Clinical trials: Cardiac Congenital Defects
Duchenne Muscular Dystrophy
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
This single center open-label pilot study will enroll 15 non-ambulatory children with Duchenne muscular dystrophy at least 8 years of age and who demonstrate pre-clinical cardiomyopathy (defined as a cardiac ejection fraction…
- Trial name
- NCT02964377
- Testing
- (+)- Epicatechin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Craig McDonald, MD
Patent Ductus Arteriosus, Prematurity
Early Versus Late Use of Ibuprofen for Patent Ductus Arteriosus (PDA) Closure
The primary objective is to evaluate the Patent Ductus Arteriosus (PDA) closure rate of early vs. late use of Ibuprofen (Ibu). The investigators believe that early use of Ibu will have a higher PDA closure rate than later use of…
- Trial name
- NCT00833365
- Testing
- Ibuprofen
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Utah
Heart Disease
Sildenafil in Single Ventricle Patients
Patients with single ventricle anatomy undergo staged surgical palliation. The result is an "in series" circulation with pulmonary blood flow and cardiac output directly related to pulmonary vascular resistance.
- Trial name
- NCT01169519
- Testing
- Sildenafil by injection
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Duke University
Cardiac Transplant, Patent Ductus Arterious
The Pharmacology and Hemodynamics of Dexmedetomidine in Children With Congenital Heart Disease
The purpose of this study is to examine the pharmacokinetics, pharmacodynamics, and pharmacogenomics of dexmedetomidine in the following three pediatric patient populations: patients with bi-directional cavopulmonary anastomosis…
- Conditions
- Cardiac Transplant, Patent Ductus Arterious, Atrial Septal Defect, Bidirectional Cavopulmonary Anastomosis
- Trial name
- NCT00480740
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Children's National Research Institute
Fabry Disease
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry…
- Trial name
- NCT00140621
- Testing
- Agalsidase beta
- Where
- 7 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Genzyme, a Sanofi Company
Emphysema, Chronic Obstructive Pulmonary Disease (COPD)
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability
The purpose of this research study is to learn about the safety of transplanting lungs obtained from non-heart-beating donors (NHBDs) that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and…
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis
- Trial name
- NCT01615484
- Testing
- Transplantation of lungs obtained from Non-Heart-Beating Donors (NHBDs) after ex-vivo perfusion w/ STEEN Solution™, STEEN Solution™
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- University of North Carolina, Chapel Hill
Congenital Heart Disease in Children, Inflammatory Response
STeroids to REduce Systemic Inflammation After Infant Heart Surgery
This study's objective is to determine the pharmacokinetics (PK)/pharmacodynamics (PD), safety and efficacy of methylprednisolone in infants undergoing heart surgery with cardiopulmonary bypass.
- Trial name
- STRESS (NCT03229538)
- Testing
- Methylprednisolone, Isotonic saline
- Where
- 23 sites in United States
- Volunteers
- 1,263 people
- Sponsor
- Kevin Hill
Tetralogy of Fallot
Pediatric REPlAcement of the PulmonaRy ValvE in Tetralogy of Fallot -
Tetralogy of Fallot (TOF) is the most common cyanotic congenital heart defect with the vast majority of survivors of corrective surgery left with some degree of right ventricular (RV) volume overload due to pulmonary…
- Trial name
- TOFandPVR (NCT03634072)
- Testing
- PVR
- Where
- 6 sites in United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital of Philadelphia
Congenital Heart Surgery, Cardiopulmonary Bypass
Use of Phenoxybenzamine [PBZ] IV to Assist High Flow Low Pressure Perfusion [HFLPP] on Cardio-pulmonary Bypass
Cardiopulmonary bypass [CPB] in small size bodies can result in decreased peripheral perfusion. This results in anaerobic metabolism as evidenced by lactic acidosis.
- Trial name
- NCT00620945
- Testing
- Phenoxybenzamine
- Volunteers
- 87 people
- Sponsor
- The Cleveland Clinic
Plastic Bronchitis, Protein-Losing Enteropathies
Inhaled Tissue Plasminogen Activator for Acute Plastic Bronchitis
Plastic bronchitis (PB) is a rare, most often pediatric disease characterized by the formation of obstructive airway casts primarily composed of fibrin.
- Conditions
- Plastic Bronchitis, Protein-Losing Enteropathies, Healthy
- Trial name
- PLATyPuS (NCT02315898)
- Testing
- Treatment-inhaled tPA
- Where
- 6 sites in United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Congenital Heart Disease
A Study to Evaluate MEDI-524 In Children With Hemodynamically Significant Congenital Heart Disease
The primary goal was to describe the safety of the investigational product when given monthly to prevent serious respiratory infection among children with significant heart disease.
- Trial name
- NCT00538785
- Testing
- Motavizumab, Palivizumab
- Where
- 152 sites in United States, Austria and 15 more
- Volunteers
- 1,236 people
- Sponsor
- MedImmune LLC
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents
This study is conducted in Europe. The aim of this study is to compare the easiness of use of Norditropin NordiFlex® device to the device previously used by patients or parents.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- NCT01245374
- Testing
- Norditropin NordiFlex®
- Where
- France
- Volunteers
- 103 people
- Sponsor
- Novo Nordisk A/S
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Primary Biliary Cholangitis, Cardiovascular Abnormalities
Early Identification of Myocardial Impairment in PBC
Primary biliary cholangitis (PBC) is a chronic inflammatory liver disease leading to cirrhosis. Researches reported patients with PBC may involve abnormalities on skeleton, thyroid and exocrine glands.
- Trial name
- EARLY-MYO-PBC (NCT03545672)
- Testing
- CMR examination
- Where
- China
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- RenJi Hospital
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Congenital Heart Disease
Washed Versus Standard Blood Cell Transfusions in Pediatric Open Heart Surgery
Background: Children having open heart surgery to repair congenital heart defects demonstrate a large inflammatory response to the heart-lung machine and to surgery itself.
- Trial name
- NCT00693498
- Testing
- Standard leukoreduced irradiated blood cell transfusion, Washed leukoreduced irradiated blood cell transfusions
- Volunteers
- 162 people
- Sponsor
- University of Rochester
Turner's Syndrome
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years.
- Trial name
- NCT01066052
- Testing
- r-hGH
- Where
- France
- Volunteers
- 115 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Heart Septal Defects, Ventricular
Closure of Muscular Ventricular Septal Defects With The AMPLATZER™ Muscular VSD Occluder
The objective is to investigate the safety of the AMPLATZER Muscular VSD Occluder for the treatment of muscular ventricular septal defects, which are hemodynamically significant and are either isolated or in conjunction with…
- Trial name
- NCT00583791
- Testing
- Device closure with the AMPLATZER Muscular VSD Occluder
- Where
- 11 sites in United States
- Volunteers
- 91 people
- Sponsor
- Abbott Medical Devices
Dilated Cardiomyopathy, Heart Failure
PUFAs and Left Ventricular Function in Heart Failure
The purpose of this study is to test the hypothesis that n-3 PUFAs improve left ventricular systolic function in patients with stable chronic HF secondary to nonischemic dilated cardiomyopathy (NICM).
- Trial name
- CS-PUFA-02 (NCT01223703)
- Testing
- n-3 PUFAs
- Where
- Italy
- Volunteers
- 133 people
- Sponsor
- Università degli Studi di Brescia
Anesthesia; Adverse Effect, Congenital Heart Disease
The Effect of Anesthesia on Neurodevelopmental Outcome (NDO)
The purpose of this study is to assess whether the type of anesthesia, narcotic-based versus inhalational anesthesia administered during cardiopulmonary bypass (CPB) surgery contributes to the wide variation in neurologic…
- Conditions
- Anesthesia; Adverse Effect, Congenital Heart Disease, Neurodevelopmental Disorders
- Trial name
- NDO (NCT03882788)
- Testing
- Isoflurane, Fentanyl (high dose), Fentanyl (low dose)
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- Stanford University