Clinical trials: Birth Defect, open to volunteers
Respiratory Distress Syndrome (RDS), Bronchopulmonary Dysplasia (BPD)
Can Betamethasone Given After Birth Help Extremely Premature Babies Come Off Breathing Support Safely and Effectively?
The goal of this study is to determine if giving a steroid medication (specifically, betamethasone) after birth can help extremely premature babies (born before 28 weeks) come off breathing machines safely and reduce their risk…
- Trial name
- NCT07447739
- Testing
- Betamethasone
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Khang Nguyen
Craniosynostoses, Hypovolemia
Hemodynamic Monitoring During Craniosynostosis Surgery: Comparing Traditional and Newer Technology Monitors (CRASY-PRAM)
Hemodynamic evaluation during pediatric anesthesia is essential to care management. Intraoperative cardiovascular instability is frequent in major surgeries, and appropriate monitoring is necessary to ensure safe anesthetic…
- Conditions
- Craniosynostoses, Hypovolemia, Hemorrhage, Hemodynamic Instability
- Trial name
- CRASY-PRAM (NCT06263075)
- Testing
- Intraoperative monitoring
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Tracheobronchomalacia, Excessive Dynamic Airway Collapse
Effect of CPAP on 6-Minute Walk Test Outcomes in Patients With ECAC
The purpose of this protocol is to perform a prospective, randomized, double-blinded, pacebo-controlled clinical trial to determine the influence of a non-invasive positive pressure ventilation device on exercise capacity and…
- Trial name
- NCT04217603
- Testing
- Continuous positive airway pressure (CPAP) device
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Beth Israel Deaconess Medical Center
Fabry Disease
Long Duration Holter ECG in Fabry Disease
The main objective is to assess the occurrence of cardiac arrhythmias and conduction disorders during a three-year follow-up using implantable Holter ECG monitoring in 40 patients with Fabry disease.
- Trial name
- NCT04440254
- Testing
- Implantation (subcutaneous) of a marketed miniaturized Holter ECG recording device
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Lumbar Spinal Stenosis, Cardiac Amyloidosis
Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis
Primary objective: To identify older adults with transthyretin cardiac amyloidosis (ATTR-CA) early in the course of the illness, at a time when disease modifying therapies are most effective.
- Conditions
- Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation
- Trial name
- NCT06034405
- Testing
- Tc99-PYP or Tc99-HDMP Scan
- Where
- 6 sites in United States
- Volunteers
- 1,663 people, healthy volunteers welcome
- Sponsor
- Columbia University
Cystic Fibrosis
Impact of a Coordinated Dietetic-adapted Physical Activity Program on the Percentage of Lean Body Mass in Adults With Cystic Fibrosis Treated With Elexacaftor-Tezacaftor-Ivacaftor: Multicentre Randomised Controlled Trial
Cystic fibrosis is an autosomal recessive inherited disease linked to various mutations in the gene coding for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, with respiratory and digestive disorders…
- Trial name
- DIAPASOM (NCT06457451)
- Testing
- DIAPASOM program
- Where
- 4 sites in France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Tours
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Retinopathy of Prematurity
Oral Propranolol for Prevention of Threshold Retinopathy of Prematurity
Extremely premature infants are at risk of developing a potentially blinding eye disease, called retinopathy of prematurity (ROP).
- Trial name
- RoProp (NCT03083431)
- Testing
- Propranolol
- Where
- 3 sites in Germany, Switzerland and 1 more
- Volunteers
- 276 people
- Sponsor
- University of Zurich
Duchenne Muscular Dystrophy (DMD)
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the…
- Trial name
- NCT07573631
- Testing
- BMN 351
- Where
- 6 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
AKI, CKD
Genetics in the Progression of Nephropathies
This study evaluates the role of genetic in the development and progression of different nephropaties with particular attention to: AKI CKD Hypertension ADPKD CKD-MBD Patients with decompensated heart failure undergoing either…
- Conditions
- AKI, CKD, Ckd-Mbd, CKD (Chronic Kidney Disease) Stage 5D
- Trial name
- NCT06416761
- Testing
- genetic polymorphisms
- Where
- Italy
- Volunteers
- 10,000 people
- Sponsor
- Ospedale San Raffaele
Dermatitis, Contact, Dermatitis
Post-Marketing Clinical Follow-Up Trial to Evaluate the Performance and Safety of the Medical Device 047 TD Dermatitis Cream in Adults and Children With Atopic and Contact Dermatitis Symptoms Confirmed by Clinical Parameters
The study aims to evaluate and confirm the performance of 047 TD Dermatitis cream in the improvement of eczematous dermatitis and contact dermatitis, throughout a reduction in disease severity and symptoms.
- Conditions
- Dermatitis, Contact, Dermatitis, Dermatitis, Chronic, Dermatitis, Eczematous
- Trial name
- NCT07056569
- Testing
- 047_TD Dermatitis cream twice a day for 28 days
- Where
- 3 sites in Italy
- Volunteers
- 90 people
- Sponsor
- Biokosmes Srl
Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradiation (TBI) in those diagnosed with Common Variable Immunodeficiency…
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome
- Trial name
- CVID/PIRD (NCT07284641)
- Testing
- Hematopoietic stem cell transplant (HSCT)
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Paul Szabolcs
NF1, Neurofibromatosis
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
This study is designed to establish the foundation for a new therapy for neurofibromatosis Type I (NF1)-related cutaneous neurofibromas (cNFs) by assessing the feasibility and efficacy of IMLYGIC in adults with NF1 and cNFs who…
- Conditions
- NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma
- Trial name
- NCT07102394
- Testing
- Imlygic
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Johns Hopkins University
Bronchopulmonary Dysplasia
Inhaled Ciclesonide Study in Preterm Infants
Our overall objective is to conduct a safety study with inhaled ciclesonide to evaluate known glucocorticoids (sGC)-related acute and intermediate toxic effects while measuring for the first time in neonates its systemic…
- Trial name
- NCT06589245
- Testing
- Alvesco Inhalant Product
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Venkatesh Sampath
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
N-acetylglutamate Synthase (NAGS) Deficiency
Orphan Europe Carbaglu® Surveillance Protocol
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information.
- Trial name
- NCT03409003
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicholas Ah Mew
Li-Fraumeni Syndrome, TP53 Gene Mutation
Li-Fraumeni & TP53 (LiFT UP): Understanding and Progress
The purpose of this research study is to learn more about variants in the TP53 gene both associated with Li-Fraumeni Syndrome (LFS), a hereditary cancer risk condition, and TP53 variants found in the blood for other reasons (e.g.
- Conditions
- Li-Fraumeni Syndrome, TP53 Gene Mutation, Hereditary Cancer Syndrome, Clonal Hematopoiesis
- Trial name
- LiFT_UP (NCT04541654)
- Testing
- Data and Specimen Collection
- Where
- 3 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- Dana-Farber Cancer Institute
Gut-Brain Disorders, GERD Without Erosive Esophagitis
Neuroregulators for the Treatment of Diseases Associated With Esophageal-brain-gut Axis Communication Abnormalities.
Gastroesophageal reflux disease (GERD) poses a challenging medical condition to manage, with up to 40% of patients showing refractory to standard medical intervention, which usually begins with a proton pump inhibitor (PPI).
- Trial name
- NCT06778824
- Testing
- Takepron, Zoloft 50Mg Tablet, Imipramine Pill
- Where
- Taiwan
- Volunteers
- 610 people
- Sponsor
- Hualien Tzu Chi General Hospital
Fetal Growth Retardation, Fetal Neurodevelopmental Disorder
Fetal Magnetic Resonance Imaging: Z-score Reference Ranges for a Large Patient Population in Normal Neuroimaging Measurement
This study aims to establish the z score reference ranges of fetal head circumference and other measurement of fetal measurements including biparietal diameter, fronto-occipital diameter, length of the corpus callosum, lateral…
- Trial name
- FMZSCORE (NCT07004283)
- Where
- China
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- Guangzhou Women and Children's Medical Center
Congenital Heart Disease, Cyanotic Congenital Heart Disease
Prenatal Maternal Mental Health and Neurodevelopment in Congenital Heart Disease
Congenital heart disease (CHD) is the leading cause of congenital malformations, representing 1% of live births. Progress in surgical care have led to the dramatic increase in the population of children and adults living with…
- Conditions
- Congenital Heart Disease, Cyanotic Congenital Heart Disease, d-Transposition of the Great Arteries, Hypoplastic Left Heart Syndrome
- Trial name
- Neuro-Moms (NCT06711666)
- Where
- 5 sites in France
- Volunteers
- 174 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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