Clinical trials: Newborn Diseases
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Tacrolimus and Mycophenolate Mofetil in Preventing Graft-Versus-Host Disease in Patients Who Have Undergone Total-Body Irradiation With or Without Fludarabine Phosphate Followed by Donor Peripheral Blood Stem Cell Transplant for Hematologic Cancer
This phase II trial studies how well tacrolimus and mycophenolate mofetil works in preventing graft-versus-host disease in patients who have undergone total-body irradiation (TBI) with or without fludarabine phosphate followed by…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00089011
- Testing
- fludarabine phosphate, total-body irradiation, mycophenolate mofetil
- Where
- 5 sites in United States, Italy
- Volunteers
- 150 people
- Sponsor
- Fred Hutchinson Cancer Center
Dilated Cardiomyopathy, Arrhythmogenic Cardiomyopathy
Image-Based Prediction of Ventricular Tachycardia Events in Non-ischemic Cardiomyopathy
Risk stratification for sudden cardiac death (SCD) in patients with non-ischemic cardiomyopathy (NICM) remains suboptimal. Although current guidelines rely on severe left ventricular systolic dysfunction (left ventricular…
- Conditions
- Dilated Cardiomyopathy, Arrhythmogenic Cardiomyopathy, Ventricular Arrhythmia, Hypertrophic Cardiomyopathies
- Trial name
- IMPROVE-NICM (NCT06730464)
- Where
- 4 sites in Italy, Portugal and 1 more
- Volunteers
- 500 people
- Sponsor
- Centro Medico Teknon
Gout
Food Effect Study of Febuxostat XR in Healthy Participants
The purpose of this study is to assess the effect of food on the bioavailability of febuxostat after a single oral dose of 80 mg febuxostat XR and to evaluate the pharmacokinetics (PK) of febuxostat after single oral doses of 40…
- Trial name
- NCT02374164
- Testing
- Febuxostat XR
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Takeda
Angelman Syndrome
Angelman Natural History Study - FAST Spain
The goal of this study is to conduct a prospective, longitudinal study to observe the natural clinical progression and disease outcome of AS patients receiving no disease-modified intervention, with the purpose of obtaining data…
- Trial name
- NCT06115109
- Testing
- Non intervention, Observational study
- Where
- 2 sites in Spain
- Volunteers
- 40 people
- Sponsor
- Puerta de Hierro University Hospital
Congenital Diaphragmatic Hernia
Gene Mutations and Rescue in Human Congenital Diaphragmatic Hernia
Congenital diaphragmatic hernia (CDH) occurs when the diaphragm, the muscle that separates the chest cavity from the abdominal cavity, does not form properly.
- Trial name
- NCT01098929
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Brugada Syndrome (BrS), Ventricular Arrhythmia
Prospective Multicentre Observational Registry of Peri-procedural Anaesthesia, Sedation and Related Medication Exposure in Patients With Brugada Syndrome
The goal of this observational study is to evaluate the perioperative risk of malignant ventricular arrhythmias in adult patients diagnosed with Brugada Syndrome undergoing anaesthetic procedures.
- Conditions
- Brugada Syndrome (BrS), Ventricular Arrhythmia, Anesthesia, Sedation
- Trial name
- PROSP_BRUGANAE (NCT07655102)
- Where
- Spain
- Volunteers
- 200 people
- Sponsor
- Hospital Clinic of Barcelona
Infant, Premature, Erythropoietin
High-Dose Erythropoietin in Extremely Premature Infants to Prevent/Attenuate Brain Injury: A Phase II Study
The highest risk for perinatal brain injury occurs among extremely premature infants who weigh less than 1250 grams at birth. Such perinatal brain injury is currently irreversible, associated with neurodevelopmental disability…
- Conditions
- Infant, Premature, Erythropoietin, Brain Injury, Intraventricular Hemorrhage
- Trial name
- NCT00589953
- Testing
- Erythropoietin
- Where
- United States
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Atlantic Health System
Human Immunodeficiency Virus, HIV
Nevirapine Dosing in Neonates for Prophylaxis of Mother-to-Child-Transmission (MTCT) of HIV Infection
The purpose of this study is to determine whether the current dose of nevirapine recommended in the Ontario Ministry of Health vertical transmission prevention protocol achieves therapeutic drug levels in newborn infants at high…
- Conditions
- Human Immunodeficiency Virus, HIV, Vertical Infection Transmission
- Trial name
- NCT02166502
- Where
- 2 sites in Canada
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- The Hospital for Sick Children
Fragile X Syndrome
Study of SPG601 in Adult Men With Fragile X Syndrome
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
- Trial name
- NCT06413537
- Testing
- SPG601
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Spinogenix
Hemophilia A
Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives.
- Trial name
- NCT03603275
- Testing
- New FVIII products
- Where
- 27 sites in United States
- Volunteers
- 3 people
- Sponsor
- Bayer
Obesity and Obesity-related Medical Conditions, Diabetes
Routine Validation and Reproducibility Testing of Laboratory Assays and Research Techniques Used for Endocrine, Cardiometabolic, and Musculoskeletal Disorder Research (VALD)
The purpose of this research study is to validate (check the accuracy of) laboratory assays, intravenous catheter insertion, and equipment or devices and their reproducibility, which is necessary to perform high quality research…
- Conditions
- Obesity and Obesity-related Medical Conditions, Diabetes, Atherosclerotic Disease, Heart Failure
- Trial name
- VALD (NCT07083557)
- Testing
- Endothelial cell collection
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Bettina Mittendorfer
Cystic Fibrosis
A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation
- Trial name
- NCT02742519
- Testing
- ivacaftor
- Where
- 6 sites in Australia, Canada and 1 more
- Volunteers
- 14 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Deglutition Disorders in Children, Congenital Heart Disease
Speech Therapy Online Approach for Children With Congenital Heart Desease in Pre and Post Surgical Period
The goal of this clinical trial is to test the results of a protocoled speech therapy online intervention in children with congenital heart disease aged between 0 and 12 months old, who feeds tube only.
- Trial name
- NCT07481565
- Testing
- Oral motor intervention
- Where
- 2 sites in Brazil
- Volunteers
- 20 people
- Sponsor
- Instituto de Cardiologia do Rio Grande do Sul
Pectus Excavatum, Pectus Carinatum
Impact of Surgical Treatments of Thoracic Deformation on Cardiopulmonary Functions
Pectus excavatum or carinatum are the most common congenital deformations of the ventral thoracic wall. Several different surgical methods with different techniques to correct these deformations have been described.
- Trial name
- NCT02163265
- Testing
- Surgery
- Where
- Austria
- Volunteers
- 34 people
- Sponsor
- Medical University Innsbruck
Herpes Simplex Virus
A Safety and Dose-Determining Study of CMX001 In Infants With Neonatal Herpes Simplex Virus (HSV) Infection Involving the Central Nervous System (CNS Disease)
This study is to identify if a Novel Antiviral Drug could be used to treat babies with Herpes Simplex Virus (HSV) with central nervous system (CNS) disease.
- Trial name
- NCT01610765
- Testing
- Novel Antiviral Drug
- Where
- 18 sites in United States
- Sponsor
- University of Alabama at Birmingham
Pompe Disease
Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease
A recombinant AAV vector has been generated to carry the codon-optimized acid alpha-glucosidase (coGAA) gene expressed from a human desmin enhancer/promoter (DES).
- Trial name
- AAV9-GAA_IM (NCT02240407)
- Testing
- Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase, Rapamycin, saline
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Florida
Marfan Syndrome, Ectopia Lentis
Classifying Ectopia Lentis in Marfan Syndrome Into Five Grades of Increasing Severity
Marfan syndrome is characterized by musculoskeletal manifestations, cardiovascular disease and ocular abnormalities, particularly ectopia lentis.
- Trial name
- NCT04319107
- Testing
- ophthalmological examinations using a slit lamp bio microscopy and a three-mirror lens, standard procedure.
- Where
- France
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Thalassemia, Splenectomy; Status
Zinc Supplementation on Cellular Immunity in Thalassemia Major
Randomized controlled trial was conducted in post-splenectomy patients aged >12 years. Subjects are randomly assigned to two groups (zinc and placebo). 1.5 mg/kg/day (max 50 mg/day) of Zinc is administered.
- Conditions
- Thalassemia, Splenectomy; Status, Immunosuppression
- Trial name
- NCT03117192
- Testing
- Zinc Sulfate, Sucrose Syrup
- Where
- Indonesia
- Volunteers
- 58 people
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
End Stage Renal Disease, AV Fistula
Percutaneous Less Invasive AV Fistula for Vascular Access in ESRD
A prospective single-arm well-controlled study to evaluate the safety and effectiveness of a less invasive means of establishing vascular access to facilitate dialysis in patients with end stage renal disease.
- Conditions
- End Stage Renal Disease, AV Fistula, Fistula, End Stage Kidney Disease
- Trial name
- NCT02363972
- Testing
- Ellipsys Vascular Access Catheter
- Where
- 5 sites in United States
- Volunteers
- 103 people
- Sponsor
- Medtronic Endovascular
Congenital Hypoplasia of Aortic Arch
Long Term Results in Hypoplastic Aortic Arch Repair in Neonates Using a Glutaraldehyde Treated Autologous Pericardial Patch
Hypoplastic aortic arch is a congenital heart defect that is prejudicial for the growth of newborns. It is often associated to other heart defects.
- Trial name
- Aortoplasty (NCT03588312)
- Testing
- Aortoplasty using autologous pericardial patch
- Where
- Belgium
- Volunteers
- 80 people
- Sponsor
- Queen Fabiola Children's University Hospital
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