Clinical trials: Newborn Diseases
Congenital Heart Diseases
Ulinastatin Injection in in Pediatric Patients Undergoing Open Heart Surgery
1. Explore the efficacy of Ulinastatin use in improving post-operative pulmonary insufficiency and safety in pediatric patients undergoing scheduled CPB open heart surgery to treat Complex Congenital Heart Disease 2.
- Trial name
- NCT02527811
- Testing
- Ulinastatin
- Where
- 5 sites in China
- Volunteers
- 450 people
- Sponsor
- Techpool Bio-Pharma Co., Ltd.
Pain, Sickle Cell Disease
Intranasal Fentanyl Versus Intravenous Morphine in the Treatment of Severe Painful Sickle Cell Crises in Children
Sickle cell anaemia is an inherited blood disorder which results in abnormal sickle shaped red blood cells which do not fit well through small blood vessels.
- Trial name
- NCT03682211
- Testing
- Fentanyl Citrate, Morphine sulphate
- Where
- Ireland
- Volunteers
- 31 people
- Sponsor
- University College Dublin
Machado-Joseph Disease / Spinocerebellar Ataxia 3
Study To Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta in Patients With Machado-Joseph Disease
This is an exploratory, randomized, parallel-group, dose escalation and dose-controlled study without a placebo arm. Eligible patients will be randomized in a 1:1 ratio (double-blind) to receive Cabaletta in 2 doses, once weekly…
- Trial name
- NCT02147886
- Testing
- Cabaletta for IV infusion once weekly during 24 weeks
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Bioblast Pharma Ltd.
Neurofibromatosis Type 1
Molecular Aspects of Preimplantation Genetic Diagnosis for NF1
Neurofibromatosis 1 (NF1) is a multisystem disorders characterized by skin abnormalities, such as café-au-lait spots and neurofibromas, learning disabilities, skeletal anomalies and vascular complications.
- Trial name
- NF1 (NCT05149469)
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Strasbourg, France
Cystic Fibrosis
A Study Comparing Continuous Infusion Antibiotics to Standard Treatment for Lung Infections in Cystic Fibrosis
Cystic fibrosis (CF) is an inherited disorder which results in increased thickness of secretions, especially in the lungs. By adulthood, the majority of patients with CF will have a bacteria living in their lungs, called…
- Trial name
- CISTIC (NCT01667094)
- Testing
- Intermittent, short infusion Ceftazidime, Continuous infusion Ceftazidime, Intermittent, short infusion Meropenem
- Where
- Australia
- Volunteers
- 50 people
- Sponsor
- The Alfred
Childhood Atopic Dermatitis
Knowledge Base in the Treatment of Atopic Dermatitis Among Thai Pediatricians
The purpose of this study is to determine knowledge base in diagnosis, investigation and treatment of pediatric atopic dermatitis among thai pediatricians.
- Trial name
- NCT01069666
- Where
- Thailand
- Sponsor
- Siriraj Hospital
Alpha Thalassemia
Genetics of Alpha Thalassemia in Israeli Ethnic Groups
Alpha thalassemia causes mild anemia and is found in many ethnic groups. Usually it is found in regions where malaria is endemic.
- Trial name
- NCT00159029
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Deborah Rund
Hemophilia B
Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea. Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.
- Trial name
- NCT02922231
- Testing
- RIXUBIS
- Where
- 5 sites in South Korea
- Volunteers
- 57 people
- Sponsor
- Baxalta now part of Shire
Thalassemia Major
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
- Trial name
- NCT01740531
- Testing
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Where
- 3 sites in Italy, Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Cerus Corporation
Nose Deformities, Acquired
Objectives and Measures Dimensions Nasal Resistance of Preterm and Term (MODERN)
During the resuscitation of preterm infants, withdrawal of non-invasive ventilation is difficult. In a recent study in children gestational age <30 weeks of amenorrhea (SA), definitive withdrawal rate from the first attempt to…
- Trial name
- MODERN (NCT02815345)
- Testing
- Rhinometry
- Where
- France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Biotinidase Deficiency
Study of ORL-1B in Patients With Biotinidase Deficiency
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
- Trial name
- NCT03269045
- Testing
- ORL-1B
- Volunteers
- 20 people
- Sponsor
- Orpha Labs
Cystic Fibrosis
Anti-IL5 and Other Biotherapies in Cystic Fibrosis
Our project is to describe retrospectively and prospectively CF patients treated with biotherapy in French CF centers. Main objective: To describe the clinical and paraclinical course of CF patients before and after treatment…
- Trial name
- MAB-CF (NCT04256772)
- Where
- France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Montpellier
ADPKD
Mineralocorticoid Antagonism and Endothelial Dysfunction in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The proposed research will determine the effectiveness of blocking aldosterone for improving the health and function of arteries in patients with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT01853553
- Testing
- Spironolactone, Sugar pill
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- University of Colorado, Denver
Cystic Fibrosis
Amiloride Solution and Tobramycin Solution for Inhalation for the Eradication of Burkholderia Dolosa in Patients With Cystic Fibrosis
The purpose of this research study is to determine if multiple doses of two inhaled drugs will help Cystic Fibrosis patients whose lungs are infected with a bacteria called Burkholderia dolosa.
- Trial name
- NCT00547053
- Testing
- Amiloride Solution for Inhalation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Boston Children's Hospital
Neonatal Respiratory Distress Syndrome
To Investigate the Effect of Pulmonary Surfactant With Different Inspired Oxygen Concentrations on the Clinical Outcomes of Very Preterm Infants Under Non-invasive Assisted Ventilation
A prospective multicenter randomized controlled study was conducted. Eligible very preterm infants with a gestational age of <32 weeks were enrolled.
- Trial name
- NCT07235345
- Testing
- According to the FiO2 threshold of PS application corresponding to randomization, PS treatment was given when FiO2 reached the threshold
- Where
- China
- Volunteers
- 500 people
- Sponsor
- Xu Falin
Huntington's Disease
A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
This is a multicenter, multinational, randomized, parallel-group, double-blind, placebo-controlled, dose range finding study to compare the efficacy and safety of different doses of pridopidine versus placebo in the treatment of…
- Trial name
- NCT02006472
- Testing
- Pridopidine
- Where
- 58 sites in United States, Australia and 10 more
- Volunteers
- 408 people
- Sponsor
- Prilenia
Stargardt Disease, Stargardt Macular Degeneration
A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
- Trial name
- ASTRA (NCT06942572)
- Testing
- SB-007
- Where
- 5 sites in United States
- Volunteers
- 57 people
- Sponsor
- Splice Bio
Rett Syndrome
Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome
Using a tele-research approach, we will recruit, enroll, guide and support carers and participants to engage in computer based activities (modified virtual reality) with the primary outcome of reducing stereotypies and increasing…
- Trial name
- NCT05012475
- Testing
- Modified Virtual Reality Gaming
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Georgetown University
Alpha 1-Antitrypsin Deficiency, Genetic Disease
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. The purpose of this study is to examine the density of the lung as measured by chest computed tomography (CT) and determine if…
- Trial name
- A1BC (NCT05297812)
- Where
- 9 sites in United States
- Volunteers
- 286 people
- Sponsor
- Columbia University
Infant ALL, Skin
Skin Condition of Term Newborns During Diaper Care for Meconium Removal
The aim of this study was to determine the effect of two different skin cleansing cotton wipes on skin condition during meconium removal diaper care in term newborns.
- Trial name
- NCT07072130
- Testing
- Control group (wet cotton), Experimental group (baby oil-impregnated cotton)
- Where
- Turkey (Türkiye)
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Acibadem University
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.