Clinical trials: Newborn Diseases
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
Hypoplastic Left Heart Syndrome
Fetal Aortic Valvuloplasty for Evolving Hypoplastic Left Heart Syndrome
The purpose of this research is to investigate the best way to manage evolving hypoplastic left heart syndrome (HLHS).
- Trial name
- NCT06342999
- Testing
- Fetal Aortic Valvuloplasty Procedure, Emerge Monorail and Over-The-Wire PTCA Dilatation Catheter, Trek RX and Mini Trek RX Coronary Dilatation Catheter
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Mauro H. Schenone
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Generalized Epidermolysis Bullosa Simplex
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)
The proposed Phase 2/3 trial with double-blind and open-label extension phases is an international, multicenter study designed to assess the efficacy and safety of diacerein 1% ointment in patients with generalized EBS.
- Trial name
- EBShield (NCT06073132)
- Testing
- AC-203, Vehicle
- Where
- 30 sites in United States, Australia and 16 more
- Volunteers
- 80 people
- Sponsor
- TWi Biotechnology, Inc.
Congenital Heart Disease, Neurodevelopment
Improving Neurodevelopment in Adolescents With Congenital Heart Disease
Executive dysfunction can profoundly impact all dimensions of a child's development. Impairments in executive function are a central component of the neurodevelopmental phenotype associated with CHD, and manifest as behavioral…
- Conditions
- Congenital Heart Disease, Neurodevelopment, Executive Function, Working Memory Training
- Trial name
- NCT02759263
- Testing
- Cogmed Working Memory Training
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Boston Children's Hospital
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
Familial Adenomatous Polyposis
Trial of eRapa to Prevent Progression in Familial Adenomatous Polyposis Patients Under Active Surveillance
Patients with Familial Adenomatous Polyposis (FAP) who are undergoing endoscopic surveillance will be given Encapsulated Rapamycin (eRapa) at one of three escalating doses/schedules for 12 months with the aim of reducing polyp…
- Trial name
- NCT04230499
- Testing
- Encapsulated Rapamycin (eRapa)
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Rapamycin Holdings Inc.
Sickle Cell Anemia
EXTEND EXpanding Treatment for Existing Neurological Disease
The primary goal of the Phase II EXTEND trial is to investigate the effects of open-label hydroxyurea treatment, escalated to maximum tolerated dose, for children with Sickle Cell Anemia and either conditional (170 - 199 cm/sec)…
- Trial name
- NCT02556099
- Testing
- Hydroxyurea
- Where
- Jamaica
- Volunteers
- 43 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Retinopathy of Prematurity Both Eyes, Retinal Disease
VEGF Concentrations After Intravitreal Bevacizumab vs Ranibizumab as a Treatment for Type 1 ROP
The Retinopathy of Prematurity (ROP) is one of the leading causes of blindness on the pediatric age worldwide. This pathology is characterized for arrest of the normal vascular and neuronal retina that because of pathological…
- Conditions
- Retinopathy of Prematurity Both Eyes, Retinal Disease, Premature Birth, Eye Diseases
- Trial name
- NCT03148132
- Testing
- Bevacizumab Injection, Ranibizumab Ophthalmic
- Where
- Mexico
- Volunteers
- 16 people
- Sponsor
- Universidad Autonoma de San Luis Potosí
Pantothenate Kinase-Associated Neurodegeneration
CoA-Z in Pantothenate Kinase-associated Neurodegeneration (PKAN)
The purpose of this study is to learn more about how people with the condition pantothenate kinase-associated neurodegeneration (PKAN) respond to a specialized study product.
- Trial name
- NCT04182763
- Testing
- CoA-Z
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Oregon Health and Science University
Pompe Disease (Late-onset)
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term…
- Trial name
- NCT06178432
- Testing
- CRG003 injection
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Huashan Hospital
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
Premature, Thermoregulation
Delaying First Bathing and Skin Barrier Function on Infant
It is still unknown when the first bath should be done in premature newborns. Investigators think that delaying the time of the first bath compared to the time in the clinic will show some positive changes in preterm newborns.
- Trial name
- TEWL (NCT04231799)
- Testing
- Bathing
- Where
- Turkey (Türkiye)
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Akdeniz University
Flexible Flatfoot
Effectiveness of Core Stability Exercises on Foot Posture, Function and Limb Alignment in Flexible Flat Feet Patients
Flexible flat foot is a very common musculoskeletal disorder of lower limbs in general population. Studies have reported that a flexible flat foot causes an alteration in the human body structures and lower limb kinetics due to a…
- Trial name
- NCT06567509
- Testing
- Core stability exercises and Short foot exercises, Conventional foot exercises and short foot exercises
- Where
- Malaysia
- Volunteers
- 85 people
- Sponsor
- International Islamic University Malaysia
Congenital Heart Disease (CHD)
Assessment of Flow With the New Four-dimensional Velocity-encoded Magnetic Resonance Imaging Technique
The purpose of our study is to validate the accuracy of 4D PC flow method in quantification of thoracic and upper abdominal blood flow volumes in children.
- Trial name
- 4D Flow (NCT02952287)
- Testing
- 4D PC MRI acquisition
- Volunteers
- 100 people
- Sponsor
- The Hospital for Sick Children
Turner Syndrome
Cardiopulmonary Exercise Testing in Girls (8-18y) with Turner Sydrome.
The goal of this clinical trial is to have a beter insight in the exercise tolerance in girls with Turner Syndrome aged 8-18 years .
- Trial name
- NCT06780514
- Testing
- Cardiopulmonary exercise test
- Where
- Belgium
- Volunteers
- 20 people
- Sponsor
- University Hospital, Ghent
Perinatal Mortality, Premature Rupture of Fetal Membranes
Predictive Score for Neonatal Mortality for Women With Premature Rupture of Membranes Between 22 and 28 Weeks of Gestation
Pretern premature rupture of the membranes (PPROM) remains the leading cause of preterm deliveries and neonatal mortality and morbidity. PPROM is defined as rupture of the fetal membranes prior to 37 weeks' gestation.
- Trial name
- PPROM (NCT03108404)
- Testing
- Validation of a diagnostic test
- Where
- France
- Volunteers
- 34 people
- Sponsor
- Centre Hospitalier Universitaire, Amiens
Hearing Loss, Sensorineural, Cystic Fibrosis
A Study of Web and Tablet-based Interactive Audiometry in Adults With CF
Adults with CF regularly receive anti-infective therapy that can result in impaired hearing. Recent studies have identified that up to 50% of adults with CF have hearing loss with resulting significant impact on quality of life.
- Trial name
- NCT04400955
- Testing
- Audiometry tablet diagnostic tool and web-based test
- Where
- United Kingdom
- Volunteers
- 156 people
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust
Clear Cell Renal Cell Cancer (ccRCC), Urothelial Carcinoma (UC)
18F-T2 PET/CT Imaging for CAIX Positive Solid Tumors
The goal of this clinical trial is to evaluate the diagnostic value of CAIX protein specific probe 18F-T2 in PET/CT imaging in participants with solid tumors.
- Conditions
- Clear Cell Renal Cell Cancer (ccRCC), Urothelial Carcinoma (UC), Colorectal Cancer, Cervical Cancer
- Trial name
- NCT07557225
- Testing
- 18F-T2 PET/CT
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Peking University First Hospital
Craniosynostosis
MRI for Non-invasive Evaluation of Brain Stress
Craniosynostosis is a birth defect that causes the bones on a baby's head to fuse together earlier than normal. This causes the baby to have an abnormally shaped head.
- Trial name
- NCT01898650
- Testing
- MR scan
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
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