Clinical trials: Newborn Infant
Molybdenum Cofactor Deficiency, Type A
Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A
- Trial name
- NCT02629393
- Testing
- ORGN001 (formerly ALXN1101)
- Where
- 11 sites in United States, Israel and 4 more
- Volunteers
- 5 people
- Sponsor
- Origin Biosciences
Mucopolysaccharidosis I, Hurler's Syndrome
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
The purpose of this study is to determine if laronidase is present in the breast milk of post-partum women receiving Aldurazyme® (laronidase) and the effects of Aldurazyme (laronidase) on the growth, development, and immunologic…
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie
- Trial name
- NCT00418821
- Testing
- Laronidase
- Where
- Italy
- Volunteers
- 2 people
- Sponsor
- Genzyme, a Sanofi Company
Cystic Fibrosis
28 Day Repeat Dose in Cystic Fibrosis Patients
The purpose of this study is to determine whether the safety, tolerability and pharmacodynamics of SB656933 in patients that have cystic fibrosis
- Trial name
- NCT00903201
- Testing
- SB656933
- Where
- 33 sites in United States, Canada and 3 more
- Volunteers
- 146 people
- Sponsor
- GlaxoSmithKline
Cystic Fibrosis, Advanced Lung Disease
Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
- Trial name
- NCT02390219
- Testing
- Lumacaftor, Ivacaftor
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Bronchopulmonary Dysplasia, Neurally Adjusted Ventilatory Assist
NAVA vs. CMV Crossover in Severe BPD
This prospective, unblinded, pilot randomized cross-over trial of 2 modes of mechanical ventilation will compare measures of pulmonary mechanics, respiratory gas exchange, and patient comfort between conventional flow triggered…
- Conditions
- Bronchopulmonary Dysplasia, Neurally Adjusted Ventilatory Assist, Mechanical Ventilation
- Trial name
- NCT04821453
- Testing
- NAVA, CMV
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Inhaled Mannitol on Mucociliary Clearance in Moderate to Severe Cystic Fibrosis
This study will provide important mechanistic information regarding the effect of inhaled mannitol (Bronchitol) in people with cystic fibrosis (PwCF) with moderate to severe disease who are already using…
- Trial name
- NCT05740618
- Testing
- Mannitol Inhalant Product
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of North Carolina, Chapel Hill
Cystic Fibrosis
Impact of Discontinuing Hypertonic Saline in People With CF on Highly Effective CFTR Modulators- A SIMPLIFY Sub-Study
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other chronic therapies can be…
- Trial name
- SIMPLIFY-HS (NCT06350461)
- Testing
- Discontinuation of hypertonic saline (HS), Continuation of hypertonic saline (HS)
- Where
- 81 sites in United States
- Volunteers
- 370 people
- Sponsor
- Nicole Hamblett
Neonatal Sepsis, Preterm Infants
Effect of Vitamin D as Adjuvant Therapy in Preterm Infants With Neonatal Sepsis
The goal of this clinical trial is to learn the effect of vitamin D as an adjunctive therapy for preterm neonates with sepsis measured by the outcomes, which are sepsis score and C-reactive protein after 7 days.
- Trial name
- NCT07245277
- Testing
- Vitamin D
- Where
- 2 sites in Indonesia
- Volunteers
- 78 people
- Sponsor
- Universitas Padjadjaran
Bronchopulmonary Dysplasia
Safety of Furosemide in Premature Infants at Risk of Bronchopulmonary Dysplasia (BPD)
This study will describe the safety of furosemide in premature infants at risk of bronchopulmonary dysplasia and determine the preliminary effectiveness and pharmacokinetics (PK) of furosemide. Funding Source - FDA OOPD
- Trial name
- NCT02527798
- Testing
- Furosemide Cohort 1, Furosemide Cohort 2, Furosemide Cohort 3
- Where
- 21 sites in United States
- Volunteers
- 82 people
- Sponsor
- University of North Carolina, Chapel Hill
Cystic Fibrosis
Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis (CF) Patients With Pseudomonas Aeruginosa (PA)
The purpose of this study was to evaluate the safety and efficacy of multiple courses of AZLI in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF3 (NCT00128492)
- Testing
- AZLI 75 mg two times a day (BID)/ three times a day (TID)
- Where
- 65 sites in United States, Australia and 2 more
- Volunteers
- 274 people
- Sponsor
- Gilead Sciences
Pain
Measuring the Amount of Methadone or Morphine in the Blood of Neonates, Infants & Children After Cardiac Surgery.
Study Population: Neonates, infants and children from birth to 5 years of age undergoing cardiac surgery with CPB. The use of methadone to provide analgesia may be increasing due to advantages compared to other commonly used…
- Trial name
- NCT01094522
- Testing
- Methadone, Morphine
- Where
- 3 sites in United States
- Volunteers
- 47 people
- Sponsor
- Stanford University
Sepsis, Necrotising Enterocolitis Neonatal
Integrated Predictive Monitoring for Sepsis and Necrotizing Enterocolitis
Behind the scenes pilot trial in the computer lab involving chart review to use neonatal intensive care unit data to create computer models predicting sepsis and necrotizing enterocolitis in very small premature infants
- Trial name
- PREMO (K) (NCT07254559)
- Testing
- No Intervention: Observational Cohort
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- University of Virginia
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
Necrotizing Enterocolitis of Newborn, Bowel Ischemic
CEUS Evaluation of Bowel Perfusion in Necrotizing Enterocolitis
There is no bedside imaging technique that can quantify dynamic bowel perfusion with high soft tissue contrast and sensitivity in necrotizing enterocolitis (NEC).
- Trial name
- NCT03549507
- Testing
- Sulfur hexafluoride lipid-type A microspheres
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital of Philadelphia
Persistent Pulmonary Hypertension of the Newborn
Markers of Inflammation and Lung Recovery in ECMO Patients for PPHN
Respiratory failure in newborns is common and has high rates of death. Where conventional intensive care strategies have failed, newborn children are referred to treatment with Extra- Corporeal Membrane Oxygenation (ECMO).
- Trial name
- Mi-ECMO (NCT02940327)
- Where
- United Kingdom
- Volunteers
- 24 people
- Sponsor
- University of Leicester
Cystic Fibrosis
Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P.
- Trial name
- NCT03905642
- Testing
- Arikayce™
- Where
- 11 sites in Belgium, Hungary and 5 more
- Volunteers
- 49 people
- Sponsor
- Insmed Incorporated
Asphyxia Neonatorum, Hypoxia
TOBY (TOtal Body hYpothermia): a Study of Treatment for Perinatal Asphyxia
Hypothesis: Prolonged whole body cooling in term infants with perinatal asphyxial encephalopathy reduces death and severe neurodevelopmental disability.
- Conditions
- Asphyxia Neonatorum, Hypoxia, Encephalopathy, Seizures
- Trial name
- NCT00147030
- Testing
- Whole body mild induced hypothermia
- Where
- United Kingdom
- Volunteers
- 325 people
- Sponsor
- Imperial College London
Cystic Fibrosis
Efficacy and Safety Study of AeroVanc for the Treatment of Persistent MRSA Lung Infection in Cystic Fibrosis Patients
The purpose of this study is to determine whether AeroVanc treatment is safe and effective in reducing the number of MRSA colony forming units in the lungs of cystic fibrosis patients.
- Trial name
- NCT01746095
- Testing
- Vancomycin hydrochloride inhalation powder
- Where
- 38 sites in United States
- Volunteers
- 87 people
- Sponsor
- Savara Inc.
Cystic Fibrosis
A Phase II , Placebo-controlled Study to Assess Efficacy of 28 Day Oral AZD9668 in Patients With Cystic Fibrosis
The purpose of this study is to investigate if treatment with AZD9668 for 28 days is effective in treating Cystic Fibrosis (CF) and if so how it compares to placebo (a substance which does not have any action).
- Trial name
- INCA (NCT00757848)
- Testing
- AZD9668
- Where
- 14 sites in Denmark, Germany and 4 more
- Volunteers
- 56 people
- Sponsor
- AstraZeneca
Respiratory Distress Syndrome, Newborn, Premature Birth
SURFAXIN® Treatment for Prevention of Bronchopulmonary Dysplasia (BPD) in Very Low Birth Weight (VLBW) Infants.
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required…
- Conditions
- Respiratory Distress Syndrome, Newborn, Premature Birth, Bronchopulmonary Dysplasia
- Trial name
- NCT00215540
- Testing
- Lucinactant 175 mg/kg, Lucinactant 90 mg/kg
- Where
- United States
- Volunteers
- 136 people
- Sponsor
- Windtree Therapeutics