Clinical trials: Fetal Developmental Abnormality
Respiratory Distress Syndrome (& [Hyaline Membrane Disease]), Respiratory Distress Syndrome (Neonatal)
Surfactant Administration Methods in Preterm Infants With RDS: A Swedish Cohort Study
The goal of this observational study is to evaluate different methods of surfactant administration in preterm infants with respiratory distress syndrome (RDS).
- Conditions
- Respiratory Distress Syndrome (& [Hyaline Membrane Disease]), Respiratory Distress Syndrome (Neonatal), Respiratory Distress Syndrome (RDS), Respiratory Distress Syndrome of Newborn
- Trial name
- NCT07550218
- Where
- Sweden
- Volunteers
- 300 people
- Sponsor
- Vastra Gotaland Region
Patent Ductus Arteriosus
Treatment of a PDA With Acetaminophen in Preterm Neonates: Exploring Various Indications
This study will evaluate the use of acetaminophen in preterm infants when a patent ductus arteriosus (PDA) is of concern. We will perform two simultaneous prospective observational studies over a 3 year period.
- Trial name
- NCT03289390
- Testing
- Acetaminophen
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Albany Medical College
Pulmonary Sequestration, Congenital Pulmonary Airway Malformation
Atomized Inhalation ICG for Treatment of Congenital Lung Malformations
To investigate the safety and effectiveness of preoperative aerosol inhalation ICG solution for thoracoscopic accurate resection of congenital lung malformations in children.
- Conditions
- Pulmonary Sequestration, Congenital Pulmonary Airway Malformation, Indocyanine Green, Thoracoscopic Surgery
- Trial name
- NCT06302985
- Testing
- atomized inhalation ICG before thoracoscope pneumonectomy
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Jiexiong Feng
Congenital Heart Disease
Effect of Local Warming for Arterial Catheterization in Pediatric Anesthesia
The increase in internal diameter (ID) and cross-sectional area (CSA) may facilitate better arterial catheterization. Since an increase in body temperature can cause peripheral vasodilation, we aimed to determine if local warming…
- Trial name
- WARM-PA (NCT06041685)
- Testing
- local warming
- Where
- South Korea
- Volunteers
- 126 people
- Sponsor
- Pusan National University Yangsan Hospital
X-linked Hypohidrotic Ectodermal Dysplasia
Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002
The goal of the ECP-002e extension study is to continue the evaluation of all EDI200-treated ECP-002 subjects up to age 10 yrs. No additional study drug administration is planned.
- Trial name
- ECP-002e (NCT01992289)
- Testing
- EDI200
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 10 people
- Sponsor
- Edimer Pharmaceuticals
Muscular Ventricular Septal Defect
Follow-up Study to Monitor the Efficacy and Safety of the Occlutech® mVSD Cases
The Occlutech® mVSD is indicated for percutaneous occlusion of hemodynamically or clinically significant muscular ventricular septal defects.
- Trial name
- mVSD Registry (NCT05329350)
- Testing
- Occlutech muscular ventricular septal defect (mVSD) Occluder
- Where
- 6 sites in Ireland, Turkey (Türkiye) and 1 more
- Volunteers
- 50 people
- Sponsor
- Occlutech International AB
Arthrogryposis Multiplex Congenita, Piezo2 Mutation Gain of Function
Piezo2-related Arthrogryposis & physiopathOLOgy 3
Study type: Observational, non-interventional, single-center, descriptive study. Goal of the study: The goal of this observational study is to characterize the intensity, variability, and qualitative features of pain in patients…
- Trial name
- PAOLO3 (NCT07360574)
- Where
- France
- Volunteers
- 12 people
- Sponsor
- University Hospital, Grenoble
Sickle Cell Disease, Renal Disease
Improving Scientific Rigor of Renal Clinical Endpoints for Sickle Cell Anemia
The investigators will attempt to develop a more accurate equation to estimate eGFR in pediatric and adult sickle cell patients
- Conditions
- Sickle Cell Disease, Renal Disease, Glomerular Disease
- Trial name
- NCT04380610
- Testing
- iohexol GFR
- Where
- 4 sites in United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.
Chronic Granulomatous Disease
Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease
This study will investigate the safety and effectiveness of a new stem cell transplant procedure for treating chronic granulomatous disease (CGD) in patients with active infection.
- Trial name
- NCT00006417
- Testing
- Stem cell transplantation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
G6PD Deficiency
Evaluation of a Diagnostic to Identify Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Brazil
Th objectives of this study are: To determine the performance of G6PD tests in detecting G6PD activity and hemoglobin (Hb) compared to a reference assay To assess the comprehension of the G6PD test packaging and labelling among…
- Trial name
- NCT04033640
- Testing
- SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System
- Where
- 2 sites in Brazil
- Volunteers
- 1,754 people, healthy volunteers welcome
- Sponsor
- PATH
Respiratory Distress Syndrome, Newborn
Fluid Restriction in Respiratory Distress of the Newborn
The object of this study is to determine the best way to care for infants with respiratory distress. The investigators are testing a method of feeding fluids to infants with respiratory distress in amounts similar to what healthy…
- Trial name
- NCT03120585
- Testing
- Fluid Management Intervention
- Where
- 2 sites in United States
- Volunteers
- 65 people
- Sponsor
- University of Oklahoma
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases.
- Conditions
- Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne, Muscular Dystrophy, Becker, Congenital Myopathy
- Trial name
- ARNseq-Musc (NCT06833489)
- Testing
- ARN extraction from muscle biopsies
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Down Syndrome
A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)
This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome.
- Trial name
- NCT02024789
- Testing
- RG1662
- Where
- 37 sites in United States, Argentina and 8 more
- Volunteers
- 173 people
- Sponsor
- Hoffmann-La Roche
Beta Thalassemia
Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram
Heart failure from myocardial iron deposition is a severe complication for patients with transfusion-dependent beta thalassemia .
- Trial name
- NCT04929574
- Testing
- Echocardiogram and Hematological Analysis
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Nanfang Hospital, Southern Medical University
MPS I
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing.
- Trial name
- NCT02702115
- Testing
- SB-318
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Sangamo Therapeutics
Cytomegalovirus Retinitis, HIV Infections
An Open Label Evaluation of the Safety and Pharmacokinetics of Ganciclovir in Children
To evaluate the pharmacokinetics of intravenous ganciclovir in children (ages 3 months - 12 years). To determine the safety and tolerance of a 2 to 3 week induction course of ganciclovir IV in immunocompromised children receiving…
- Trial name
- NCT00002015
- Testing
- Ganciclovir
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Roche Global Development
Von Willebrand Disease (VWD)
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in pediatric and adult participants…
- Trial name
- NCT03879135
- Testing
- rVWF, rFVIII
- Where
- 33 sites in United States, Austria and 7 more
- Volunteers
- 38 people
- Sponsor
- Baxalta now part of Shire
Hypoplastic Left Heart Syndrome, Single Right Ventricle
Cardiac Progenitor Cell Infusion to Treat Univentricular Heart Disease (PERSEUS)
The purpose of this study is to investigate the efficacy of intracoronary infusion of cardiac progenitor cells in patients with univentricular heart disease.
- Conditions
- Hypoplastic Left Heart Syndrome, Single Right Ventricle, Single Left Ventricle
- Trial name
- NCT01829750
- Testing
- Cardiac progenitor cell infusion
- Where
- Japan
- Volunteers
- 34 people
- Sponsor
- Okayama University
Growth Hormone Deficiency, Dwarfism
rhGH Therapy on Hepatic Drug Metabolism
The purpose of the study is to understand the effect of rhGH therapy on hepatic drug metabolism in children with idiopathic growth hormone deficiency.
- Trial name
- NCT00458991
- Testing
- Dextromethorphan and Caffeine
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- University of Louisville
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