Clinical trials: Congenital Abnormalities
Patent Ductus Arteriosus, Congenital Heart Disease
PDA for Kidneys Study
The goal of this observational study is to gather more information on kidney oxygen levels in babies with a patent ductus arteriosus (PDA), and evaluate the relationships between kidney oxygen levels, PDA status and kidney…
- Trial name
- PDA4K (NCT06658496)
- Testing
- Near Infrared Spectroscopy
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Wisconsin, Madison
Huntington Disease
Huntington's Disease Biobank: Advancing Remote Monitoring and Deep Phenotyping
This observational study aims to identify novel biomarkers of disease onset and progression in Huntington's disease by integrating remote monitoring with fluid biomarkers.
- Trial name
- NCT06941662
- Testing
- computer vision, remote monitoring, blood, urine, wrist bands, mobile-app
- Where
- United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Stanford University
Atopic Dermatitis
Protopic Adherence and Efficacy in Korean Eczema Patient
The purpose of this study is to determine whether the dosage of 0.1% tacrolimus ointment applied on unit area of eczema influence the efficacy of treatment.
- Trial name
- NCT02126176
- Where
- South Korea
- Volunteers
- 50 people
- Sponsor
- Pusan National University Hospital
Fabry Disease
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on…
- Trial name
- FAVORIT (NCT06880250)
- Where
- 23 sites in Russia
- Volunteers
- 100 people
- Sponsor
- NPO Petrovax
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
Palbociclib in Treating Patients With Relapsed or Refractory Rb Positive Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating Alterations in Cell Cycle Genes (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well palbociclib works in treating patients with Rb positive solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with activating alterations (mutations) in cell cycle genes…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Ewing Sarcoma, Recurrent Glioma
- Trial name
- NCT03526250
- Testing
- Laboratory Biomarker Analysis, Palbociclib, Pharmacological Study
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Congenital Heart Disease
Transition Care Model for Adolescents With Congenital Heart Disease
The primary aim of this study is to assess the effectiveness of a standardized transition care model (TC) on the perceived mental and physical health of adolescents with congenital heart disease (CHD).
- Trial name
- TELEMACO (NCT05713591)
- Testing
- Transition care model
- Where
- Italy
- Volunteers
- 964 people, healthy volunteers welcome
- Sponsor
- IRCCS Policlinico S. Donato
Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies
The Natural History of Familial Dysautonomia
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.
- Conditions
- Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies, Hereditary Sensory and Autonomic Neuropathy 3
- Trial name
- NCT03920774
- Where
- 2 sites in United States, Israel
- Volunteers
- 400 people
- Sponsor
- NYU Langone Health
Angulation Deformity of Long Bone (Disorder), Acquired Deformity of Knee Joint (Disorder)
Use of Motion Sensors in Correction Procedures of Long Bone Deformities in the Pediatric Age Group
Motion sensors will be used in long bone deformity surgery in pediatric patients. In patients whose deformity status is determined before the surgery, 2 wires will be placed in the same plane from the proximal and distal of the…
- Trial name
- NCT06516497
- Testing
- Group using traditional method in long bone deformity correction, Group using computer-aided sensors in long bone deformity correction
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Ataturk University
Cancers
Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)
The purpose of this study is to safely remove ovarian tissue in pre-pubertal pediatric patients, who are at risk for infertility from their medical treatment, for freezing for future restoration of fertility and hormone function.
- Trial name
- NCT05299710
- Testing
- Laparoscopic surgery
- Where
- United States
- Volunteers
- 250 people
- Sponsor
- Erin Rowell
Musculoskeletal Abnormality, Pediatric
Reliability and Acceptability of Real-time Virtual Video Pediatric Gait, Arms, Legs and Spine (V-pGALS)
The use of telemedicine in musculoskeletal assessment has been historically low. The current COVID 19 global pandemic has forced a paradigm shift with many centers rapidly adopting virtual visits to conduct care resulting in…
- Trial name
- NCT04668820
- Testing
- video Pediatric Gait, Arms, Legs and Spine (V-pGALS)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Marmara University
Dry Eye, Neuropathic Pain
Neurosensory Abnormalities in SymptomAtic Ocular Surface Patients (NASA)
This study is designed to measure how common nerve abnormalities are within a group of patients who feel discomfort within their eyes.
- Trial name
- NASA (NCT04838223)
- Testing
- Hyperosmolar Response, Proparacaine Challenge Test, Contact Lens Dry Eye Questionnaire-8 (CLDEQ-8) Optional; only for Contact Lens Wearers
- Where
- 16 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Tufts Medical Center
Huntington Disease
A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease
A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease
- Trial name
- NCT05541627
- Testing
- AB-1001 Gene Therapy
- Where
- France
- Volunteers
- 5 people
- Sponsor
- AskBio France, SAS, a subsidiary of AskBio Inc.
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Lennox-Gastaut Syndrome
Investigation of Cognitive Outcomes With Cannabidiol Oral Solution
This study is being conducted to evaluate the effects of GWP42003-P on cognition in pediatric participants, aged 3 to 10 years, with Lennox-Gastaut Syndrome (LGS).
- Trial name
- EPI-COG (NCT04133480)
- Testing
- GWP42003-P
- Sponsor
- Jazz Pharmaceuticals
Synovial Diseases, Pigmented Villonodular Synovitis
Clinical Study of Magnetic Resonance Imaging and Deep Learning of Joint Synovial Disease
Through the high-throughput feature extraction of magnetic resonance images, the deep learning prediction model of joint synovial lesions is constructed used for the diagnosis, differential diagnosis and curative effect…
- Conditions
- Synovial Diseases, Pigmented Villonodular Synovitis, Gout, Rheumatoid Arthritis
- Trial name
- NCT04952896
- Testing
- Synovitis diagnosis
- Where
- China
- Volunteers
- 350 people
- Sponsor
- Peking University Third Hospital
Ankyloglossia, Oral Mucoceles
The Effect of Pre-emptive Analgesia on the Postoperative Pain in Children Undergoing Pediatric Stomatology Day Surgeries
The aim of this double-blinded, randomized, controlled trial was to test whether pre-emptive analgesia with ibuprofen suppository is effective for children undergoing pediatric stomatology day surgeries in the postoperative…
- Conditions
- Ankyloglossia, Oral Mucoceles, Supernumerary Teeth
- Trial name
- NCT05878262
- Testing
- ibuprofen suppository
- Where
- China
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Neonatal SEPSIS
Prognostic Value of Red Cell Distribution Width (RDW) in Neonatal Sepsis
1. Evaluate the relationship of RDW and severity and mortality in patients with neonatal sepsis . 2. Using RDW as a simple, inexpensive, applicable and rapid test to detect prognosis of neonatal sepsis .
- Trial name
- NCT03403062
- Volunteers
- 100 people
- Sponsor
- Assiut University
Duchenne Muscular Dystrophy
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
- Trial name
- SIDEROS-E (NCT03603288)
- Testing
- idebenone 150 mg film-coated tablets
- Where
- 39 sites in United States, Austria and 7 more
- Volunteers
- 161 people
- Sponsor
- Santhera Pharmaceuticals
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Control of Steatorrhea in Participants With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
This study by Aptalis (formerly Axcan) assesses the efficacy and safety of Panzytrat® 25,000 compared to Kreon® 25,000 in the control of steatorrhea in participants with cystic fibrosis (CF) and exocrine pancreatic insufficiency…
- Trial name
- NCT01327703
- Testing
- Panzytrat® 25,000, Kreon® 25,000
- Where
- 13 sites in Germany, Poland
- Volunteers
- 87 people
- Sponsor
- Forest Laboratories
Neonatal SEPSIS, Prematurity
Neonatal Bacterial Colonization Study
This study will evaluate the effect of skin antisepsis and/or emollient therapy on bacterial colonization dynamics in very low birth weight, hospitalized infants.
- Conditions
- Neonatal SEPSIS, Prematurity, Very Low Birth Weight Baby, Bloodstream Infection
- Trial name
- NCT03896893
- Testing
- Skin antisepsis, Emollient
- Where
- South Africa
- Volunteers
- 80 people
- Sponsor
- University of Stellenbosch