Clinical trials: Defects
Single-ventricle
Simple Non-invasive Breathing Device to Improve Pulmonary Flow Pusatility in Single Ventricle Post Fontan
This is a pilot study using a novel, minimal risk, portable, hands-free oral-positive pressure device (oPEP) in patients with Fontan palliation that will examine whether using this device in both the acute and chronic phase will…
- Trial name
- NCT05634226
- Testing
- Hands-free oral-positive pressure device (oPEP)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Tarek Alsaied
Neural Tube Defects, Anemia
Post T-plant Infusion of Allogeneic Cytokine Induced Killer (CIK) Cells as Consolidative Therapy in Myelodysplastic Syndromes/Myeloproliferative Disorders
Allogeneic stem cell transplantation (transplant of blood cells from another individual) is a treatment option for patients with myelodysplasia or myeloproliferative Disorders.
- Conditions
- Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure
- Trial name
- NCT01392989
- Testing
- CIK cells, Cyclosporine, Mycophenolate Mofetil
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Everett Meyer
Cerebral Palsy, Muscle Disorder
A New Training to Enhance Physical Activity in Adolescents With Cerebral Palsy
The purpose of this research study is to test the feasibility and response of a new exercise protocol on improving physical activity in adolescents with cerebral palsy.
- Conditions
- Cerebral Palsy, Muscle Disorder, Physical Disability, Posture Disorders in Children
- Trial name
- FOLIC (NCT05865418)
- Testing
- Loaded high intensity training, High intensity circuit training (HICT)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- University of Alabama at Birmingham
Congenital Heart Disease, Heart Failure
Spironolactone in Adult Congenital Heart Disease
The purpose of this study is to see if the study drug called spironolactone reduces fibrous (stiffening) in heart muscle tissue and improves heart function.
- Conditions
- Congenital Heart Disease, Heart Failure, Endomyocardial Fibrosis
- Trial name
- NCT01069510
- Testing
- Spironolactone 25mg
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Oregon Health and Science University
Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC].
- Conditions
- Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
- Trial name
- RISE (NCT04729751)
- Testing
- Maralixibat
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 27 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Gingival Recession, Localized
Effectiveness of Modified-free Gingival Graft for Treatment of Localized Gingival Recession Defects
The aim of the present study is to evaluate, whether use of the modified free gingival graft (mod-FGG) technique improves treatment outcomes after surgical root coverage at mandibular incisors with gingival recession defects.
- Trial name
- NCT04718545
- Testing
- Mucogingival surgery
- Where
- Sweden
- Volunteers
- 30 people
- Sponsor
- Göteborg University
Knee Injury
Evaluation of a Composite Cancellous and Demineralized Bone Plug (CR-Plug) for Repair of Knee Osteochondral Defects
The objective of this study is to determine how effective a plug made out of human bone (called "CR-Plug") will be in the treatment of smaller, focal defects (like the injury the patient has to their knee).
- Trial name
- NCT00984594
- Testing
- Primary injury site, Backfill site
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- RTI Surgical
Trauma-related Wound, Burns
StrataGraft® Skin Tissue as an Alternative to Autografting Full-thickness Complex Skin Defects
About 20 participants will be enrolled in this trial if they have had an accident that damages both the dermal (outside) and epidermal (inside) layers of skin on up to 49% of their body.
- Conditions
- Trauma-related Wound, Burns, Skin Wound
- Trial name
- NCT03005054
- Testing
- StrataGraft Skin Tissue, Autograft
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- Stratatech, a Mallinckrodt Company
Jervell and Lange Nielsen Syndrome
Diltiazem in Jervell and Lange-Nielsen Syndrome
This study will test the effect of diltiazem, a calcium channel blocking drug, on the QT interval in patients with Jervell and Lange-Nielsen syndrome.
- Trial name
- NCT06534671
- Testing
- diltiazem
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Vanderbilt University Medical Center
Congenital Heart Disease
Influence of Genetic Polymorphisms on Ventricular Structure and Function in Patients With Single Ventricle Anatomy
There are many kids born with congenital heart disease. Some of these defects may lead to the formation of a single ventricle (the heart having only one pumping chamber).
- Trial name
- NCT00165984
- Where
- United States
- Volunteers
- 224 people
- Sponsor
- Emory University
Alagille Syndrome (ALGS)
A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time.
- Trial name
- NCT05543174
- Testing
- TAK-625
- Where
- 8 sites in Japan
- Volunteers
- 7 people
- Sponsor
- Takeda
Patent Ductus Arteriosus, Preterm Infant
Randomized Controlled Trial to Evaluate the Safety and Efficacy of Acetaminophen in Preterm Infants Used in Combination With Ibuprofen for Closure of the Ductus Arteriosus
The purpose of the present study is to determine whether treatment of hemodynamically significant patent ductus arteriosus with a combined therapy of intravenous Ibuprofen and oral acetaminophen has higher success rate in closing…
- Conditions
- Patent Ductus Arteriosus, Preterm Infant, Bronchopulmonary Dysplasia
- Trial name
- NCT03701074
- Testing
- Ibuprofen and acetaminophen
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of South Alabama
Prematurity, Respiratory Distress Syndrome
Curosurf and Survanta Treatment(CAST)of RDS in Very Premature Infants
Approval of surfactant by the FDA in 1989 for the treatment of Respiratory Distress Syndrome (RDS) in premature infants greatly improved survival rates.
- Conditions
- Prematurity, Respiratory Distress Syndrome, Patent Ductus Arteriosus
- Trial name
- CAST (NCT00767039)
- Testing
- Survanta (beractant), Curosurf (poractant)
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Alan Fujii
Migraine Headaches, Patent Foramen Ovale
PREMIUM Migraine Trial
The Purpose of this study is to evaluate the impact of percutaneous closure of a patent foramen ovale (PFO) (a hole in the heart), using the AMPLATZER PFO Occluder, on the incidence of migraine headaches.
- Trial name
- NCT00355056
- Testing
- AMPLATZER PFO Occluder
- Where
- 30 sites in United States
- Volunteers
- 230 people
- Sponsor
- Abbott Medical Devices
Myelomeningocele, Pregnancy
Evaluation of Hysterotomy Site After Open Fetal Surgery
This study is to review how the uterus heals after having open fetal surgery. Open fetal surgery causes a scar perhaps two: one from the open fetal surgery and a second from delivery by cesarean section; rarely, the same area of…
- Trial name
- Hysterotomy (NCT02493062)
- Testing
- Sonohysterogram
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- St. Louis University
Coarctation of the Aorta
Coarctation Of the Aorta Stent Trial
The purpose of this study is to determine whether Cheatham Platinum bare metal stents are safe and effective in the treatment of native and recurrent aortic coarctation in selected children, adolescents and adults.
- Trial name
- COAST (NCT00552812)
- Testing
- Stent therapy of aortic coarctation
- Where
- United States
- Volunteers
- 105 people, healthy volunteers welcome
- Sponsor
- Richard E. Ringel
Hypoplastic Left Heart Syndrome, Total Anomalous Pulmonary Venous Return
Targeting Normoxia in Neonates With Cyanotic Congenital Heart Disease in the Intra-operative and Immediate Post-operative Period
This clinical trial is studying the use of different levels of oxygen exposure during and after cardiopulmonary bypass in eligible infants to learn about its safety during heart surgery.
- Conditions
- Hypoplastic Left Heart Syndrome, Total Anomalous Pulmonary Venous Return, Truncus Arteriosus, Pulmonary Atresia With Ventricular Septal Defect
- Trial name
- T-NOX (NCT04452188)
- Testing
- Normoxia (with controlled re-oxygenation), Standard of care ventilation
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- University of Michigan
Degeneration Articular Cartilage Knee
Evaluation of Safety and Exploratory Efficacy of CARTISTEM®, a Cell Therapy Product for Articular Cartilage Defects
The purpose of this study is to determine whether CARTISTEM, a cell therapeutic product, is safe and effective in the treatment of articular cartilage defects of the knee as a result of ageing, trauma, or degenerative diseases.
- Trial name
- NCT01733186
- Testing
- CARTISTEM®
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Medipost, Inc.
Alagille Syndrome
Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as…
- Trial name
- ICONIC (NCT02160782)
- Testing
- LUM001 (Maralixibat)
- Where
- 9 sites in Australia, Belgium and 4 more
- Volunteers
- 31 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Congenital Heart Disease, Single Ventricle
Improving Functional Capacity in Fontan Patients Through Inspiratory Muscle Training
Mechanisms that typically result in increased cardiac output, such as inotropic support, increased heart rate, and decreased afterload, have a blunted effect in Fontan circulation.
- Trial name
- NCT01977222
- Testing
- THRESHOLD(TM) INSPIRATORY MUSCLE TRAINER
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Boston Children's Hospital