Clinical trials: Newborn Infant
Infant, Newborn
Comparative Gastrointestinal Tolerance of Various Infant Formulas in Healthy Term Infants
The objective of this study is to assess the comparative gastrointestinal (GI) tolerance of normal term infants to two experimental soy-based formulas, relative to a commercially available soy-based formula.
- Trial name
- NCT00798382
- Testing
- soy protein formula, Commercially available soy formula, Soy formula experimental #2
- Where
- 17 sites in United States
- Volunteers
- 154 people, healthy volunteers welcome
- Sponsor
- Abbott Nutrition
Cystic Fibrosis
A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)
The purpose of this study evaluates the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who were heterozygous for F508del and a minimal function mutation (F/MF participants).
- Trial name
- NCT05033080
- Testing
- VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA
- Where
- 139 sites in United States, Australia and 10 more
- Volunteers
- 435 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Mucopolysaccharidosis Type II (MPS II)
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system.
- Trial name
- NCT04571970
- Testing
- RGX-121
- Where
- 2 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- REGENXBIO Inc.
Glycogen Storage Disease Type II
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD.
- Trial name
- Baby-COMET (NCT04910776)
- Testing
- avalglucosidase alfa
- Where
- 16 sites in United States, Belgium and 7 more
- Volunteers
- 17 people
- Sponsor
- Sanofi
Lysosomal Acid Lipase Deficiency, Wolman Disease
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
- Trial name
- NCT01358370
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Breastfeeding
Pilot Study of the FEEDBACK System for Use With Infants in the Neonatal Intensive Care Unit at University of Virginia
The purpose of this study is to assess the accuracy of FEEDBACK for measuring breastmilk intake of a pre-term baby. Mothers and babies will use the FEEDBACK system during a breastfeeding session in the NICU.
- Trial name
- NCT03702686
- Testing
- FEEDBACK System
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Molex Ventures LLC
Surgery
Calibration and Validation of Masimo's O3 Regional Oximetry Device in Neonates, Infants and Children
The study will collect cerebral oximetry data from pediatric and neonatal subjects using the Masimo O3 regional oximetry device, for the purposes of device calibration and validation.
- Trial name
- NCT03123354
- Testing
- O3 regional oximeter sensor
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Masimo Corporation
Jaundice, Neonatal
Evaluation of Bilirubin Estimates in Newborns From Smartphone Digital Images in a Nepali Population
This cross-sectional study evaluates the accuracy of a novel smartphone application that estimates bilirubin levels in newborns at Dhulikhel Hospital, Kathmandu Nepal.
- Trial name
- NCT05625880
- Testing
- Picterus Jaundice Pro
- Where
- Nepal
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Picterus AS
Cystic Fibrosis
Study Evaluating Fosfomycin/Tobramycin for Inhalation in Cystic Fibrosis Patients With Pseudomonas Aeruginosa Lung Infection
The purpose of this study is to evaluate the safety and efficacy of 2 dose combinations of fosfomycin/tobramycin for inhalation (FTI), following a 28-day course of Aztreonam for Inhalation (AZLI) in patients with cystic fibrosis…
- Trial name
- NCT00794586
- Testing
- FTI, AZLI
- Where
- 36 sites in United States
- Volunteers
- 120 people
- Sponsor
- Gilead Sciences
Neonatal Abstinence Syndrome
Donor Human Milk in Neonatal Abstinence Syndrome
This study is designed to develop pilot data on the acceptability and benefit of donor human milk for infants undergoing pharmacologic treatment for NAS.
- Trial name
- DHM&NAS (NCT02182973)
- Testing
- donor human milk
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neolac Inc dba Medolac Laboratories
Pseudomonas Infection
Cipro Inhaler for Cystic Fibrosis Children Ages 6-12
Ciprofloxacin PulmoSphere Inhalation Powder appears to be an effective and adequate antibiotic treatment for cystic fibrosis patients with P. aeruginosa colonisation.
- Trial name
- NCT00910351
- Testing
- Ciprofloxacin (Cipro, BAYQ3939)
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- Bayer
Aspergillosis, Cystic Fibrosis
The Effect of PC945 on Aspergillus Fumigatus Lung Infection in Patients With Cystic Fibrosis
This study tests the effects of an experimental drug PC945 in people with cystic fibrosis whose lungs are infected by the fungus Aspergillus fumigatus.
- Trial name
- NCT03870841
- Testing
- PC945
- Where
- 2 sites in United Kingdom
- Volunteers
- 4 people
- Sponsor
- Pulmocide Ltd
Phenylketonurias
Evaluation of PKU Explore France
PKU explore France is an exploratory study to evaluate the acceptability of PKU explore, a food for special medical purposes, for use in the dietary management of phenylketonuria in infants from 6 months to 3 years of age…
- Trial name
- NCT04898829
- Testing
- PKU explore
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Vitaflo International, Ltd
Netherton Syndrome
A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease.
- Trial name
- NCT05211830
- Testing
- SXR1096 cream
- Where
- 5 sites in Austria, France and 2 more
- Volunteers
- 9 people
- Sponsor
- Sixera Pharma
Cystic Fibrosis
Study of GLPG1837 in Subjects With Cystic Fibrosis (S1251N Mutation)
At least 6 cystic fibrosis patients with the S1251N mutation will be treated for 4 weeks, consisting of two consecutive treatment periods of two weeks evaluating one dose of GLPG1837 each.
- Trial name
- SAPHIRA2 (NCT02690519)
- Testing
- GLPG1837 dose 1, GLPG1837 dose 2
- Where
- 5 sites in Belgium, Netherlands
- Volunteers
- 7 people
- Sponsor
- Lakefront Biotherapeutics NV
Colic
Preliminary Efficacy, Safety and Pharmacokinetics Study of Nepadutant in Infant With Feeding Intolerance
The present pilot study is aimed to obtain preliminary data on the effect of three ascending oral dose levels of nepadutant on the relief of symptoms associated with feeding intolerance.
- Trial name
- NCT01532518
- Testing
- Nepadutant
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- Menarini Group
Respiratory Distress Syndrome, Newborn
Observational Study on the Prophylactic Use of Curosurf in Neonatal Respiratory Distress Syndrome (RDS)
The aim of Alizé is to describe a population of premature babies (gestational age (GA) < 32 weeks) in real life situation and the management of RDS.
- Trial name
- NCT01102543
- Where
- France
- Volunteers
- 972 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
Cystic Fibrosis
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age.
- Trial name
- NCT05882357
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Canada and 5 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years
This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.
- Trial name
- EARLY (NCT01082367)
- Testing
- TOBI
- Where
- 19 sites in Canada, Egypt and 8 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals