Clinical trials: Birth Defect
Homozygous Familial Hypercholesterolemia
LOWER: Lomitapide Observational Worldwide Evaluation Registry
This global product exposure registry is a multicentre, long-term, prospective, observational cohort study (exposure registry), designed to evaluate the long term safety and effectiveness of lomitapide.
- Trial name
- LOWER (NCT02135705)
- Testing
- Lomitapide
- Where
- 71 sites in United States, Argentina and 6 more
- Volunteers
- 260 people
- Sponsor
- Amryt Pharma
Developmental Dysplasia of the Hip (DDH)
Truncal Blocks for Pediatric With Developmental Dysplasia of the Hip Undergoing Open Reduction
The aim of the study is to evaluate the effectiveness of ultrasound-guided transversalis fascia plane block (TFPB) and quadratus lumborum block (QLB) on post-operative analgesia in pediatric patients with Developmental Dysplasia…
- Trial name
- NCT03189966
- Testing
- transversalis fascia plane block, quadratus lumborum block
- Where
- 2 sites in China
- Volunteers
- 110 people
- Sponsor
- Second Affiliated Hospital of Wenzhou Medical University
Coarctation of Aorta
Prognosis and Integrative Assessment of Aortic Coarctation Patients in China
This is a systemic research of Chinese aortic coarctation patients, aiming to determine risk factors and serial biomarkers of aortic coarctation in prognosis.
- Trial name
- NCT04011956
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Beijing Institute of Heart, Lung and Blood Vessel Diseases
Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM)
AI-enabled Screening and Diagnosis of Cardiomyopathies Using Coronary CTA
The goal of this observational and diagnostic study is to develop and validate an artificial intelligence assisted approach for coronary computer tomography angiography-(CCTA)-based screening and diagnosis of cardiomyopathies in…
- Conditions
- Cardiovascular Diseases, Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Restrictive Cardiomyopathy
- Trial name
- Atlantis (NCT06748261)
- Testing
- CCTAI model
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Shanghai Zhongshan Hospital
Congenital Heart Disease, Pulmonary Hypertension
MRI and Computational Simulation Cardiology Study
The purpose of this study is to (1) quantify cardiovascular anatomy and physiology using magnetic resonance imaging under both resting and exercise conditions in patients who have congenital heart disease and in age-matched…
- Trial name
- NCT03149341
- Testing
- Magnetic Resonance Imaging
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- Stanford University
Sickle Cell Disease, Pregnancy, High Risk
SCRIPT: Sickle Cell Risk in Pregnancy Tool
Sickle Cell Disease (SCD), common in persons of Black ancestry, affects the shape of hemoglobin, the oxygen-carrying part of red blood cells (RBC).
- Conditions
- Sickle Cell Disease, Pregnancy, High Risk, Pregnancy Complications
- Trial name
- SCRIPT (NCT06529042)
- Testing
- Non-Interventional
- Where
- 3 sites in United States, Canada
- Volunteers
- 1,000 people
- Sponsor
- Mount Sinai Hospital, Canada
Juvenile Idiopathic Arthritis (JIA), Familial Mediterranean Fever (FMF )
Gait Profile and Variables in Pediatric Rheumatic Disease Using a Smart Insole System
In pediatric rheumatic diseases, joint swelling, effusion, tenderness, and painful restriction of joint movement, particularly in weight-bearing joints, frequently alter walking function.
- Conditions
- Juvenile Idiopathic Arthritis (JIA), Familial Mediterranean Fever (FMF ), Healthy Controls
- Trial name
- NCT06705673
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Istanbul University - Cerrahpasa
Retinopathy of Prematurity
REDEXAM - Reducing Painful Eye Examinations in Preterm Infants
More than 8000 babies born >8 weeks early or weighing less than 1500g at birth in the United Kingdom annually are at risk of a serious eye problem - retinopathy of prematurity (ROP).
- Trial name
- REDEXAM (NCT01861470)
- Where
- United Kingdom
- Volunteers
- 500 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Cystic Fibrosis
A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1
The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic…
- Trial name
- NCT04059094
- Testing
- BI 1265162
- Where
- 26 sites in United States, Belgium and 6 more
- Volunteers
- 52 people
- Sponsor
- Boehringer Ingelheim
Craniosynostoses
CraNIRS Clinical Study
Craniosynostosis is a rare disorder characterized by a premature fusion of one or more sutures of the skull. Craniosynostosis is usually diagnosed in the first years of life.
- Trial name
- CraNIRS (NCT04086056)
- Testing
- NIRS monitoring
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Hospices Civils de Lyon
Abnormalities, Blood Coagulation Disorder
Study of Abnormal Blood Clotting in Children With Stroke
Effective treatment and prevention strategies for childhood stroke and porencephaly can only be developed once the causes are understood.
- Conditions
- Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident
- Trial name
- NCT00001927
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cystic Fibrosis
Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
- Trial name
- NCT00803205
- Testing
- Ataluren
- Where
- 38 sites in United States, Belgium and 9 more
- Volunteers
- 238 people
- Sponsor
- PTC Therapeutics
Ductus Arteriosus, Patent, Preterm Infants
New Management Strategy of PDA for VLBW Preterm Infants
Patent ductus arteriosus (PDA) is one of the most common complications in premature infants. Successful pharmacological closure of PDA with indomethacin was first reported in 1976.
- Trial name
- NCT00239512
- Testing
- Indomethacin and Ibuprofen
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- China Medical University Hospital
Prader-Willi Syndrome
Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation
The goal of this study is to help teens with Prader-Willi Syndrome (PWS) and their families learn practical strategies for managing issues like irritability, meltdowns, and anxiety.
- Trial name
- RT-PWS (NCT07607730)
- Testing
- Regulating Together group therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Mercy Hospital Kansas City
Atopic Dermatitis
Long-term Safety and Efficacy of Lebrikizumab in Adult and Adolescent Participant With Moderate-to-Severe Atopic Dermatitis
The main purpose of the study is to assess the long-term tolerability and effectiveness of lebrikizumab in adult and adolescent participants with moderate-to-severe atopic dermatitis (AD).
- Trial name
- ADlong (NCT05916365)
- Testing
- Lebrikizumab
- Where
- 32 sites in Germany, Poland
- Volunteers
- 174 people
- Sponsor
- Almirall, S.A.
Congenital Heart Disease
Exercise Training in Children With Congenital Heart Defect
The purpose of this study was to investigate the effects of exercise training on exercise capacity, physical activity (PA), health-related quality of life (HRQoL), and the psychosocial factors affecting PA in sedentary congenital…
- Trial name
- NCT00930800
- Testing
- Exercise
- Where
- Taiwan
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
FASD
Dog-Assisted Therapy for Children and Adolescents With FASD
The rationale of the present study was to evaluate the efficacy of DAT in children and adolescents with FASD in relation to its effects on social skills, internalized and externalized symptomatology and on severity of FASD…
- Trial name
- NCT04038164
- Testing
- Dog-Assisted Therapy and pharmacological treatment
- Where
- Spain
- Volunteers
- 39 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Congenital Heart Disease (CHD)
Physical Rehabilitation in Patients With Complex Congenital Heart Disease
Studies have shown that patients with congenital heart disease have functional changes compared to healthy individuals. Several interventions can be performed to minimize these changes; physical rehabilitation is one of these…
- Trial name
- Rehabkids (NCT07089264)
- Testing
- Physical rehabilitation
- Where
- Brazil
- Volunteers
- 20 people
- Sponsor
- Hospital do Coracao
Hirschsprung Disease, Febrile Urinary Tract Infection (Disorder)
Does Hirschsprung Disease Increase the Risk of Febrile Urinary Tract Infection in Children
Febrile urinary tract infection (FUTIs) are the most common bacterial infections in children under the age of 2 years. They represent 7% of children presenting with fever without a source.
- Trial name
- NCT04837963
- Where
- France
- Volunteers
- 630 people
- Sponsor
- University Hospital, Montpellier
Acute Intermittent Porphyria (AIP)
Identification of Acute Intermittent Porphyria Modifying Genes
This study proposes to identify the predisposing/protective modifying genes that underlie the acute attacks in symptomatic patients with Acute Intermittent Porphyria (AIP), an autosomal dominant inborn error of heme biosynthesis.
- Trial name
- NCT05502133
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai