Clinical trials: Newborn Diseases
Neurofibromatosis 1, Neurofibroma
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
This study is being conducted to determine if Whole Body MRI (WBMRI) can be used to identify Atypical Neurofibromas (ANF) in Neurofibromatosis Type 1 (NF1) patients with high tumor burden.
- Conditions
- Neurofibromatosis 1, Neurofibroma, Atypical Neurofibroma, Atypical Neurofibromatosis
- Trial name
- NCT03820778
- Testing
- Whole Body MRI
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's National Research Institute
Cystic Fibrosis, Adolescent Behavior
SAFETIM-needs : Exploring Adolescent's and Parent's Needs During Transition in French CF Centers
prospective multicentric study protocol in french CF center, exploring adolescent avec parents needs during transition from pediatric CF center to adult CF center
- Trial name
- SAFETIM-needs (NCT03298685)
- Testing
- semi structured interviews for all adolescents and their parent
- Where
- 4 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
Constipation - Functional, Abdominal Pain (AP)
Co-Development and Validation of Colonic Symptom Pictograms for Young People
Severe constipation affects 10% of children, often leading to significant physical and emotional distress. While constipation is commonly associated with impaired bowel movements, it is fundamentally linked to abnormalities in…
- Conditions
- Constipation - Functional, Abdominal Pain (AP), Nausea and Vomitting, Bloating
- Trial name
- NCT07337343
- Testing
- Pictogram comprehension and validation
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Auckland, New Zealand
Iliocaval Compression Syndrome
Role of IVUS in Management of Iliocaval Obstruction
To assess the role of IVUS in 1. Providing diagnostic important data as: luminal diameters, cross-sectional area, assessment of potential thrombus volume which enables optimal choice of appropriate angioplasty technique…
- Trial name
- NCT04024215
- Testing
- intravascular ultrasound
- Where
- Egypt
- Volunteers
- 70 people
- Sponsor
- Assiut University
Congenital Diaphragmatic Hernia, Acute Kidney Injury
Incidence of Acute Kidney Injury and Risk Factors in Newborns With Congenital Diaphragmatic Hernia
The main aim of this project is to elucidate the incidence of acute kidney injury (AKI) in newborns with congenital diaphragmatic hernia during stay in the Pediatric intensive care unit. (PICU).
- Conditions
- Congenital Diaphragmatic Hernia, Acute Kidney Injury, Multiple Organ Failure, Pulmonary Hypertension
- Trial name
- NCT06050525
- Where
- Sweden
- Volunteers
- 109 people
- Sponsor
- Karolinska Institutet
Retinitis Pigmentosa
Safety and Efficacy of the Alpha AMS Subretinal Implant
The aim of this single-centre study is to assess the safety and efficacy of the Retina Implant Alpha AMS (Retina Implant AG, Reutlingen, Germany) in participants with severe visual impairment secondary to outer retinal…
- Trial name
- OX-RI (NCT02720640)
- Testing
- Implant 'on' vs implant 'off'
- Where
- United Kingdom
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Mitral Valve Insufficiency, Heart Failure
Effectiveness of Surgical Mitral Valve Repair Versus Medical Treatment for People With Significant Mitral Regurgitation and Non-ischemic Congestive Heart Failure
Mitral regurgitation (MR), also known as mitral insufficiency, is a condition in which the heart's mitral valve, located between two of the heart's main chambers, does not firmly shut, allowing blood to leak backwards within the…
- Trial name
- SMMART-HF (NCT00608140)
- Testing
- Surgical mitral valvuloplasty with placement of annular ring (SMVR), Optimal medical therapy (OMT)
- Where
- 9 sites in United States, Canada
- Volunteers
- 2 people
- Sponsor
- Duke University
Lennox-Gastaut Syndrome, Dravet Syndrome
The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy
The investigators propose to study the effects of cannabidiol (CBD) on cardiac electrical function and the autonomic nervous system in children with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), when the CBD is…
- Trial name
- CBD1 (NCT02815540)
- Testing
- 12-Lead ECG, Cannabidiol
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Gillette Children's Specialty Healthcare
Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia
Sorafenib in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia
This phase I trial is studying the side effects and best dose of sorafenib in treating patients with relapsed or refractory acute myeloid leukemia, acute lymphoblastic leukemia, or chronic myelogenous leukemia.
- Conditions
- Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
- Trial name
- NCT00131989
- Testing
- sorafenib tosylate, pharmacological study, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- National Cancer Institute (NCI)
Usher Syndromes
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.
- Trial name
- SLO RP (NCT04355689)
- Testing
- NPI-001
- Where
- 4 sites in Australia
- Volunteers
- 49 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Polycystic Kidney Diseases
Pioglitazone and Lumbar Bone Marrow Fat in Chronic Kidney Disease
Chronic Kidney Disease (CKD) is associated with bone changes and very high fracture rates. A component of bone is marrow. Bone marrow fat is increased in patients with CKD compared to those in the normal population of the same…
- Trial name
- NCT02739750
- Testing
- No intervention
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Indiana University
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Arrhythmogenic Cardiomyopathy, PKP2-ACM
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Trial name
- SNAPSHOT- PKP2 (NCT06976606)
- Where
- 6 sites in United States
- Volunteers
- 40 people
- Sponsor
- Lexeo Therapeutics
Adult Rhabdomyosarcoma, Lung Metastases
Intensity-Modulated Radiation Therapy in Treating Younger Patients With Lung Metastases
This pilot clinical trial studies intensity-modulated radiation therapy (IMRT) in treating younger patients with lung metastases.
- Conditions
- Adult Rhabdomyosarcoma, Lung Metastases, Metastatic Ewing Sarcoma, Previously Treated Childhood Rhabdomyosarcoma
- Trial name
- NCT01586104
- Testing
- intensity-modulated radiation therapy
- Where
- 7 sites in United States
- Volunteers
- 20 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
POX Range of Healthy Newborns at Different Altitudes, POX Threshold for Screening Major CHD in Neonates
POX Range and the Threshold for Screening Major CHD in Neonates at Different Altitudes
major CHD (Congenital Heart Disease) screening by POX (pulse oximetry) in sea-level areas is not fit for low, medium and high altitude areas, but the normal range of pulse oximetry at different altitude areas has not been studied…
- Trial name
- NCT04238104
- Testing
- no intervention
- Where
- 8 sites in China
- Volunteers
- 2,200 people
- Sponsor
- Children's Hospital of Fudan University
Out-Of-Hospital Cardiac Arrest, Myocardial Infarction, Acute
Benefit of Hypothermia in OHCA Complicating AMI
To determine the clinical effectiveness of hypothermia treatment in patients with out-of-hospital cardiac arrest complicating acute myocardial infarction.
- Conditions
- Out-Of-Hospital Cardiac Arrest, Myocardial Infarction, Acute, Hypothermia Neonatal
- Trial name
- Hypothermia (NCT06141252)
- Testing
- Hypothermia
- Where
- South Korea
- Volunteers
- 2,925 people
- Sponsor
- Yonsei University
Von Willebrand Disease
Collection of Blood in Normal Subjects and Subjects With Von Willebrand Disease (VWD)
Von Willebrand disease is an inherited bleeding disorder that impacts the blood's ability to clot properly. Von Willebrand disease is cause by the lack or not working substance in the blood known as Von Willebrand factor.
- Trial name
- NCT00630448
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Generalized Lipodystrophy
Efficacy and Safety of Mibavademab in Adult and Pediatric Patients With Generalized Lipodystrophy
This study is researching a new drug called mibavademab (called "study drug"). The study involves participants with a condition called Generalized Lipodystrophy (GLD).
- Trial name
- LAGO (NCT07220785)
- Testing
- Mibavademab
- Where
- 9 sites in United States, Brazil and 1 more
- Volunteers
- 28 people
- Sponsor
- Regeneron Pharmaceuticals
Choroideremia
A Study to Assess Choroideremia (CHM) Health Outcomes
The primary objectives of this study are to describe disease progression and severity by age in participants with CHM, to assess health-related quality-of-life, resource utilization and work productivity, and to assess…
- Trial name
- NCT04750785
- Testing
- No Intervention
- Where
- 8 sites in United States
- Volunteers
- 46 people
- Sponsor
- Biogen
Premature Birth
NIRS Levels of Infants in Neonatal Intensive Care
Aim: This study was conducted to determine the effect of nursing care practices on the Near Infrared Spectroscopy (NIRS) levels of premature infants in the neonatal intensive care unit (NICU).
- Trial name
- NCT05246150
- Testing
- nursing care practices
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Eskisehir Osmangazi University
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