Clinical trials: Infant, Newborn, Disease
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance…
- Trial name
- NCT02516410
- Testing
- VX-661 plus ivacaftor combination, Ivacaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 168 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
Respiratory Syncytial Virus Infection, Premature Birth
Palivizumab for Prevention of Severe Respiratory Syncytial Virus Infection in Russian Children
100 Russian children of 2 years of age and less in high-risk populations (preterm, and/or with heart and lung problems) will receive palivizumab (Synagis) 15 mg/kg intramuscularly as prophylaxis to severe respiratory syncytial…
- Conditions
- Respiratory Syncytial Virus Infection, Premature Birth, Bronchopulmonary Dysplasia, Congenital Heart Disease
- Trial name
- NCT01006629
- Testing
- palivizumab
- Where
- 19 sites in Russia
- Volunteers
- 100 people
- Sponsor
- Abbott
Bronchopulmonary Dysplasia
129Xe MRI in Pediatric Population With BPD
Hyperpolarized (HP) gas magnetic resonance imaging (MRI) of the lungs offers additional information that cannot be obtained with CT scan, the current gold standard for imaging this disorder.
- Trial name
- NCT04035629
- Testing
- MagniXene, hyperpolarized 129Xe MRI
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Xemed LLC
Bronchopulmonary Dysplasia
Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature Infants
The objective of this study is to evaluate the efficacy and safety of a single intratracheal administration of Pneumostem® for treatment of Bronchopulmonary Dysplasia (BPD) in high-risk premature infants by comparing…
- Trial name
- NCT01828957
- Testing
- Pneumostem®, Normal Saline
- Where
- 2 sites in South Korea
- Volunteers
- 69 people
- Sponsor
- Medipost Co Ltd.
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
Healthy Volunteers, Atopic Dermatitis
A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome
The study is divided in 3 parts, starting with the safety assessment of BPR277 ointment in Healthy volunteers (Part 1). If found to be well tolerated in Part 1, BPR277 ointment will be assessed in two different patients groups to…
- Conditions
- Healthy Volunteers, Atopic Dermatitis, Netherton Syndrome
- Trial name
- 3 (NCT01428297)
- Testing
- BPR277 ointment (controlled application), BPR277 ointment, BPR277
- Where
- 4 sites in United States, Netherlands
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis (CF)
Safety, Tolerability and Pharmacokinetics Study of AIR DNAse Administered by Inhalation to Healthy Adult Volunteers
This study will be performed to assess the safety, tolerability and PK of escalating inhaled AIR-DNase doses administered as a single dose followed by multiple doses for 5 consecutive days in healthy subjects.
- Trial name
- NCT02605590
- Testing
- AIR DNase
- Where
- Israel
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Protalix
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
Low-Birth-Weight Infant, Premature Infants
Analyzer of Human Milk: Applicability in Clinical Practice in Order to Achieve the Best Nutrition for Infants
An observational and prospective study involving about 30 healthy lactating mothers whose preterm infant(s) is/are hospitalized at Neonatal Intensive Care Unit after childbirth and who can provide enough maternal milk (3mL) to be…
- Trial name
- MISTU (NCT02971904)
- Testing
- human milk collection
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Danone Early Life Nutrition Brazil
Cystic Fibrosis
MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT01180634
- Testing
- Aeroquin
- Where
- 112 sites in United States, Australia and 3 more
- Volunteers
- 330 people
- Sponsor
- Amgen
Retinopathy of Prematurity
Study to Assess the Efficacy, Safety, and Tolerability of Intravitreal Aflibercept Compared to Laser Photocoagulation in Patients With Retinopathy of Prematurity
The primary objective of the study is to assess the efficacy of aflibercept compared to laser in patients diagnosed with retinopathy of prematurity (ROP).
- Trial name
- BUTTERFLEYE (NCT04101721)
- Testing
- aflibercept, laser photocoagulation
- Where
- 52 sites in United States, Bulgaria and 11 more
- Volunteers
- 127 people
- Sponsor
- Regeneron Pharmaceuticals
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
Infantile Colic
An Infant Formula Trial on Dietary Management of Infantile Colic
A randomised, double blind, controlled, multi-centre study to assess the efficacy of an infant formula in the dietary management of infantile colic.
- Trial name
- NCT03329222
- Testing
- Specific hydrolysed proteins, Standard cow's milk with prebiotics mixture
- Where
- 2 sites in Singapore, Thailand
- Volunteers
- 5 people
- Sponsor
- Danone Asia Pacific Holdings Pte, Ltd.
Asphyxia Neonatorum
Biomarkers And Neurological Outcome in Neonates 2
Follow-up of participants of BANON1 study at age of at least 2 years. BANON1 (ClinicalTrials.gov ID: NCT03357250): Validation of biomarkers based on 24 months neuro-developmental outcome data in a human population for their…
- Trial name
- BANON2 (NCT04714775)
- Where
- 13 sites in Germany, Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- InfanDx AG
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
Intracranial Aneurysm
Diversion-p64 Post Market Clinical Follow-Up Study to Assess Safety and Effectiveness of the p64 Flow Modulation Device
The purpose of this study is to assess safety and effectiveness of the p64 Flow Modulation Device.
- Trial name
- NCT02600364
- Where
- 26 sites in Argentina, Belgium and 8 more
- Volunteers
- 450 people
- Sponsor
- Phenox GmbH
Epilepsy, Benign Neonatal
Efficacy and Safety of Intravenous Phenobarbital in Neonatal Seizures
This is a randomized, double-blind, parallel-group, Phase 3 study to evaluate the efficacy of the administration of phenobarbital sodium injection in neonates who have suffered from electrographic or electroclinical seizure.
- Trial name
- NCT04320940
- Testing
- Phenobarbital Sodium Injection
- Where
- 8 sites in United States, Jordan
- Volunteers
- 4 people
- Sponsor
- NEMA Research, Inc.
Retinopathy of Prematurity
Real World Study of the Effectiveness and Safety of Conbercept Ophthalmic Injection in the Treatment of Retinopathy of Prematurity - Multicenter, Retrospective and Observational Study Based on Real World Data
This study is a retrospective, multi-center real world study. The real world data comes from the electronic medical record system and disease database of the research centers .The patient's demographic information, disease…
- Trial name
- NCT04985448
- Where
- 2 sites in China
- Volunteers
- 1,000 people
- Sponsor
- Chengdu Kanghong Biotech Co., Ltd.
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.