trial name
- NCT06523582
- Where
- 3 sites in Mexico
- Volunteers
- 750 people, healthy volunteers welcome
- Sponsor
- Universidad Nacional Autonoma de Mexico
Death, Sudden, Cardiac, Ventricular Fibrillation
PREVENTION-ACHD Risk Score
Sudden cardiac death (SCD) is one of the major causes of mortality in adults with congenital heart disease (ACHD). Risk stratification for sudden cardiac death in this patient group is challenging and at the current moment there…
- Conditions
- Death, Sudden, Cardiac, Ventricular Fibrillation, Ventricular Tachycardia, Congenital Heart Disease
- Trial name
- PREVENTIONACHD (NCT03957824)
- Testing
- PREVENTION-ACHD risk score
- Volunteers
- 783 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Hypervascular Tumor and Arteriovenous Malformation
A Multicenter, Open-label Study for E7040 in Japanese Subjects With Hypervascular Tumor and Subjects With Arteriovenous Malformation
To evaluate the efficacy and safety of transcatheter arterial embolization with E7040 in Japanese subjects with hypervascular tumor or arteriovenous malformation
- Trial name
- NCT01677624
- Testing
- E7040
- Where
- 10 sites in Japan
- Volunteers
- 28 people
- Sponsor
- Eisai Co., Ltd.
Hemophilia A
Outcomes of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast
- Trial name
- NCT05279924
- Testing
- Prophylaxis with Emicizumab
- Where
- Côte d’Ivoire
- Volunteers
- 33 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Congenital Heart Disease (CHD)
Biventricular Pacing in Children With Congenital Heart Disease
Surgery with cardiopulmonary bypass (CPB) for congenital heart disease (CHD) causes low cardiac index (CI). With the increasing success of surgery for CHD, mortality has decreased and emphasis has shifted to post-operative…
- Trial name
- NCT02644824
- Testing
- Biventricular Pacing (BiVp)
- Where
- Canada
- Volunteers
- 43 people
- Sponsor
- The Hospital for Sick Children
Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy
An Integrative-"Omics" Study of Cardiomyopathy Patients for Diagnosis and Prognosis in China
This is a multi-omics research of Chinese cardiomyopathies patients, aiming to determine genetic risk factor and serial biomarkers of cardiomyopathies in diagnosis and prognosis.
- Conditions
- Dilated Cardiomyopathy, Hypertrophic Cardiomyopathy, Arrhythmogenic Right Ventricular Cardiomyopathy, Left Ventricular Non-compaction
- Trial name
- AOCC (NCT03076580)
- Where
- 3 sites in China
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Beijing Institute of Heart, Lung and Blood Vessel Diseases
Neonatal Diseases and Abnormalities, Neonatal Mortality
Melatonin for Pulmonary Hypertension in Full Term Neonates
Published evidence has provided a possible role of melatonin and the treatment of pulmonary hypertension through its strong antioxidant properties and improving vascular function in animals.
- Conditions
- Neonatal Diseases and Abnormalities, Neonatal Mortality, Pulmonary Arterial Hypertension, Pulmonary Arterial Hypertension, Children
- Trial name
- NCT06685679
- Testing
- Melatonin
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Ain Shams University
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Patent Ductus Arteriosus, Congenital Heart Disease
PDA for Kidneys Study
The goal of this observational study is to gather more information on kidney oxygen levels in babies with a patent ductus arteriosus (PDA), and evaluate the relationships between kidney oxygen levels, PDA status and kidney…
- Trial name
- PDA4K (NCT06658496)
- Testing
- Near Infrared Spectroscopy
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Wisconsin, Madison
Gaucher Disease
Nutritional Status of Gaucher Disease Patients
This cross-sectional study aims to evaluate the nutritional status of patients with Gaucher disease attending Assiut University Children's Hospital using anthropometric measurements and body composition analysis.
- Trial name
- NCT07845188
- Volunteers
- 84 people
- Sponsor
- Assiut University
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