Clinical trials: NEWBORN
Heart Failure, Cardiogenic Shock
Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System
There are two primary goals of this multicenter clinical trial that combines an FDA device trial and a phase II drug trial in the same study cohort. These two goals are to: 1.
- Conditions
- Heart Failure, Cardiogenic Shock, Congenital Heart Disease, Cardiomyopathies
- Trial name
- TROLLEY (NCT06080074)
- Testing
- Cardiohelp device (VA-ECMO), Heparin, Bivalirudin
- Where
- 5 sites in United States
- Volunteers
- 50 people
- Sponsor
- Stanford University
Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia
Glucose Homeostasis in Pseudohypoparathyroidism
It is increasingly recognized that Pseudohypoparathyroidism type 1A (PHP1A) is associated with an increased risk of type 2 diabetes but the mechanism is unknown.
- Conditions
- Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia, Albright Hereditary Osteodystrophy
- Trial name
- NCT03761290
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Vanderbilt University Medical Center
Microtia, Dysbiosis
Microbiome Metabolites and Alcohol in HIV to Reduce CVD RCT
Among people living with HIV, heavy drinking increases the risk of heart disease and death. Studies suggest that alcohol changes the number and kind of bacteria in your gut and these changes increase the risk of heart disease and…
- Conditions
- Microtia, Dysbiosis, Alcohol Drinking, HIV Infections
- Trial name
- META HIV CVD (NCT05288790)
- Testing
- Probiotic
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- Vanderbilt University Medical Center
Congenital Diaphragmatic Hernia
North American Fetal Therapy Network Prospective Registry for Long-term Outcome Following Fetoscopic Endoluminal Tracheal Occlusion in Severe Left and Right Congenital Diaphragmatic Hernia
The purpose of this research is to determine if babies who undergo a Fetoscopic Endoluminal Tracheal Occlusion (FETO) procedure survive more often and have less long-term complications than babies who have similarly severe…
- Trial name
- NCT07564297
- Testing
- Fetoscopic Endoluminal Tracheal Occlusion, Standard of Care
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- Mayo Clinic
Renal Fanconi Syndrome, Photophobia
Biomarker for Cystinosis Disease: BioCystinosis (BioCystinosis)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Cystinosis disease from the blood
- Conditions
- Renal Fanconi Syndrome, Photophobia, Hypophosphatemia
- Trial name
- BioCystinosis (NCT02837523)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Low-Risk Pregnant Women at Full-Term Gestation, Healthy Fetal Population
Advancing Early Bone Health: New Frontiers for Osteoporosis Prevention
All factors that influence the peak bone mass achieved at skeletal maturity are important in determining an individual's risk of developing osteoporosis later in life.
- Conditions
- Low-Risk Pregnant Women at Full-Term Gestation, Healthy Fetal Population, Healthy Neonatal Population
- Trial name
- REMS-BONE (NCT06750523)
- Testing
- Cord blood sampling, urine sampling, Life style questionnaire
- Where
- 2 sites in Italy
- Volunteers
- 200 people
- Sponsor
- University of Parma
22q11.2 Deletion Syndrome, Velo-Cardio-Facial Syndrome
Cognitive Remediation in 22q11DS
The goal of this study is to collect preliminary data on the efficacy of a cognitive remediation program in improving the neurocognitive deficits in children with chromosome 22q11.2 deletion syndrome (22q11DS).
- Trial name
- NCT01781923
- Testing
- Cognitive remediation program, Small group social skills training
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Duke University
Atopic Dermatitis, Atopic Dermatitis (AD)
Crisaborole 2% Versus Tacrolimus 0.1% in Children With Mild to Moderate Atopic Dermatitis
This randomized, open-label, parallel-group study will compare the efficacy and safety of Crisaborole 2% ointment and Tacrolimus 0.1% ointment in children with mild to moderate atopic dermatitis.
- Trial name
- NCT07162896
- Testing
- Crisaborole 2%, Tacrolimus 0.1%
- Where
- Pakistan
- Volunteers
- 66 people
- Sponsor
- Shalamar Institute of Health Sciences
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Friedreich Ataxia
Methylprednisolone Treatment of Friedreich Ataxia
This study will explore whether methylprednisolone treatment is safe, well-tolerated, and beneficial in patients that are diagnosed with Friedreich Ataxia (FRDA).
- Trial name
- NCT02424435
- Testing
- Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Children's Hospital of Philadelphia
Haemophilia
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
In the past, due to economic and medical resource constraints, the hemophilia comprehensive care in China was suboptimal. The BCH data of both retrospective and prospective studies reveals that for 4-6y and 6-9y patients with…
- Trial name
- CHIPS (NCT02999308)
- Testing
- Observe the patient's condition and then change the regimen
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Beijing Children's Hospital
Atopic Dermatitis, Eczema, Atopic
Dupilumab Step-down Strategy to Maintain Remission in Adult and Adolescents Patients With Atopic Dermatitis
The goal of this clinical trial is to compare a step-down strategy of spacing dupilumab injections with a standard maintenance treatment in adolescents and adults with controlled Atopic dermatitis (AD) for at least six months.
- Trial name
- MADULO (NCT05642208)
- Testing
- Dupilumab step-down
- Where
- 32 sites in France, Reunion
- Volunteers
- 256 people
- Sponsor
- Nantes University Hospital
Hemoglobin Disorder
Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Cell Transplant or Gene Therapy
This is a prospective, longitudinal, non-therapeutic study which includes routine assessment for long-term effects, as per FDA guidelines after receipt of an allogeneic HCT or autologous genetically modified cellular products for…
- Trial name
- NCT06646640
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- St. Jude Children's Research Hospital
Genetic Disease, Immune Dysfunction
Natural History of Type 1 Interferonopathies: Insights From a European Cohort
Type I interferonopathies are rare autoinflammatory disorders caused by genetic defects and associated with significant morbidity and mortality. These diseases are refractory to conventional immunosuppressive therapies.
- Conditions
- Genetic Disease, Immune Dysfunction, Neurological Diseases or Conditions, Autoimmune Diseases
- Trial name
- EU-IFNp (NCT07040774)
- Where
- 32 sites in Austria, Belgium and 9 more
- Volunteers
- 500 people
- Sponsor
- Imagine Institute
Thalassemia, Iron Overload
Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox
Thalassemia Major patients developed Iron Overload due to blood transfusions and intestinal iron absorption. Renal function caused by Iron overload was studied in a previous study and shows principally tubular disfunction.
- Trial name
- NCT01905774
- Testing
- Deferasirox
- Where
- Israel
- Volunteers
- 36 people
- Sponsor
- HaEmek Medical Center, Israel
Parvovirus B19 Infection
Parvovirus B19 Infection in Hereditary Hemolytic Anemias Patients
Although many studies investigated the prevalence and manifestations of HPV-B19 infection in patients with sickle cell anemia (SCA), thalassemia, and hereditary spherocytosis (HS) separately, there is limited information about…
- Trial name
- NCT04822090
- Testing
- Supportive treatment with or without red cell transfusion/intravenous immune globulin (IVIG)
- Where
- Egypt
- Volunteers
- 244 people
- Sponsor
- Sohag University
Muscular Dystrophies, Muscular Dystrophy
Muscle Tissue Bank for Muscular Dystrophy
This protocol proposes to establish gene expression profiles of muscular dystrophies for correct diagnosis and for development of experimental therapies for these diseases.
- Trial name
- NCT01950897
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Cystic Fibrosis, Lungtransplantation
Upper and Lower Airway Colonization in Cystic Fibrosis Patients After Lung Transplantation
A hot topic in lung transplantation is the treatment of persisting sinus disease/colonization in CF patients to prevent descending graft colonization and chronic allograft dysfunction.
- Conditions
- Cystic Fibrosis, Lungtransplantation, Paranasal Sinus Diseases
- Trial name
- NCT02591524
- Testing
- Bronchoalveolar and Nasal Lavage
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Hannover Medical School
Cystic Fibrosis
Evaluation of Sit-to-stand Test in Patients With Cystic Fibrosis and Matched Controls.
Introduction: In recent years, since the discovery of the cystic fibrosis (CF) transmembrane conductance regulator gene in human skeletal muscle, there appears to be growing interest in the measurement of muscle function in CF.
- Trial name
- STS-CF (NCT03524859)
- Where
- Spain
- Volunteers
- 79 people, healthy volunteers welcome
- Sponsor
- Universidad Católica San Antonio de Murcia
ARDS, TTM
TwichView-guided Atricurium Dose Adjustment Trial
The goal of this clinical trial is to learn if using a monitor that measures muscle response can lower the amount of muscle relaxant medicine needed in adults who are treated in an intensive care unit (ICU) and need a continuous…
- Conditions
- ARDS, TTM, ECMO, Neuromuscular Blockade Monitoring
- Trial name
- NCT07807046
- Testing
- EMG-based quantitative TOF-guided atracurium titration
- Where
- Taiwan
- Volunteers
- 120 people
- Sponsor
- Fu Jen Catholic University Hospital