Clinical trials: Newborn Infant
Pulmonary Hypertension, Persistent, of the Newborn
A Non-Interventional Follow Up Study For Subjects Who Received Sildenafil for Persistent Pulmonary Hypertension of the Newborn (PPHN)
This single center study will monitor the developmental progress of PPHN patients who received iv sildenafil in study A1481276. Two visits will be conducted, at 12 and 24 months following the end of study drug infusion.
- Trial name
- NCT01801982
- Testing
- non-interventional
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Pediatric: Genetic Syndrome
Genomic Medicine for Ill Neonates and Infants (The GEMINI Study)
The Genomic Medicine for Ill Neonates and Infants (The GEMINI Study) is a research study aimed at comparing the clinical and economic utility of performing rapid whole genomic sequencing versus a targeted genomic sequencing panel…
- Trial name
- GEMINI (NCT03890679)
- Testing
- rapid whole genomic sequencing (rWGS)
- Where
- 6 sites in United States
- Volunteers
- 400 people
- Sponsor
- Tufts Medical Center
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
- Trial name
- NCT04545515
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 34 sites in Australia, Canada and 8 more
- Volunteers
- 120 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
OPTION: A Trial to Assess the Safety & Efficacy of MS1819 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
The primary objectives of this study are to assess the safety and efficacy of MS1819-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
- Trial name
- OPTION (NCT03746483)
- Testing
- MS1819, Porcine PERT
- Where
- 14 sites in United States, Poland
- Volunteers
- 41 people
- Sponsor
- Entero Therapeutics
Hypoxic Ischemic Encephalopathy
Pilot Study of Head Cooling in Preterm Infants With Hypoxic Ischemic Encephalopathy
The hypothesis is that premature infants' can have enough cooling applied to cool their brain to decrease CNS injury without cooling their body.
- Trial name
- NCT00620711
- Testing
- Olympic Cool Cap
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Vanderbilt University
Premature Infants
A Randomized Trial of Outpatient Oxygen Weaning Strategies in Premature Infants
The investigators hypothesize that Recorded Home Oximetry (RHO) utilization will not increase rates of respiratory-related re-hospitalizations and ED visits, and will not impair growth compared to standard oxygen management…
- Trial name
- NCT01994954
- Testing
- RHO
- Where
- 8 sites in United States
- Volunteers
- 196 people
- Sponsor
- University of Massachusetts, Worcester
Neonatal Brachial Plexus Palsy
NMES Efficacy on Patients With NBPP
Neonatal Brachial Plexus Palsy (NBPP), characterized by weakness and sensory loss in the affected arm, results from damage in the prenatal period to the nerves extending from the spine to the arm.
- Trial name
- NCT01999465
- Testing
- Standard NMES device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Michigan
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
- Trial name
- NCT02971839
- Testing
- VX-561, IVA
- Where
- 14 sites in United States
- Volunteers
- 11 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Neonatal Encephalopathy, Hypoxic-Ischemic Encephalopathy Mild
Mild Encephalopathy in the Newborn Treated With Darbepoetin
This is a Phase II multicenter placebo-controlled randomized, feasibility/safety trial. Infants >34 week gestational age with perinatal acidemia and mild neonatal encephalopathy on the modified Sarnat neurologic examination at…
- Trial name
- MEND (NCT03071861)
- Testing
- Darbepoetin Alfa, Normal Saline
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of New Mexico
Atopic Dermatitis
5-Year Safety Study of Pimecrolimus Cream 1% in Infants 3 - 12 Months of Age With Mild to Moderate Atopic Dermatitis
The primary purpose of this study is to investigate the safety of pimecrolimus cream 1% in the long-term treatment (up to 5 years) of atopic dermatitis (eczema) in patients less than 12 months of age compared to topical…
- Trial name
- NCT00120523
- Testing
- Pimecrolimus, Topical corticosteroids
- Where
- 31 sites in United States, Germany
- Volunteers
- 2,418 people
- Sponsor
- MEDA Pharma GmbH & Co. KG
Congenital Hyperinsulinism
Effect of Exendin-(9-39) on Glycemic Control in Subjects With Congenital Hyperinsulinism
The purpose of this study is to determine if Exendin-(9-39), an antagonist of the glucagon-like peptide-1 (GLP-1) receptor with effects on the pancreatic beta cell, increases fasting blood glucose levels in subjects with…
- Trial name
- NCT00571324
- Testing
- Exendin-(9-39), Vehicle
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Diva De Leon
Cystic Fibrosis
The Impact of Insulin Therapy on Protein Turnover in Pre-Diabetic Cystic Fibrosis Patients
Insulin replacement therapy may be effective in breaking the cycle of protein catabolism, undernutrition and overall clinical deterioration in pre-diabetic, insulin insufficient CF youth because of its potent anabolic effect.
- Trial name
- NCT02496780
- Testing
- novolog insulin, levemir insulin
- Where
- 2 sites in United States
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Cystic Fibrosis
Early Intervention in Cystic Fibrosis Exacerbation
Individuals with cystic fibrosis (CF) develop chronic lung infections and suffer intermittent acute exacerbations of their lung disease.
- Trial name
- eICE (NCT01104402)
- Testing
- Home lung function and symptom monitoring
- Where
- 3 sites in United States
- Volunteers
- 267 people
- Sponsor
- Johns Hopkins University
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Cystic Fibrosis, Prediabetes
Prevention of Cystic Fibrosis Diabetes
Acute systemic hyperglycemia causes oxidative stress and a pro-inflammatory response. The pro-inflammatory cytokines induced by hyperglycemia are toxic to islet insulin producing cells, and thus worsen glucose intolerance.
- Trial name
- Prevent (NCT00967798)
- Testing
- Sitagliptin
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- Emory University
Coarctation of the Aorta, Pulmonary Artery Stenosis
Growth Trial: Study of the Renata Minima Stent
The objective of the clinical investigation is to access clinical safety and effectiveness of the Minima Stent in neonates, infants, and young children requiring intervention for common congenital vascular stenosis (i.e.…
- Trial name
- NCT05086016
- Testing
- Catheterized Stenting
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Renata Medical
Hypoglycemia, Newborn Morbidity
Efficacy of Continuous Glucose Monitoring in Neonates With Hypoglycemia
Our aim is to determine the feasibility of using continuous glucose monitors (CGMs) in infants with low blood glucose to improve how we care for these infants.
- Trial name
- NCT02300285
- Testing
- Continuous Glucose Monitoring
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Boston Children's Hospital
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care (Intralipid) from 7 up to 90 days to develop Essential Fatty Acid…
- Trial name
- NCT04555044
- Testing
- Clinolipid, Intralipid
- Where
- 9 sites in United States
- Volunteers
- 101 people
- Sponsor
- Baxter Healthcare Corporation
Cystic Fibrosis
Safety Study of Ivacaftor in Subjects With Cystic Fibrosis
The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosis transmembrane conductance…
- Trial name
- NCT00457821
- Testing
- Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg
- Where
- 15 sites in United States, Canada and 1 more
- Volunteers
- 39 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Chronic Kidney Diseases
Peer i-Coaching for Activated Self-Management Optimization in Adolescents and Young Adults With Chronic Conditions
The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood…
- Conditions
- Sickle Cell Disease, Chronic Kidney Diseases, Systemic Lupus Erythematosus, Childhood Cancer
- Trial name
- PiCASO (NCT03938324)
- Testing
- PiCASO Intervention Group
- Where
- United States
- Volunteers
- 222 people
- Sponsor
- Duke University