Clinical trials: Newborn Diseases
Hemophilia
Genetic Susceptibility to Factor VIII Inhibitors
This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII.
- Trial name
- NCT00344435
- Where
- Sweden
- Volunteers
- 1,187 people
- Sponsor
- National Cancer Institute (NCI)
Bronchopulmonary Dysplasia, Lung Diseases
Risk Factors in Bronchopulmonary Dysplasia (Newborn Lung Project)
To investigate risk factors in bronchopulmonary dysplasia (BPD) and to elucidate the relationship between BPD, acute lung disease severity, respiration-related variables, water balance, nutrition, familial predisposition, and…
- Conditions
- Bronchopulmonary Dysplasia, Lung Diseases, Respiratory Distress Syndrome
- Trial name
- NCT00005289
- Volunteers
- 2,202 people
- Sponsor
- University of Wisconsin, Madison
Atopic Dermatitis
Observational Study to Evaluate the Actual Use of Elidel® in Chinese Patients With Mild to Moderate Atopic Dermatitis
The primary objective of the present multicentre, prospective, non-interventional study (NIS) is gathering knowledge on the actual use and effectiveness of Elidel® in Chinese patients with mild to moderate AD affecting sensitive…
- Trial name
- NCT04976868
- Testing
- Pimecrolimus 1% Top Cream
- Where
- 10 sites in China
- Volunteers
- 130 people
- Sponsor
- Mylan Inc.
Solid Tumor (Excluding CNS), Liver Cell Carcinoma
Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells
This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety…
- Conditions
- Solid Tumor (Excluding CNS), Liver Cell Carcinoma, Malignant Rhabdoid Tumor, Yolk Sac Tumor
- Trial name
- IMPACT (NCT07148050)
- Testing
- SC-CAR.GPC3xIL15.21 CAR T cells
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Seattle Children's Hospital
Aging, Skin Abnormalities
Efficacy of Polypodium Leucotomos Extract Decreasing UVA Premutagenic and Photoaging Markers
With this study, the investigators will like to determine if taking a dose of the study medication, called Polypodium leucotomos (PL), prevents some of the changes in the skin caused by the adverse effects of UVA, a type of…
- Trial name
- NCT00520910
- Testing
- Polypodium leucotomos
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Miami
Transfusion Dependent Beta-Thalassemia
A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
This is a single-arm, open label, single-dose study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CD34+ Human Hematopoietic Stem Cells that was transduced…
- Trial name
- NCT05762510
- Testing
- GMCN-508B (LentiRed)
- Where
- China
- Volunteers
- 5 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Bronchopulmonary Dysplasia, Vitamin D Overdose
25OH Vitamin D Overdoses and Risk of Bronchopulmonary Dysplasia or Death
Several studies have demonstrated that vitamin D deficiency at birth is a risk factor of bronchopulmonary dysplasia. However, in an animal model of bronchopulmonary dysplasia vitamin D overdose has also been associated with an…
- Trial name
- 25OHVDORBPDD (NCT05944055)
- Testing
- Is 25 OH vitamin D overdose a risk factor of bronchopulmonary dysplasia or death ?
- Where
- France
- Volunteers
- 173 people
- Sponsor
- Hospices Civils de Lyon
Hemoglobinopathies, Myelodysplastic Syndromes
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome
- Trial name
- NCT00673608
- Testing
- deferasirox
- Where
- 4 sites in Australia
- Volunteers
- 118 people
- Sponsor
- Novartis
Vestibular Schwannoma, Meningioma
Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma
This will be a multi-center, proof of concept phase 0 study to assess the suppression of p-AKT in Vestibular Schwannoma (VS) and meningiomas by AR-42 in adult patients undergoing tumor resection.
- Conditions
- Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
- Trial name
- NCT02282917
- Testing
- AR-42
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts Eye and Ear Infirmary
Maxillary Sinusitis
Study to Measure the Impact of Antibiotics on Bacterial Flora in Adults With Acute Sinusitis
The purpose of this study is to measure the speed of microbial eradication due to azithromycin or telithromycin in acute maxillary sinusitis (AMS).
- Trial name
- NCT00245440
- Testing
- Azithromycin, Telithromycin
- Where
- United States
- Volunteers
- 192 people
- Sponsor
- CPL Associates
Lafora Disease
A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease
This study will test the safety and efficacy of multiple doses of ION283 administered as intrathecal (IT) injections by lumbar puncture (LP). All subjects will receive ION283.
- Trial name
- Lafora (NCT06609889)
- Testing
- ION283
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Berge Minassian
ATTR Amyloidosis Wild Type, ATTR Gene Mutation
ATTR Cardiac Amyloidosis in a Selected Population
Patients operated for carpal tunnel syndrome (CTS) at Ålesund Hospital (200 patients) will have a biopsy to diagnose ATTR amyloidosis. Patients with positive biopsy will be examined to decide wether they have cardiac amyloidosis.
- Conditions
- ATTR Amyloidosis Wild Type, ATTR Gene Mutation, Carpal Tunnel Syndrome, Cardiac Amyloidosis
- Trial name
- NCT06458374
- Where
- Norway
- Volunteers
- 180 people
- Sponsor
- Helse Møre og Romsdal HF
Bicuspid Aortic Valve (BAV), Aortic Aneurysm
Research on Aneurysm Growth Prediction in Vascular Dilation Caused by Bicuspid Aortic Valve Based on VDM and CFD
Bicuspid aortic valve (BAV) is the most common congenital valvular malformation, characterized by heterogeneous phenotypic subtypes that predispose patients to secondary aortic pathologies, including valvular dysfunction and…
- Conditions
- Bicuspid Aortic Valve (BAV), Aortic Aneurysm, TAVR
- Trial name
- NCT07782918
- Testing
- Transcatheter Aortic Valve Replacement
- Where
- 2 sites in China
- Volunteers
- 1,000 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Neonatal Sepsis
Clinical Value of Metagenomic Sequencing in Neonatal Sepsis
This study aimed to enroll 2000 neonatal patients with suspected sepsis or clinical diagnosed sepsis. These patients will undergo both conventional methods and metagenomics sequencing to detect the pathogenic microorganisms of…
- Trial name
- NCT05416918
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Children's Hospital of Fudan University
Cystic Fibrosis, Chronic Pancreatitis
Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis…
- Trial name
- NCT05069597
- Testing
- CREON
- Where
- 21 sites in United States
- Volunteers
- 30 people
- Sponsor
- AbbVie
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
Neonatal Screening, Kernicterus
Bilistick Point-of-care System 2.0 Bilirubin Validation
This is a validation study involving the Bilistick System 2.0 point-of-care bilirubin measuring device. The validation will be conducted by comparing bilirubin measurements utilizing the standard-of-care blood sample collected…
- Conditions
- Neonatal Screening, Kernicterus, Newborn Jaundice, Point-of-Care Testing
- Trial name
- NCT06058910
- Testing
- Bilistick System 2.0 point-of-care testing device
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Kettering Health Network
Aortic Valve Disease
Podocan and Wnt Pathway in the Development of Aortopathy in Bicuspid Valve Disease
Bicuspid Aortic Valve (BAV) disease is a common cardiac anomaly that is associated with valvular abnormalities, both stenosis and regurgitation, and aortopathy.
- Trial name
- NCT02806310
- Testing
- Blood draw
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Bassett Healthcare
Nurse's Role, Infantile Colic
Interventional Practices and Their Effectiveness in Infants With İnfantyl Colic
Infantile colic is a developmental disorder that reduces comfort levels in infants with intense crying crises and has negative effects on the infant and family.
- Trial name
- NCT06299358
- Testing
- Foot massage, Mother heart sound, Foot massage and maternal heart sound
- Where
- Turkey (Türkiye)
- Volunteers
- 90 people
- Sponsor
- Karamanoğlu Mehmetbey University
Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
A Study to Evaluate S241656 Alone or in Combination in Participants With Selected Myeloid Malignancies
The objective of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of S241656 and to determine the recommended dose for expansion (RDE) of S241656 in participants with relapsed/refractory (R/R) acute…
- Conditions
- Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome Acute Myeloid Leukemia
- Trial name
- NCT07719348
- Testing
- S241656, Posaconazole
- Volunteers
- 64 people
- Sponsor
- Servier
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