Clinical trials: Defects
Hypoplastic Left Heart Syndrome
Prophylactic Peritoneal Dialysis Decreases Time to Achieve a Negative Fluid Balance After the Norwood Procedure
Babies born with hypoplastic left heart syndrome (HLHS) have three separate, complex heart surgeries before they turn three years of age. The first surgery typically happens in the first two weeks of life.
- Trial name
- NCT01215240
- Testing
- Peritoneal dialysis
- Where
- Canada
- Volunteers
- 22 people
- Sponsor
- University of Alberta
Bicuspid Aortic Valve, Thoracic Aortic Aneurysm
The Effect of Losartan in Bicuspid Aortic Valve Patients
The specific aims of this study are to: Establish baseline levels of circulating MMP-2 and -9 , TIMP-1and- 2 and TGFB levels in individuals with bicuspid aortic valve and ascending aortic or aortic sinus measurements >40mm.
- Trial name
- NCT01390181
- Testing
- Cozaar
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Circulatory Perfusion Disorder, Congenital Heart Disease
Early Use of Vasopressin in Post-Fontan Management
This is an investigator initiated, prospective, single-center, double-blinded, randomized, placebo-controlled trial of post-operative low dose vasopressin infusions as an early treatment of low systemic perfusion in pediatric…
- Conditions
- Circulatory Perfusion Disorder, Congenital Heart Disease, Single-ventricle
- Trial name
- VAMP (NCT03088345)
- Testing
- Vasopressin, Arginine
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Medical College of Wisconsin
Alagille Syndrome
An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome
This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol.
- Trial name
- IMAGINE-II (NCT02117713)
- Testing
- LUM001 (Maralixibat)
- Where
- 11 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Bardet-Biedl Syndrome, POMC Deficiency
Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway
A trial to compare the weekly and daily formulations of setmelanotide in participants with genetic defects in the melanocortin-4 receptor pathway.
- Trial name
- NCT05194124
- Testing
- Setmelanotide 2 mg, Setmelanotide 2.5 mg, Setmelanotide 3 mg
- Where
- 7 sites in United States, Canada and 4 more
- Volunteers
- 19 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Acute Kidney Injury, Hypoxia-Ischemia, Brain
Tissue Oxygenation During Treatment of Infant Congenital Heart Defects
Background: Acute kidney injury (AKI) is a common and serious postoperative complication in children with congenital heart disease.
- Conditions
- Acute Kidney Injury, Hypoxia-Ischemia, Brain, Mesenteric Ischemia
- Trial name
- NCT03941015
- Where
- China
- Volunteers
- 245 people
- Sponsor
- Beijing Anzhen Hospital
Dysfunctional RVOT Conduits in Patients With Congenital Heart Defects
Melody PB1016 Surveillance Study
Non-randomized, multi-center, prospective surveillance study to investigate and assess the residual risks, and to confirm the currently established safety and performance of the Melody TPV PB1016.
- Trial name
- NCT02347189
- Testing
- Melody Transcatheter Pulmonary Valve PB1016
- Where
- 8 sites in United States, Austria and 2 more
- Volunteers
- 39 people
- Sponsor
- Medtronic Heart Valves
ATM Gene Mutation, BRCA1 Gene Mutation
Niraparib Before Surgery in Treating Patients With High Risk Localized Prostate Cancer and DNA Damage Response Defects
This phase II trial studies how well niraparib, when given before surgery, works in treating patients with high risk prostate cancer that has not spread to other parts of the body (localized) and alterations in deoxyribonucleic…
- Conditions
- ATM Gene Mutation, BRCA1 Gene Mutation, BRCA2 Gene Mutation, BRIP1 Gene Mutation
- Trial name
- NCT04030559
- Testing
- Niraparib, Niraparib Tosylate Monohydrate, Radical Prostatectomy
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of California, Davis
Autism Spectrum Disorder, Cerebral Palsy
READYorNot[TM] Brain-Based Disabilities Trial
The purpose of this study is to find out if there is a benefit to using the MyREADY Transition[TM] BBD App for brain-based disabilities, compared to not using it.
- Conditions
- Autism Spectrum Disorder, Cerebral Palsy, Epilepsy, Fetal Alcohol Spectrum Disorders
- Trial name
- NCT03852550
- Testing
- e-health application
- Where
- 9 sites in Canada
- Volunteers
- 52 people
- Sponsor
- McMaster University
Congenital Disorder
Pacemaker Therapy in Adults With Congenital Heart Defects
Review the feasibility, safety and outcomes in adults with congenital heart disease who undergo pacemaker implantation for bradycardia, tachycardia or heart failure indications
- Trial name
- NCT00303511
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Long QT Syndrome
Pharmacokinetics of Eleclazine in Adults With Normal and Impaired Hepatic Function
The primary objective of this study is to evaluate the pharmacokinetic (PK) profile of oral eleclazine and its metabolite, GS-623134, in participants with normal and impaired hepatic function.
- Trial name
- NCT02412098
- Testing
- Eleclazine
- Where
- 7 sites in United States, Germany and 2 more
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Congenital Heart Defects
Is Levosimendan Superior to Milrinone Regarding Acute Kidney Injury After Cardiac Surgery for Congenital Heart Disease?
The aim of the study is to assess the ability of Levosimendan to reduce the postoperative acute kidney injury in pediatric patients undergoing surgery for congenital heart disease (CHDs).
- Trial name
- MiLe-1 (NCT02232399)
- Testing
- Levosimendan, Milrinone
- Where
- 2 sites in Finland, Sweden
- Volunteers
- 72 people
- Sponsor
- Göteborg University
Hypoplastic Left Heart Syndrome, Growth Failure
Comparison of Feeding Strategies for Hypoplastic Left Heart Syndrome Infants
The purpose of the study is to determine if a continuous feeding regimen as compared to an intermittent bolus feeding regimen leads to improved weight gain in infants with hypoplastic left heart syndrome (HLHS) after stage 1…
- Trial name
- NCT02657629
- Testing
- Continuous Feeding Regimen, Intermittent Bolus Feeding Regimen
- Volunteers
- 28 people
- Sponsor
- Columbia University
Segmental Long Bone Defects
Comparative Efficacy of the Masquelet Versus Titanium Mesh Cage Techniques for the Treatment of Large Long Bone Defects
The United States Department of Defense (DoD) is funding exciting new research at the University of Texas Medical Branch (UTMB) Department of Orthopaedic Surgery and Rehabilitation that can be a major improvement in the treatment…
- Trial name
- NCT02015390
- Testing
- Autogenous RIA bone grafting, Allogeneic bone grafting
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Persistent Corneal Epithelial Defect
A Study of the KIO-201 (Ocular Bandage Gel) for Improving Persistent Corneal Epithelial Defects
The objective of this study is to evaluate the safety and effectiveness of topical KIO-201 in patients with persistent corneal epithelial defects (PED). KIO-201 will be administered six (6) times per day while awake for 4 weeks.
- Trial name
- NCT05436288
- Testing
- KIO-201, a Crosslinked Thiolated Carboxymethyl Hyaluronic Acid 0.75% (CMHA-S)
- Where
- Mexico
- Volunteers
- 10 people
- Sponsor
- Kiora Pharmaceuticals, Inc.
Long QT Syndrome Type 3
Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Adults With Long QT Syndrome Type 3
The primary objective of this study is to evaluate the effect of oral eleclazine on mean daytime QTcF interval after 24 weeks of treatment with elecalzine in participants with long QT syndrome Type 3.
- Trial name
- NCT02300558
- Testing
- Eleclazine
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 41 people
- Sponsor
- Gilead Sciences
Periodontitis
Clinical and Radiographic Evaluation of Demineralized Freeze-Dried Bone Allograft Versus Platelet Rich Fibrin for the Treatment of Periodontal Intrabony Defects in Humans
Inflammatory periodontal disease often results in loss of bone around the teeth. Bone defects that have a certain size and shape (called intrabony or intraosseous defects) may be improved by using a graft material in the bony…
- Trial name
- NCT02154906
- Testing
- DFDBA, autologous platelet rich fibrin
- Where
- United States
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
22Q11.2 Deletion Syndrome
Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)
To evaluate the safety and tolerability of ZYN002 administered as a transdermal gel formulation, for up to 38 weeks, in participants ages 4 to <18 years, in the treatment of 22q.11.2 Deletion syndrome (22qDS).
- Trial name
- INSPIRE (NCT05149898)
- Testing
- ZYN002
- Where
- 3 sites in United States, Australia
- Volunteers
- 20 people
- Sponsor
- Harmony Biosciences Management, Inc.
Meningomyelocele, Spinal Dysraphism
Management of Myelomeningocele Study (MOMS)
Spina bifida (myelomeningocele) is a complex birth defect in which a portion of the spinal cord is not fully developed. The overlying bones and skin are incompletely formed and the underdeveloped area of the spinal cord is…
- Trial name
- MOMS (NCT00060606)
- Testing
- Prenatal Myelomeningocele Repair Surgery, Postnatal Myelomeningocele Repair Surgery
- Where
- 3 sites in United States
- Volunteers
- 183 people, healthy volunteers welcome
- Sponsor
- The George Washington University Biostatistics Center
Heart Defects, Congenital, Heart Failure, Congestive
Trial of Angiotensin Converting Enzyme Inhibition in Infants With Single Ventricle--Pediatric Heart Network
This study will evaluate the efficacy and safety of administering an angiotensin converting enzyme inhibitor (ACE-I) (enalapril) to infants with a functional single ventricle.
- Trial name
- ISV (NCT00113087)
- Testing
- Enalapril
- Where
- 9 sites in United States, Canada
- Volunteers
- 230 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)