Clinical trials: Congenital Malformations
Bronchopulmonary Dysplasia, Gastroesophageal Reflux
Transpyloric Versus Gastric Feeding in Bronchopulmonary Dysplasia
The goal of this clinical trial is to learn if transpyloric tube feeding (feeding directly into the small intestine) versus gastric tube feeding tolerably and effectively reduces gastroesophageal reflux in infants born premature…
- Trial name
- NCT06534359
- Testing
- Transpyloric tube feeding, Gastric tube feeding
- Where
- 4 sites in United States
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Philadelphia
Marfan Syndrome
Biological Collection for Marfan and Related Syndromes
The present study will establish a collection of biological samples from Marfan patients or with associated diseases to be used for research purposes only, with due respect for confidentiality.
- Trial name
- MARFANS (NCT04970459)
- Testing
- collection of samples of blood and urine
- Where
- France
- Volunteers
- 300 people
- Sponsor
- University Hospital, Toulouse
Cystic Fibrosis
Small Circulating RNA as Molecular Markers of Lung Disease in Cystic Fibrosis
The aim of our study is to assess miRNAs expression profiles in the circuling blood of patients with cystic fibrosis and highlight "signatures" that could reflect the pulmonary status of patients
- Trial name
- MIRDIAMUCO (NCT02992080)
- Testing
- miRNAs isolation from blood samples of patients and control
- Where
- 2 sites in France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Colorectal Cancer, Colon Cancer
CB-839 + Capecitabine in Solid Tumors and Fluoropyrimidine Resistant PIK3CA Mutant Colorectal Cancer
This study has two portions. The main goal of the Phase I portion of this research study is to see what doses of CB-839 and capecitabine can safely be given to patients without having too many side effects.
- Conditions
- Colorectal Cancer, Colon Cancer, Rectal Cancer, Solid Tumor
- Trial name
- NCT02861300
- Testing
- CB-839, Capecitabine
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- David Bajor, MD
Long QT Syndrome, Sleep Apnea
Long QT Syndrome and Sleep Apnea
Obstructive sleep apnea (OSA) has been associated with cardiac repolarization abnormalities and implicated in sudden cardiac death.
- Trial name
- NCT03678311
- Testing
- Continuous Positive Airway Pressure (CPAP)
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- The Cleveland Clinic
Phenylketonuria
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
The primary purpose of this study is to assess the safety and tolerability of AG-181 in subjects with Phenylketonuria (PKU).
- Trial name
- NCT07241234
- Testing
- AG-181
- Where
- 5 sites in United States, Poland
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Maxillofacial Trauma, Face Cancer
Evaluation of Using 3D Printed PEEK Facial Implants in Repairing Maxillofacial Deformities
Objective: Evaluation of using 3D printed polyether ether ketone facial implants in repairing maxillofacial deformities. Research materials and methods: The research sample consisted of 10 patients with facial deformities…
- Conditions
- Maxillofacial Trauma, Face Cancer, Mucormycosis
- Trial name
- NCT05348434
- Testing
- 3D printed polyether ether ketone facial implants in repairing maxillofacial deformities
- Where
- Syria
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Latakia University
Heart Defects, Congenital, Hyperglycemia
SPECS: Safe Pediatric Euglycemia in Cardiac Surgery
Critically ill children, including children undergoing heart surgery, commonly develop elevated blood glucose (also known as "blood sugar") levels during their illness, which can lead to poor health outcomes and an increased risk…
- Trial name
- SPECS (NCT00443599)
- Testing
- Insulin, Usual Care
- Where
- 2 sites in United States
- Volunteers
- 989 people
- Sponsor
- Boston Children's Hospital
Postural Orthostatic Tachycardia Syndrome, Acute Hepatic Porphyria
Evaluating the Prevalence of Acute Hepatic Porphyria in Postural Tachycardia Syndrome
Postural Tachycardia Syndrome (POTS) is the most common autonomic disorder and is estimated to affect 3,000,000 individuals in the United States, with 80-85% of patients being women.
- Trial name
- NCT05344599
- Testing
- Autonomic Function Testing, Genetic Testing, Urine Testing
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Vanderbilt University Medical Center
X-Linked Adrenoleukodystrophy
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice.
- Trial name
- NCT03278899
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- NeuroVia, Inc.
Functional Gastrointestinal Disorders, Infant Conditions
Prevalence of FGIDs and Probiotics Study
This is a study to evaluate the prevalence of FGIDs in infants using the Thai version of Rome IV diagnostic questionnaire for functional gastrointestinal disorders in infants and evaluate the efficacy of Limosilactobacillus…
- Trial name
- PROOF (NCT06309199)
- Testing
- Biogaia
- Where
- Thailand
- Volunteers
- 512 people, healthy volunteers welcome
- Sponsor
- Chulalongkorn University
Vaso-Occlusive Crises, Sickle Cell Disease
Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease
Sickle cell disease is a severe monogenic genetic disorder caused by an autosomal recessive mutation of the β-globin gene, leading to production of abnormal hemoglobin (HbS).
- Trial name
- PROSTASICKLE (NCT07498309)
- Testing
- Iloprost
- Where
- France
- Volunteers
- 144 people
- Sponsor
- University Hospital, Rouen
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
Sickle Cell Anemia
Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis
People who have Sickle Cell Anemia (HbSS) produce red blood cells with shorter lifespans. These red blood cells breakdown faster, and this is called hemolysis.
- Trial name
- NCT01848691
- Testing
- Measurement of carbon monoxide level in exhaled breath
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Ashutosh Lal
Chromosome Aberrations
The Relationship Between Fetal Membrane Thickness and Fetal Chromosomal Aneuploidies
This observational study aims to recruit pregnant women between 18 to 24 weeks of gestation to investigate the relationship between amniotic membrane thickness and fetal chromosomal abnormalities.
- Trial name
- PLACE2102 (NCT06141213)
- Testing
- Ultrasound screening
- Where
- China
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- Tianjin Central Hospital of Gynecology Obstetrics
Fabry Disease
Lipidomics and Functional Analyses of Platelets in Fabry Disease
This study aims to evaluate whether platelets are biochemically and functionally altered in Fabry disease (FD) and therefore possibly implicated in FD manifestations such as cerebrovascular events.
- Trial name
- NCT02649660
- Where
- 2 sites in Switzerland
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Spital Linth
Solid Tumor
Artificial Intelligence to Search for Abnormalities in Ambulatory Cancer Patients
During treatment, cancer patients may experience side effects related to their disease but also to the different treatments they receive.
- Trial name
- IASAAC (NCT05412420)
- Testing
- Patient Self-Reporting of Symptoms
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Institut de Cancérologie de Lorraine
Molar Incisor Hypomineralization
Prevalence of Molar Incisor Hypomineralization Among a Group of Egyptian Children in Fayoum Governorate Schools
Determining the Prevalence of Molar Incisor Hypomineralization Among a Group of Egyptian Children in Fayoum Governorate Schools via clinical examination.
- Trial name
- NCT04420520
- Volunteers
- 135 people
- Sponsor
- Cairo University
Atopic Dermatitis
A Study to Evaluate Adverse Events and Change in Disease Activity Comparing Oral Upadacitinib to Subcutaneous Dupilumab in Adolescent and Adult Participants With Moderate to Severe Atopic Dermatitis
Atopic dermatitis (AD) is a skin condition that may cause a rash and itching due to inflammation of the skin. Therapies spread over the skin may not be enough to control the AD in trial participants who require systemic…
- Trial name
- Level Up (NCT05601882)
- Testing
- Dupilumab, Upadacitinib
- Where
- 257 sites in United States, Australia and 27 more
- Volunteers
- 920 people
- Sponsor
- AbbVie
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring