Clinical trials: Newborn Infant
SpO2, Nasal Alar Collapse, Bilateral
Project Mountain - Comparing SpO2 and SaO2 for Accuracy
The main goal of this study is to look at the performance of the neonatal, infant, and pediatric Philips SpO2 sensors with the Philips FAST Pulse Oximetry technology.
- Conditions
- SpO2, Nasal Alar Collapse, Bilateral, Oxygen, Measurement
- Trial name
- NCT06372106
- Testing
- SaO2 Sampling
- Where
- 5 sites in United States
- Volunteers
- 560 people, healthy volunteers welcome
- Sponsor
- Philips Clinical & Medical Affairs Global
Respiratory Distress Syndrome In Premature Infants
Safety and Efficacy Study of the Draeger Babylog VN500 Device in HFOV Mode in VLBW Neonates
The purpose of this study is to determine the safety and effectiveness of the Babylog VN500 in high frequency oscillatory ventilation (HFOV) mode as a method for treating very low birth weight (VLBW) neonates requiring invasive…
- Trial name
- NCT02445040
- Testing
- Babylog VN500 in HFOV Mode
- Where
- 14 sites in United States
- Volunteers
- 225 people
- Sponsor
- Draeger Medical Systems, Inc.
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Cystic Fibrosis
A Study to Compare US Marketed Creon Manufactured With a Modernized Process at an Alternate Manufacturing Site and Manufactured With the Approved Manufacturing Process at an Alternate Active Pharmaceutical Ingredient Site, in Participants With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
Part 1 is a study to demonstrate that Creon (pancrelipase) delayed release (DR) capsules manufactured with a modernized process (MP) is non-inferior to currently marketed pancrelipase DR capsules in participants with exocrine…
- Trial name
- NCT03924947
- Testing
- Pancrelipase
- Where
- 13 sites in United States
- Volunteers
- 36 people
- Sponsor
- AbbVie
Cystic Fibrosis
Safety, Tolerability and Pharmacokinetics of BIIL 284 BS in Adult and Pediatric Cystic Fibrosis (CF) Patients
Safety, tolerability and pharmacokinetics following repeated doses (15-day dosing)
- Trial name
- NCT02269189
- Testing
- BIIL 284 BS, high dose, BIIL 284 BS, low dose
- Volunteers
- 36 people
- Sponsor
- Boehringer Ingelheim
Cystic Fibrosis
ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del Mutation
The purpose of this program is to provide elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) to CF patients in critical need who are 6 to 11 years of age with at least one F508del mutation in response to unsolicited physician…
- Trial name
- NCT04702360
- Testing
- ELX/TEZ/IVA, IVA
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®
This study evaluates the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients with Cystic Fibrosis previously treated with Pulmozyme®.
- Trial name
- NCT02722122
- Testing
- AIR DNase™
- Where
- 5 sites in Israel
- Volunteers
- 15 people
- Sponsor
- Protalix
Jaundice, Neonatal Hyperbilirubinemia
Observational Follow-up of Participants From Clinical Trial 64,185-202 (NCT00850993)
Observational follow-up of participants from earlier interventional trial 64,185-202 (NCT00850993). No interventions were administered during this follow-up study.
- Trial name
- NCT02033096
- Testing
- Stannsoporfin, Phototherapy (as needed)
- Volunteers
- 42 people
- Sponsor
- InfaCare Pharmaceuticals Corporation, a Mallinckrodt Company
Cystic Fibrosis
Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)
The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
- Trial name
- NCT06506773
- Where
- 50 sites in United States, Australia and 9 more
- Volunteers
- 79 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Safety and Tolerability Study of Denufosol in 2-4 Year Olds
The purpose of this study is to evaluate the safety and tolerability of denufosol 60 mg TID in pediatric CF patients 2 to 4 years of age
- Trial name
- REACH-1 (NCT01181622)
- Testing
- denufosol tetrasodium Inhalation Solution, 0.9% w/v sodium chloride solution
- Volunteers
- 25 people
- Sponsor
- Merck Sharp & Dohme LLC
Cystic Fibrosis
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein.
- Trial name
- NCT00458341
- Testing
- Ataluren
- Where
- 3 sites in Belgium, France
- Volunteers
- 30 people
- Sponsor
- PTC Therapeutics
Pregnancy Related
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period [LMP]).
- Trial name
- NCT07556913
- Testing
- Elafibranor
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Ipsen
Cystic Fibrosis
Safety, Tolerability, Pharmacokinetics, and Efficacy of JBT-101 (Lenabasum) in Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and efficacy of JBT-101 in adult subjects with cystic fibrosis (CF).
- Trial name
- NCT02465450
- Testing
- JBT-101 (lenabasum)
- Where
- 27 sites in United States, Belgium and 5 more
- Volunteers
- 85 people
- Sponsor
- Corbus Pharmaceuticals Inc.
Cystic Fibrosis
Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.
- Trial name
- NCT03258424
- Testing
- PTI-428
- Where
- 2 sites in United Kingdom
- Volunteers
- 15 people
- Sponsor
- Proteostasis Therapeutics, Inc.
Neonatal Sepsis
Late-onset Sepsis in Term and Pre-term Neonates and Infants up to 3 Months of Age
This study evaluated the safety, pharmacokinetics and efficacy of ceftobiprole in term and pre-term newborn babies and infants up to 3 months of age with late-onset sepsis (LOS).
- Trial name
- NCT05856227
- Testing
- Ceftobiprole medocaril
- Where
- 14 sites in United States, Bulgaria and 6 more
- Volunteers
- 9 people
- Sponsor
- Basilea Pharmaceutica
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Infant Newborn
Growth and Tolerance of Young Infants Fed Infant Formulas
This is a randomized, multi-center, double blind, parallel study to compare growth and tolerance of healthy term infants fed either control or experimental infant formula.
- Trial name
- NCT01380886
- Testing
- experimental infant formula with alternate protein source, Infant formula powder
- Where
- 12 sites in United States
- Volunteers
- 209 people, healthy volunteers welcome
- Sponsor
- Abbott Nutrition
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
MPS II, Hunter Syndrome (MPS II)
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
The objective of this study is to assess the long-term safety and efficacy of Hunterase for approximately 1 year in subjects who completed the end of study (EOS) visit (Visit 54) tests in the Phase 3 Hunterase study and patients…
- Conditions
- MPS II, Hunter Syndrome (MPS II), Hunter Syndrome, Hunterase
- Trial name
- NCT07344376
- Testing
- Hunterase
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- GC Biopharma Corp
Respiratory Tract Infections
A Study of Rilematovir in Infants and Children and Subsequently in Neonates Hospitalized With Acute Respiratory Tract Infection Due to Respiratory Syncytial Virus (RSV)
The purpose of the study is to evaluate the efficacy of rilematovir compared to placebo treatment with respect to the clinical outcome on the RSV Recovery Scale (RRS).
- Trial name
- DAISY (NCT04583280)
- Testing
- Rilematovir, Rilematovir X mg/kg
- Where
- 142 sites in United States, Argentina and 24 more
- Volunteers
- 28 people
- Sponsor
- Janssen Research & Development, LLC