Clinical trials: Preterm Neonate
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics
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 Distress Syndrome, Newborn, Premature Birth
SURFAXIN® Treatment for Prevention of Bronchopulmonary Dysplasia (BPD) in Very Low Birth Weight (VLBW) Infants.
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required…
- Conditions
- Respiratory Distress Syndrome, Newborn, Premature Birth, Bronchopulmonary Dysplasia
- Trial name
- NCT00215540
- Testing
- Lucinactant 175 mg/kg, Lucinactant 90 mg/kg
- Where
- United States
- Volunteers
- 136 people
- Sponsor
- Windtree Therapeutics
Neonates
NICU-TECH RM9L-RS Probe ME Feasibility Study
Use of a new ultrasound device designed for babies to look at the catheters in veins and arteries as well as the blood vessel itself.
- Trial name
- NCT01181596
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- GE Healthcare
Cystic Fibrosis
Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
- Trial name
- NCT00803205
- Testing
- Ataluren
- Where
- 38 sites in United States, Belgium and 9 more
- Volunteers
- 238 people
- Sponsor
- PTC Therapeutics
Respiratory Distress Syndrome
Trial To Assess The Safety And Tolerability Of Lucinactant For Inhalation In Preterm Neonates 26 to 28 Weeks PMA
This study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in up to four escalating doses to preterm neonates 26 to 28 weeks gestational age who are receiving nCPAP for RDS…
- Trial name
- NCT02528318
- Testing
- Lucinactant for inhalation, nCPAP alone
- Where
- 20 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Windtree Therapeutics
Preterm Birth
17OHP for Reduction of Neonatal Morbidity Due to Preterm Birth (PTB) in Twin and Triplet Pregnancies
Hypothesis: Among women with twin or triplet pregnancies, weekly injections of 17-alpha-hydroxyprogesterone caproate (17OHP), started before 24 weeks of gestation, will reduce neonatal morbidity by reducing the rate of preterm…
- Trial name
- 170HP (NCT00163020)
- Testing
- 17-alpha-hydroxyprogesterone caproate injectable
- Where
- 18 sites in United States
- Volunteers
- 321 people
- Sponsor
- Obstetrix Medical Group
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion (Part A)
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care (Intralipid) from 7 up to 90 days to develop Essential Fatty Acid…
- Trial name
- NCT04555044
- Testing
- Clinolipid, Intralipid
- Where
- 9 sites in United States
- Volunteers
- 101 people
- Sponsor
- Baxter Healthcare Corporation
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
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
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
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
Hemophilia A, Hemophilia B
Study of Ataluren (PTC124) in Hemophilia A and B
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting.
- Trial name
- NCT00947193
- Testing
- Ataluren
- Where
- 13 sites in United States, Canada and 2 more
- Volunteers
- 13 people
- Sponsor
- PTC Therapeutics
Respiratory Syncytial Virus Infections
Prospective Study for the Use of Palivizumab (Synagis®) in High-risk Children in Germany
The SYNAGIS Registry was carried out in order to gather comprehensive real-world data on the use of palivizumab in children at high risk for serious respiratory syncytial virus (RSV) disease.
- Trial name
- NCT01155193
- Volunteers
- 30,804 people
- Sponsor
- AbbVie (prior sponsor, Abbott)
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Bronchopulmonary Dysplasia
Inhaled Nitric Oxide (INO) for the Prevention of Bronchopulmonary Dysplasia (BPD) in Preterm Infants
This phase 3, multi-center, double blind, placebo-controlled, randomized clinical trial will attempt to demonstrate if preterm infants who require mechanical ventilation and/or positive pressure support at any point during days 5…
- Trial name
- NCT00931632
- Testing
- Inhaled Nitric Oxide
- Where
- 33 sites in United States, Canada
- Volunteers
- 451 people
- Sponsor
- Mallinckrodt
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
Respiratory Distress Syndrome (RDS)
A Study in Preterm Neonates With Respiratory Distress Syndrome (RDS) Comparing CUROSURF® Administration Through Less Invasive Surfactant Administration (LISA) and Conventional Administration
This study compared the administration of porcine surfactant (poractant alfa, Curosurf®) through a less invasive method (LISA), using a thin catheter, CHF 6440 (LISACATH®), during non-invasive ventilation (CPAP, NIPPV, BiPAP)…
- Trial name
- LISPAP (NCT02772081)
- Testing
- LISA combination product (Curosurf+catheter CHF6440), Curosurf through conventional administration (endotracheal tube)
- Where
- 26 sites in United States
- Volunteers
- 33 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Respiratory Distress Syndrome
Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates
The primary objective of this study is to evaluate the safety and tolerability of aerosolized surfactant, specifically lucinactant for inhalation, administered in escalating inhaled doses to preterm neonates 29 to 34 weeks…
- Trial name
- NCT02074059
- Testing
- Lucinactant for Inhalation, nCPAP alone
- Where
- 11 sites in United States
- Volunteers
- 80 people
- Sponsor
- Windtree Therapeutics
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