Clinical trials: Neonates, active
Prion Disease
PrP-targeting siRNA Safety & Mechanism Study
The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients and in pre-symptomatic adults at genetic risk for prion disease.
- Trial name
- PRiSM (NCT07444580)
- Testing
- PrP-siRNA
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Broad Institute of MIT and Harvard
Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn
Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU
XPhePiccosMR is a prospective, single-arm, open label, observational study that aims to evaluate the acceptability and tolerability of XPhe Piccos, a slow-release, phenylalanine-free protein substitute in the form of tiny…
- Conditions
- Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn, Brain Disorder, Metabolic, Brain Diseases
- Trial name
- XPhePiccosMR (NCT07802366)
- Testing
- Slow release, phenylalanine-free protein substitute tablets
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- metaX Institut fuer Diatetik GmbH
Netherton Syndrome
Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome
To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome
- Trial name
- NCT05789056
- Testing
- QRX003, 4% Lotion
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- Quoin Pharmaceuticals
Premature, Feeding Disorders
Effect OF Lactase Enzyme Supplements ON Intolerance IN Preterm Neonates
This study aims to study the effect of lactase enzyme in preventing feeding intolerance in preterm neonates in neonatal intensive care unit at Alexandria University Children's Hospital.
- Conditions
- Premature, Feeding Disorders, Lactase Deficiency, Congenital
- Trial name
- NCT07088302
- Testing
- lactase administration
- Where
- Egypt
- Volunteers
- 120 people
- Sponsor
- Alexandria University
Cervical Fusion
Clinical and Radiological Outcomes of Posterior Cervical Fusion Supplemented With Interfacet Spacers
This is a prospective study to assess fusion rates and cervical sagittal parameters following posterior cervical arthrodesis procedures supplemented with CIS.
- Trial name
- NCT04041583
- Testing
- Cervical Interfacet Spacers
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Ohio State University
Tetralogy of Fallot, Cardiac Death, Sudden
Repaired Tetralogy of Fallot Italian Registry
The CMR/CT WG of the Italian pediatric cardiology society set up a multi-center observational clinical database of repaired-TOF evaluated.
- Conditions
- Tetralogy of Fallot, Cardiac Death, Sudden, Cardiac Arrhythmia, Cardiac Event
- Trial name
- RETE-Fallot (NCT05288894)
- Testing
- Cardiac magnetic resonance imaging
- Where
- Italy
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Azienda Ospedaliero, Universitaria Ospedali Riuniti
Sickle Cell Disease
Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease
In a hybrid type I effectiveness-implementation trial, our three-center research teams aim to examine whether empowering adults with sickle cell disease (SCD) with patient-facing SCD-specific guidelines through an mHealth…
- Trial name
- PF-Guide (NCT06919224)
- Testing
- mHealth app + booklets
- Where
- 4 sites in United States
- Volunteers
- 287 people
- Sponsor
- Ohio State University
NEC, Necrotizing Enterocolitis
Study of Normal Intestinal Development and Disease in Premature and Term Neonates
Current research on early intestinal development is primarily performed in mouse models. While useful in many other ways, mouse models are not ideal for studying human intestine development as the timing of this process differs…
- Conditions
- NEC, Necrotizing Enterocolitis, Intestinal Disease, Human Development
- Trial name
- NiiCE (NCT06681129)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Boston Children's Hospital
Developmental Dysplasia of the Hip
Intraoperative Contrast-Enhanced Ultrasound Evaluation of Blood Flow at the Time of Surgical Hip Reduction for DDH
This study evaluates the feasibility and utility of contrast-enhanced ultrasound to provide real-time assessment of blood flow to the femoral head in infants undergoing surgical reduction for developmental dysplasia of the hip.
- Trial name
- NCT03107520
- Testing
- Intraoperative contrast-enhanced ultrasound (CEUS), Lumason
- Where
- United States
- Volunteers
- 125 people
- Sponsor
- Children's Hospital of Philadelphia
Duchenne Muscular Dystrophy, Duchenne Disease
Vasodilator and Exercise Study for DMD (VASO-REx)
Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved…
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children
- Trial name
- NCT06290713
- Testing
- Tadalafil, Exercise Training
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Florida
Haemoglobinopathies
Observational Study for Patients With Hemoglobinopathies and Rare Inherited Anemia and Covid 19
The COVID-19 pandemic is causing many deaths around the world, putting a strain on health services. Patients with pre-existing chronic conditions are most affected by the SARS-COV2 infection.
- Trial name
- NCT04746066
- Where
- 2 sites in Italy
- Volunteers
- 10,000 people
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
Respiratory Distress Syndrome, Newborn, Infant, Premature
Measuring Diaphragm Electrical Activity in Neonates Using a Smaller Inter-electrode Distance
Neurally Adjusted Ventilatory Assist (NAVA) is a mode of ventilation where the electrical activity of the diaphragm (EDI) - a signal representing the baby's respiratory drive - is used to control the timing and amount of assist…
- Trial name
- NCT05427929
- Testing
- NVR 4mm Edi catheter
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Mount Sinai Hospital, Canada
Myotonic Dystrophy 1, Diabetes
Myotonic Dystrophy - Vascular and Cognition
The cognitive disorders of adult forms of myotonic dystrophies type 1 are heterogeneous (impairment of executive functions, visio construction and theory of the mind, which can progress to the stage of dementia).
- Conditions
- Myotonic Dystrophy 1, Diabetes, Carbohydrate Intolerance
- Trial name
- DM-VASCOG (NCT04656210)
- Testing
- MRI, neuropsychological tests
- Where
- France
- Volunteers
- 85 people
- Sponsor
- University Hospital, Lille
Corneal Edema, Corneal Endothelial Dystrophy
Study to Assess Safety and Tolerability of Multiple Doses of EO2002
The goal of this clinical study is to assess the safety of multiple intracameral injections of EO2002 with and without topical Ripasudil.
- Conditions
- Corneal Edema, Corneal Endothelial Dystrophy, Endothelial Dysfunction, Fuchs Dystrophy
- Trial name
- NCT05636579
- Testing
- EO2002, Ripasudil
- Where
- Mexico
- Volunteers
- 12 people
- Sponsor
- Asociación para Evitar la Ceguera en México
Class II Malocclusion, Mandibular Deficiency
Dentoskeletal Effects of 3D-Printed Fixed Twin Block Versus MARA Appliances in Management of Skeletal Class II
Objective: to evaluate the dentoskeletal effects of CAD/CAM 3D-Printed fixed twin block versus MARA appliances in the management of skeletal class II due to Mandibular Deficiency in Preadolescence Class II malocclusion is…
- Trial name
- NCT07340242
- Testing
- MARA appliance, fixed twin block
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Mansoura University
Recessive Dystrophic Epidermolysis Bullosa, Dysphagia
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
Budesonide is an anti-inflammatory medication that works by decreasing the immune system's overreaction. In the treatment of eosinophilic esophagitis, especially with Jorveza or a preparation made in a pharmacy, it reduces the…
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa, Dysphagia, Esophageal Stenosis
- Trial name
- EBUDE (NCT07684105)
- Testing
- The retrospective data collected will be entered into an eCRF and then analyzed centrally
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Necrotizing Enterocolitis (NEC)
NETs: Protection or Harm in Neonatal Inflammation or Infection
This is a prospective in vitro cell biology study of polymorphonuclear leukocyte (PMN) protein synthesis in response to PAF. PMNs from cord blood of premature human infants at risk for NEC (birth weight between 501 - 1500 grams)…
- Trial name
- NCT00747851
- Where
- United States
- Volunteers
- 388 people, healthy volunteers welcome
- Sponsor
- University of Utah
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell, Sickle Cell Disease
Impact of Regional Anesthesia on Inflammatory Mechanisms During Vaso-occlusive Crisis in Sickle Cell Patients
Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control.
- Conditions
- Sickle Cell, Sickle Cell Disease, Vaso-Occlusive Crises
- Trial name
- ALRCVO-MP (NCT07001189)
- Where
- Guadeloupe
- Volunteers
- 34 people
- Sponsor
- Centre Hospitalier Universitaire de la Guadeloupe
Bronchopulmonary Dysplasia (BPD), Premature Births
PK, Safety and Preliminary Efficacy Study of Montelukast in Critically Ill Infants With Developing Bronchopulmonary Dysplasia
The purpose of the study is to learn how safe montelukast may be in premature infants at significant risk for Bronchopulmonary Dysplasia (BPD) and to determine how much and how quickly montelukast moves from the stomach into the…
- Conditions
- Bronchopulmonary Dysplasia (BPD), Premature Births, Critical Illness
- Trial name
- PRISM (NCT07101640)
- Testing
- montelukast 4 mg granule
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Duke University
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