Clinical trials: Newborn Infant
Cystic Fibrosis
A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)
This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function…
- Trial name
- NCT04599465
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 41 sites in Australia, Belgium and 5 more
- Volunteers
- 69 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Severe Bronchopulmonary Dysplasia
PNEUMOSTEM for the Prevention and Treatment of Severe BPD in Premature Infants
This study is to evaluate the efficacy and safety of PNEUMOSTEM® for the Prevention and Treatment of Severe Bronchopulmonary Dysplasia (Severe BPD) in Premature Infants.
- Trial name
- NCT03392467
- Testing
- PNEUMOSTEM
- Where
- 2 sites in South Korea
- Volunteers
- 60 people
- Sponsor
- Medipost Co Ltd.
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Cystic Fibrosis
Study of Safety, Tolerability & Efficacy in Cystic Fibrosis Patients With Abnormal Glucose Tolerance
This is a Phase 2, multi-center, double blind, placebo controlled study to evaluate the safety and tolerability of PBI-4050, and its effects on the pancreatic, pulmonary functions and on various biomarkers in Cystic Fibrosis…
- Trial name
- NCT02955888
- Testing
- PBI4050
- Where
- 7 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Liminal BioSciences Ltd.
Cystic Fibrosis
Pharmacokinetics of GLPG2737 in Male Subjects With Cystic Fibrosis
This is a single dose, open label study in adult male subjects with cystic fibrosis to investigate the pharmacokinetics, safety and tolerability of GLPG2737.
- Trial name
- NCT03450720
- Testing
- GLPG2737 single dose
- Where
- Belgium
- Volunteers
- 6 people
- Sponsor
- Lakefront Biotherapeutics NV
Atopic Dermatitis / Eczema
To Evaluate the in Use Tolerance of E45 Eczema Repair Emollient in Babies and Children With (Very(Dry/Atopic Skin
The investigation will evaluate, under dermatological control, the cutaneous in-use tolerance of E45 Eczema Repair Emollient with two or three times daily application, over a period of two weeks on children and babies with…
- Trial name
- NCT02857062
- Testing
- E45 Eczema Repair Emollient
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Reckitt Benckiser Healthcare (UK) Limited
Cystic Fibrosis
To Assess the Safety, Tolerability and Pharmacokinetics of AZD5634 Following Inhaled and Intravenous (IV)Dose Administration
This is a Phase 1, first-in-human (FIH) single ascending dose study being conducted to better understand the safety, tolerability and pharmacokinetics of AZD5634 in healthy subjects
- Trial name
- NCT02679729
- Testing
- AZD5634 for inhalation, AZD5634 for infusion
- Where
- 2 sites in United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Hemolytic Disease of the Fetus and Newborn
A Study on Hemolytic Disease of the Fetus and Newborn (HDFN) Through Global Registry
The purpose of this non-interventional study is to prospectively evaluate the risk of anemia (decreased red blood cells) in fetuses (baby before birth) and neonates (baby just after birth) of pregnant participants who are at risk…
- Trial name
- GERANIUM (NCT07194070)
- Testing
- Standard of Care
- Where
- 17 sites in United States, Australia and 7 more
- Volunteers
- 175 people
- Sponsor
- Janssen Research & Development, LLC
Neonatal Hypoxic-Ischemic Encephalopathy (HIE)
CoolCap Trial, Treatment of Perinatal Hypoxic-Ischemic Encephalopathy
This is a research study of head cooling. Its goal is to determine whether cooling babies' heads can reduce or prevent brain damage that may have resulted from temporarily reduced oxygen supply to the brain.
- Trial name
- NCT00383305
- Testing
- Cool-Cap
- Where
- 27 sites in United States, Canada and 2 more
- Volunteers
- 235 people
- Sponsor
- Olympic Medical
Persistent Pulmonary Hypertension of the Newborn
Persistent Pulmonary Hypertension of the Newborn
The AC-052-391-study is a phase 3 study to investigate whether adding bosentan to inhaled nitric oxide in newborns with persistent pulmonary hypertension of newborns (PPHN) is a supporting and safe therapy and to evaluate the…
- Trial name
- FUTURE 4 (NCT01389856)
- Testing
- Bosentan
- Volunteers
- 23 people
- Sponsor
- Actelion
Neonatal Abstinence Syndrome
tAN to Mitigate Withdrawal Behaviors in Neonates
This first in-human-neonates, open-label pilot trial is designed to determine whether use of tAN in newborns with NOWS receiving oral morphine allows for faster weaning of morphine and decrease morphine use altogether.
- Trial name
- NCT04588519
- Testing
- Spark Biomedical Roo Transcutaneous Auricular Neurostimulation (tAN) System
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Spark Biomedical, Inc.
Spinal Muscular Atrophy Type I
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1.
- Trial name
- NCT07070999
- Testing
- GB221
- Where
- Brazil
- Volunteers
- 22 people
- Sponsor
- Gemma Biotherapeutics
Tuberculosis
Study to Evaluate Safety and Immunogenicity of VPM1002 in Comparison With BCG in Newborn Infants in South Africa
Goal of VPM is the development of a recombinant urease C-deficient listeriolysin expressing BCG vaccine strain (VPM1002) as a safe, well tolerated and efficacious vaccine against tuberculosis (TB) for residents in endemic areas…
- Trial name
- NCT01479972
- Testing
- VPM1002, BCG
- Where
- South Africa
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Serum Life Science Europe GmbH
Acid Lipase Deficiency, Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type
Biomarker for Wolman Disease (BioWolman)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Wolman disease blood (plasma)
- Conditions
- Acid Lipase Deficiency, Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type, Cholesterol Ester Storage Disease
- Trial name
- BioWolman (NCT02383641)
- Where
- 3 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
SpO2
Philips FAST SpO2 Technology With Masimo Sensors for SpO2 Monitoring in the Neonatal, Infant, and Pediatric Populations
The objective of this study is to verify the form, fit and function of the Philips FAST technology with Masimo neonatal, infant and pediatric sensors for SpO2 monitoring in the respective indicated populations.
- Trial name
- NCT07223177
- Testing
- Masimo Sensor (RD SET and/or LNCS) connected to Philips Monitor using Philips FAST SpO2 Algorithm
- Where
- 2 sites in United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
Neonatal Staphylococcal Sepsis
Safety and Pharmacokinetics Study in VLBW Neonates With BSYX-A110
"Phase I/II, Randomized, Double Blind, Placebo Controlled, Dose Escalating, Safety and Pharmacokinetics Study in Very Low Birth Weight Neonates of Four Doses of BSYX-A110 for the Prevention of S.
- Trial name
- N002 (NCT00631878)
- Testing
- Pagibaximab (formerly BSYX-A110)
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Biosynexus Incorporated
Mucopolysaccharidosis II
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
This is a six-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity…
- Trial name
- NCT04007536
- Testing
- No Intervention
- Where
- 6 sites in United States, Netherlands and 1 more
- Volunteers
- 18 people
- Sponsor
- Denali Therapeutics Inc.
Congenital Hyperinsulinism
CSI-Glucagon for Prevention of Hypoglycemia in Children With Congenital Hyperinsulinism
This is a Phase 2, multi-center, randomized, placebo-controlled, double-blind trial with open-label follow-up designed to assess the efficacy of Xeris Glucagon delivered as a continuous subcutaneous infusion to prevent…
- Trial name
- NCT02937558
- Testing
- Glucagon
- Where
- 5 sites in United States
- Volunteers
- 5 people
- Sponsor
- Xeris Pharmaceuticals
Phenylketonuria, Maternal
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
This is a Phase 4 observational study designed to assess the impact of Palynziq ® (pegvaliase) treatment in pregnant women with PKU and on their offspring who were exposed to pegvaliase at any time during pregnancy and…
- Trial name
- PALomino (NCT05579548)
- Testing
- Pegvaliase
- Where
- 4 sites in United States, Canada and 2 more
- Volunteers
- 50 people
- Sponsor
- BioMarin Pharmaceutical
Cystic Fibrosis
Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF)…
- Trial name
- NCT03633526
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Vertex Pharmaceuticals Incorporated