Clinical trials: Newborn Infant
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire
Neonatal Anemia
Milking Versus Delayed Cord Clamping in Full Term Neonates
To compare the short term risks and benefits of cord milking 5 times toward the neonate with delayed cord clamping for 120 seconds in the full term neonate delivered by cesarean section.
- Trial name
- MDCCT (NCT02454101)
- Testing
- cord milking, delayed cord clamping
- Where
- Egypt
- Volunteers
- 300 people
- Sponsor
- Ahmed Mohamed El Kotb Abdel Fattah
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…
- Trial name
- STRIVE (NCT00909532)
- Testing
- Ivacaftor
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 167 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Bronchopulmonary Dysplasia
Inhaled Nitric Oxide (INO) for the Prevention of Bronchopulmonary Dysplasia (BPD) in Preterm Infants
This phase 3, multi-center, double blind, placebo-controlled, randomized clinical trial will attempt to demonstrate if preterm infants who require mechanical ventilation and/or positive pressure support at any point during days 5…
- Trial name
- NCT00931632
- Testing
- Inhaled Nitric Oxide
- Where
- 33 sites in United States, Canada
- Volunteers
- 451 people
- Sponsor
- Mallinckrodt
Sjögren-Larsson Syndrome
A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)
This is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, pharmacokinetic (PK), and exploratory activity of topically-applied NS2 dermatologic cream administered…
- Trial name
- NCT02402309
- Testing
- Active topical NS2 1% dermatologic cream
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Aldeyra Therapeutics, Inc.
Netherton Syndrome
Natural History of Netherton Syndrome
The goal of this non-interventional study (NIS) is to collect real-world data to describe the natural history of Netherton Syndrome (NS).
- Trial name
- NCT05902663
- Where
- 4 sites in United States, Italy
- Volunteers
- 4 people
- Sponsor
- Boehringer Ingelheim
Neonatal Abstinence Syndrome
Fetal and Infant Effects of Maternal Buprenorphine Treatment
This research will track the longitudinal neurobehavioral development of the buprenorphine-exposed fetus across gestation through 1 month of age in an effort to determine the safety of this medication for use during gestation…
- Trial name
- NCT01561079
- Testing
- Buprenorphine
- Where
- 2 sites in United States
- Volunteers
- 127 people
- Sponsor
- Johns Hopkins University
Respiratory Syncytial Virus Infection
Clinical Study of Palivizumab in Japanese Newborns, Infants and Young Children at the Age of 24 Months or Less With Immunocompromised Medical Conditions
To evaluate safety, efficacy and pharmacokinetics of palivizumab in children at the age of 24 months or less with immunocompromised medical conditions.
- Trial name
- NCT01466062
- Testing
- Palivizumab
- Where
- 6 sites in Japan
- Volunteers
- 28 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Hypophosphatasia (HPP)
Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)
This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.
- Trial name
- NCT00744042
- Testing
- asfotase alfa
- Where
- 10 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cystic Fibrosis
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
This is a first-in-human and proof-of-concept study of VX-445. The study includes 6 parts. Parts A, B, and C were conducted in healthy subjects.
- Trial name
- NCT03227471
- Testing
- IVA, TEZ/IVA, VX-445
- Where
- 38 sites in United States, Australia and 2 more
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Nasal Epithelial Cells/Blood Lymphocyte Markers for Cystic Fibrosis (CF)/CF Pulmonary Exacerbations
Study Hypothesis: We hypothesize that cellular markers from nasal epithelial cells and blood lymphocytes can serve as potential biomarkers reflect the underlying inflammatory state of the lung and will be helpful in determining…
- Trial name
- NCT00381628
- Testing
- epithelial cells and blood lymphocyte extraction
- Where
- United States
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Umbilical Hernia
Analgesic Efficacy After Umbilical Hernia Repair in Children
Umbilical hernia repair is a common painful outpatient procedure performed in children. Often analgesia for this procedure is provided by using local infiltration of the surgical site by the surgeons and perioperative opioids and…
- Trial name
- NCT00578136
- Testing
- Bupivacaine
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Children's Hospital of Philadelphia
Infant, Premature, Diseases, Infant, Small for Gestational Age
Optimizing Individual Nutrition in Preterm Very Low Birth Weight Infants
In preterm infants fed human milk, milk needs to be fortified to meet nutrient recommendations. Fortification can be 1) standard, 2) individualized (adjusted based on daily human milk nutrient analysis and milk volume), or 3)…
- Trial name
- NCT02372136
- Testing
- Individualized Nutrition, Optimized nutrition
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Texas Southwestern Medical Center
Candidiasis, Invasive
Safety, Tolerability, and Efficacy of Caspofungin Versus Amphotericin B Deoxycholate in the Treatment of Invasive Candidiasis in Neonates and Infants (MK-0991-064)
The study will evaluate the safety, tolerability, and efficacy of caspofungin as compared with amphotericin B deoxycholate in the treatment of invasive candidiasis in neonates and infants.
- Trial name
- NCT01945281
- Testing
- Caspofungin, Amphotericin B Deoxycholate
- Volunteers
- 51 people
- Sponsor
- Merck Sharp & Dohme LLC
Adrenal Insufficiency
Treatment of Adrenal Insufficiency in Children
The study will be conducted in a total of 24 subjects before their 6th birthday, requiring replacement therapy for adrenal insufficiency due to either CAH, primary adrenal failure or hypopituitarism.
- Trial name
- NCT02720952
- Testing
- Infacort®
- Where
- Germany
- Volunteers
- 24 people
- Sponsor
- Neurocrine UK Limited
Feeding; Difficult, Newborn, Growth Failure
Prophylactic Probiotics to Extremely Low Birth Weight Prematures
Mortality and incidence of severe complications is still high among extremely premature infants. Common causes of severe complications in this population are poor nutrition, necrotizing enterocolitis, and severe infections.
- Conditions
- Feeding; Difficult, Newborn, Growth Failure, Necrotizing Enterocolitis, Sepsis
- Trial name
- PROPEL (NCT01603368)
- Testing
- Lactobacillus reuteri
- Where
- 2 sites in Sweden
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Ostergotland County Council, Sweden
Cystic Fibrosis, Irritable Bowel Syndrome
Neuromodulation for Children With Cystic Fibrosis Experiencing Chronic Abdominal Pain
Chronic abdominal pain is extremely common in individuals with Cystic Fibrosis (CF). Therapy for chronic abdominal pain is very limited and generally consists of osmotic laxatives or drugs that are used to treat irritable bowel…
- Conditions
- Cystic Fibrosis, Irritable Bowel Syndrome, Chronic Pain
- Trial name
- NCT05515250
- Testing
- Use of neurostimulation device
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Louis University
Cystic Fibrosis
Deposition of Inhaled Prolastin in Cystic Fibrosis Patients
The objective of this trial is to determine the optimal region of the lung for depositing Prolastin (alpha-1 antitrypsin; AAT) by inhalation in order to treat cystic fibrosis (CF).
- Trial name
- CF2 (NCT00486837)
- Testing
- Alpha1-Proteinase Inhibitor (Human)
- Volunteers
- 72 people
- Sponsor
- Grifols Therapeutics LLC
Neonatal Brachial Plexus Palsy
Validation Study Of A Digital Measuring Device For Central Hand Representation In Children With A Neonatal Brachial Plexus Palsy
Hand function is one of the most complex and sophisticated sensorimotor skills, controlled by the interplay of precise motor efferent and multisensory afferent stimuli.
- Trial name
- NCT06950879
- Testing
- Measurement
- Where
- Belgium
- Volunteers
- 18 people
- Sponsor
- University Hospital, Ghent
Cystic Fibrosis, Healthy
Utilizing Exhaled Breathe Condensate Collection to Study Ion Regulation in Cystic Fibrosis
Our aims were to determine if exhaled breath condensate (EBC) could detect differences in ion regulation between cystic fibrosis (CF) and healthy and measure the effect of the albuterol on EBC ions in these populations.
- Trial name
- NCT01880723
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- University of Arizona