Clinical trials: Newborn Infant
GERD
Efficacy and Safety of Esomeprazole Once Daily for the Treatment of GERD in Neonatal Patients
The purpose of this study is to assess the difference between esomeprazole and placebo in the treatment of signs and symptoms as observed by 8-hour video and cardiorespiratory monitoring in neonatal patients.
- Trial name
- NCT00427635
- Testing
- Esomeprazole
- Where
- 3 sites in Australia, Germany and 1 more
- Volunteers
- 32 people
- Sponsor
- AstraZeneca
Cystic Fibrosis
A Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF…
- Trial name
- NCT03045523
- Testing
- GLPG2222 150 mg q.d., GLPG2222 300 mg q.d.
- Where
- 22 sites in Australia, Belgium and 4 more
- Volunteers
- 37 people
- Sponsor
- Lakefront Biotherapeutics NV
Severe Combined Immunodeficiency
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe combined immunodeficiency (SCID). The donor cells are exposed to a protein that has been shown in the laboratory to improve the ability…
- Trial name
- NCT02860559
- Testing
- TBX-1400
- Where
- 2 sites in Israel
- Volunteers
- 8 people
- Sponsor
- Taiga Biotechnologies, Inc.
Cystic Fibrosis
Trial to Assess the Safety, Tolerability and Pharmacokinetics of GS-9411 in Healthy Male Volunteers
The purpose of this study is to evaluate the safety and tolerability of escalating doses of GS-9411 in healthy male volunteers. GS-9411 is a sodium channel inhibitor, that may restore airway hydration and mucociliary clearance in…
- Trial name
- NCT00800579
- Testing
- GS-9411
- Where
- Australia
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Catheter, Newborn, Infant, Disease
Evaluation of Intravascular Monitoring of Partial PRessure of Oxygen for Neonatal intensiVE Care Patients -2
The study is a non-blinded trial with one single intervention in sick newborn infants. The Neosense umbilical catheter will be used instead of the routinely used umbilical catheter on infants where the treating physician has…
- Conditions
- Catheter, Newborn, Infant, Disease, Lung Diseases
- Trial name
- IMPROVE-2 (NCT05433779)
- Testing
- Neosense Umbilical Catheter
- Where
- 5 sites in Sweden
- Volunteers
- 8 people
- Sponsor
- Neosense Technologies
Cystic Fibrosis
Study of Ataluren (PTC124) in Cystic Fibrosis
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study…
- Trial name
- NCT02107859
- Testing
- Ataluren
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 61 people
- Sponsor
- PTC Therapeutics
Phenylalanine Hydroxylase Deficiencies
Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive to BH4.
- Trial name
- NCT00432822
- Testing
- tetrahydrobiopterin (BH4)
- Volunteers
- 50 people
- Sponsor
- Orphanetics Pharma Entwicklungs GmbH
Cystic Fibrosis
A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis
This is a Phase 2, randomized, double-blind, placebo- and active-controlled, parallel group, multicenter study to evaluate the safety, tolerability, and efficacy of VX-440 in dual and triple combination with tezacaftor (TEZ…
- Trial name
- NCT02951182
- Testing
- TEZ, IVA, VX-440
- Where
- 40 sites in United States, Australia and 8 more
- Volunteers
- 74 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Formula Feeding of Healthy Full Term Infants, Breast Feeding of Healthy Full Term Infants
Impact of Infant Feeding on Newborn Metabolomic Profile
The purpose of this study is the evaluation with untargeted modalities, of the urine metabolomic profile in a group of infants fed with a standard formula, a group of infants fed with a formula supplemented with functional…
- Trial name
- NCT01606683
- Testing
- Infant formula with GOS, beta-palmitate, acidified milk, Standard infant formula without functional ingredients, No intervention
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- Heinz Italia SpA
Persistent Fetal Circulation Syndrome
Sildenofil in Persistent Pulmonary Hypertension in Newborns
This study hopes to evaluate the effectiveness of early combined use of Sildenafil and nitric oxide (iNO) in newborns with Persistent pulmonary hypertension (PPHN) and or hypoxemic respiratory failure and assess whether this…
- Trial name
- Sildeno (NCT01558466)
- Testing
- Sildenafil, diluent
- Where
- Qatar
- Volunteers
- 100 people
- Sponsor
- Hamad Medical Corporation
Cystic Fibrosis
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
- Trial name
- KONTINUE (NCT01707290)
- Testing
- Ivacaftor
- Where
- 38 sites in United States, Belgium and 2 more
- Volunteers
- 125 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are <24…
- Trial name
- NCT03277196
- Testing
- IVA
- Where
- 29 sites in United States, Australia and 4 more
- Volunteers
- 86 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide…
- Trial name
- NCT06079398
- Testing
- Navepegritide
- Where
- 18 sites in United States, Australia and 13 more
- Volunteers
- 72 people
- Sponsor
- Ascendis Pharma A/S
Cystic Fibrosis, Pulmonary
Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single doses of ARO-ENaC in healthy adult volunteers; and to evaluate the safety, tolerability, PK and efficacy of multiple doses of…
- Trial name
- NCT04375514
- Testing
- ARO-ENaC
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome
Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures
This study will be conducted to evaluate the safety, pharmacokinetics (PK), and efficacy of adjunctive GWP42003-P in participants < 2 years of age with tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS), or Dravet…
- Conditions
- Seizure in Participants With Tuberous Sclerosis Complex, Seizure in Participants With Dravet Syndrome, Seizure in Participants With Lennox-Gastaut Syndrome
- Trial name
- NCT04485104
- Testing
- GWP42003-P
- Where
- 11 sites in United States, Italy and 1 more
- Volunteers
- 3 people
- Sponsor
- Jazz Pharmaceuticals
Cystic Fibrosis, Failure to Thrive
Clinical Study With an Enteral Formula With Symbiotic and DHA for Malnourished Children
The purpose of this study is to determine whether a nutritional supplement is effective in the treatment of malnutrition in pediatric patients with failure to thrive or cystic fibrosis.
- Conditions
- Cystic Fibrosis, Failure to Thrive, Malnutrition
- Trial name
- VITJUNIOR (NCT02128984)
- Testing
- Symbiotic Formula with DHA and antioxidants, Standard Formula
- Where
- 8 sites in Peru, Spain
- Volunteers
- 109 people
- Sponsor
- Laboratorios Ordesa
Cystic Fibrosis
Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.
- Trial name
- NCT01262352
- Testing
- Ivacaftor
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 21 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Neonatal Respiratory Distress Syndrome
A Study To InvestigateThe Safety, Tolerability And Efficacy Of Nebulised Curosurf® In Preterm Neonates With Respiratory Distress Syndrome (RDS)
The present study will mainly aim at investigating the safety, tolerability and efficacy of different escalating single doses administration of nebulised Curosurf®, in preterm neonates with RDS (Respiratory Distress Syndrome)…
- Trial name
- NCT03235986
- Testing
- nCPAP (nasal Continuous Positive Airway Pressure) + Nebulised Curosurf® (Part I), nCPAP (nasal Continuous Positive Airway Pressure) + Nebulised Curosurf® (Part II), nCPAP (nasal Continuous Positive Airway Pressure) alone (Part I)
- Where
- Italy
- Volunteers
- 166 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Sepsis Newborn, Malaria
Immune Dysfunction in Newborn Sepsis
The aim of the project is to study neonatal immune dysfunction associated to the risk of newborn sepsis in a malaria endemic area in Benin.
- Conditions
- Sepsis Newborn, Malaria, Immune Responses
- Trial name
- RECIPAL (NCT03780712)
- Testing
- Non applicable
- Volunteers
- 585 people
- Sponsor
- BioMérieux
Infantile Colic
Effect of Probiotic Limosilactobacillus Reuteri (L. Reuteri) on Crying and Fussing Time in Infants With Colic
This is a double-blind, randomized, placebo-controlled, parallel-group study in infants with colic with the primary objective to evaluate crying and fussing time.
- Trial name
- NCT06462651
- Testing
- L. reuteri
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- BioGaia AB