Clinical trials: Birth Defect, open to volunteers
Orthognathic Surgical Procedures, Psychiatric Disorders
Incidence of Body Dysmorphic Disorder (BDD) in a Population of Patients Undergoing Orthognathic Surgery
Assessment of the incidence of Body dismorphic disorder in a population candidated to orthognathic surgery
- Conditions
- Orthognathic Surgical Procedures, Psychiatric Disorders, Prognathism
- Trial name
- IBDOS (NCT06879275)
- Testing
- Questionnaire and Physical Exam
- Where
- Italy
- Volunteers
- 90 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Multicystic Dysplastic Kidney, Polycystic Kidney Disease
Amnioinfusion for Fetal Renal Failure
The goal of this clinical trial is to learn if serial amnioinfusions can improve the chances of survival for fetuses with severe kidney problems that cause low amniotic fluid (anhydramnios).
- Conditions
- Multicystic Dysplastic Kidney, Polycystic Kidney Disease, Fetal Renal Anomaly, Anhydramnios
- Trial name
- NCT06728228
- Testing
- Amnioinfusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Fetal Care Center, PLLC
Lipoprotein Lipase Deficiency
Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency
Lipoprotein lipase (LPL) is an enzyme that plays an important role in removing triglycerides (TG) (molecules that transport dietary fat) from the blood.
- Trial name
- AGL12 (NCT04227678)
- Testing
- Heparin, liquid meal
- Where
- Canada
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Université de Sherbrooke
Patency of the Ductus Arteriosus Acetaminophen Extreme Prematurity
Long Term Follow-up of the TREOCAPA Study (TREOCAPA-LT)
The ductus arteriosus (DA) is a large channel connecting the main pulmonary trunk with the descending aorta. In extremely preterm infants, the DA frequently fails to close and this results in a condition called patent ductus…
- Trial name
- TREOCAPA-LT (NCT06064825)
- Testing
- 2 year follow-up of neurodevelopment using a parental questionnaire
- Where
- 8 sites in France
- Volunteers
- 500 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Heart Defects, Congenital, Cardiovascular Disease Other
Congenital Heart Disease Physical Activity Lifestyle Study V.2
The Congenital Heart Disease Physical Activity Lifestyle Intervention Study (CHD-PALS) V.2 seeks to determine the efficacy of a lifestyle intervention program for adolescents and young adults (AYAs) with congenital heart disease…
- Trial name
- CHD-PALS V2 (NCT06003023)
- Testing
- Physical Activity Monitoring, Tailored Exercise Prescription, Healthy Lifestyle Education
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- Jamie Jackson
Obstructive Sleep Apnea (OSA), Down Syndrome
Endotype DIrected Treatment for OSA in Down Syndrome
Down syndrome is the most common genetic cause of intellectual disability. People with Down syndrome often have obstructive sleep apnea (OSA), a condition where people have difficulties with breathing while asleep.
- Trial name
- EDIT OSA (NCT07280468)
- Testing
- ato-oxy, Oxygen
- Where
- 5 sites in United States
- Volunteers
- 200 people
- Sponsor
- University of Arizona
Duchenne Muscular Dystrophy (DMD)
The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial
Duchenne Muscular Dystrophy (DMD) is an X-linked recessive disorder characterized by progressive muscle degeneration and frequent developmental, cognitive and behavioral impairments, occurring in one in 5000 live births of boys.
- Trial name
- DMD-DUAL-FUNC (NCT06887491)
- Testing
- Conventional physiotherapy program, Conventional physiotherapy program + Dual task program
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people
- Sponsor
- Lokman Hekim University
Sickle Cell Disease (SCD), Invasive Bacterial Infections
Epidemiology of Invasive Bacterial Infections in Children With Sickle Cell Disease in France Between 2020 and 2025
Children with sickle cell disease are at high risk of invasive bacterial infections, which may lead to serious complications. Preventive measures, including antibiotic prophylaxis and vaccination, have changed the epidemiology of…
- Trial name
- DREPIBAC (NCT07639008)
- Where
- 31 sites in France
- Volunteers
- 350 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Tourette Syndrome, Tic Disorders
Trajectories of Change in Tourette Syndrome
This K23 Career Development Award is designed to provide the training needed for the PI to achieve her long-term career goal of conducting independent, programmatic intervention research in developmental populations.
- Trial name
- NCT06679790
- Testing
- Comprehensive Behavioral Intervention for Tics
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Infant, Premature, Diseases, Birth, Preterm
The NEU-STIM Trial
The aim of this study is to determine the effect of repetitive tactile stimulation compared to selective stimulation on oxygenation of the infant at 5 minutes after birth.
- Trial name
- NEU-STIM (NCT05942924)
- Testing
- Repeated tactile stimulation, Selective tactile stimulation
- Where
- 43 sites in Austria, Belgium and 17 more
- Volunteers
- 3,280 people
- Sponsor
- Leiden University Medical Center
Spinal Cord Injury, Spinal Cord Injury No Sensory/Motor in S4-S5
The Aim of This Research is to Better Understand How Complete Spinal Cord Injury May Change Erogenous Zones in Adult Males
A complete spinal cord injury (SCI) in men is characterized by the total loss of motor skills and sensations below the injury. The loss of penile sensation, the erogenous zone primarily involved in sexual response, is involved in…
- Trial name
- QualiEroSCI (NCT06908850)
- Where
- France
- Volunteers
- 15 people
- Sponsor
- Fondation Garches
Patent Foramen Ovale, Ischemic Stroke
Discontinuation of Antithrombotic Treatment Following Patent Foramen Ovale Closure in Young Patients With Cryptogenic Stroke
To determine the safety of antithrombotic treatment discontinuation 12 months following successful transcatheter PFO closure.
- Conditions
- Patent Foramen Ovale, Ischemic Stroke, Bleeding Ulcer
- Trial name
- HALTI (NCT04475510)
- Testing
- Antiplatelet treatment discontinuation
- Where
- Canada
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Josep Rodes-Cabau
Congenital Heart Disease (CHD)
Cord Clamping Among Neonates With Congenital Heart Disease
The goal of this clinical trial is to compare 2 different timepoints for clamping the umbilical cord at birth for term-born infants with a prenatal diagnosis of congenital heart disease (CHD).
- Trial name
- CORD-CHD (NCT06153459)
- Testing
- Umbilical Cord Clamping at ~30 seconds, Umbilical Cord Clamping at ~120 seconds, Umbilical Cord Milking
- Where
- 21 sites in United States, Canada
- Volunteers
- 500 people
- Sponsor
- Carl Backes, MD
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
Bronchopulmonary Dysplasia
Multidimensional Phenotype Classification in Grade 3 Bronchopulmonary Dysplasia
Bronchopulmonary Dysplasia (BPD), or chronic lung disease of prematurity, is the most consequential complication of preterm birth and is strong predictor of childhood pulmonary and neurodevelopmental disability, particularly in…
- Trial name
- NCT06475976
- Testing
- Chest computed tomography (CT) with angiography, Bronchoscopy with bronchoalveolar lavage, Echocardiography
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Testing Drug Efficacy in Cystic Fibrosis Through N-of-1 Trials
The purpose of this study is to validate and utilize a personalized medicine approach to identify potential treatments with current FDA approved CFTR modifiers for non-approved CF gene mutations.
- Trial name
- Nof1 (NCT04580368)
- Testing
- CFTR Modulators
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Neonatal Encephalopathy, Newborn Asphyxia
Cooling in Mild Encephalopathy
The goal of this randomised control trial is to establish the safety and efficacy of whole-body hypothermia for babies with mild hypoxic ischaemic encephalopathy, inform national and international guidelines, and establish…
- Trial name
- COMET (NCT05889507)
- Testing
- Whole body hypothermia, Targeted normothermia, Supportive neonatal intensive care
- Where
- 39 sites in Italy, United Kingdom
- Volunteers
- 426 people
- Sponsor
- Imperial College London
Anderson-Fabry Disease
Functional Capacity in Anderson-Fabry Disease Patients
The goal of this observational study is to observe the relation between excercise parameters - assessed by CPET - and rest/stress hemodynamic parameters - assessed by echocardiogram and CMR - in patients with a genetic diagnosis…
- Trial name
- OPTIMA-FD (NCT06884358)
- Where
- 5 sites in Italy
- Volunteers
- 100 people
- Sponsor
- IRCCS Policlinico S. Donato
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