Clinical trials: Neonatal Disorder
Systemic Lupus, Autoimmune Diseases
Biocollection of Rare Pediatric-onset of Autoimmune and Autoinflammatory Diseases
Rare diseases are defined as those that affect one person in 2,000, or around three million people in France. The majority of rare diseases are caused by genetics and tend to be severe when they begin in childhood.
- Conditions
- Systemic Lupus, Autoimmune Diseases, Autoinflammatory Disease, Genetic Disease
- Trial name
- GENIALII (NCT06435468)
- Testing
- Blood sample for genetic analysis, Blood sample for immunological response assessments, Blood sample to identify relevant biomarker of the disease
- Where
- 13 sites in France
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Hypercholesterolemia, Familial, Heterozygous Familial Hypercholesterolemia
A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158)
The purpose of this study is to determine the pharmacokinetics of laropiprant following administration of a single dose of 1 (Panel A) and 2 (Panel B) combination tablets of MK-0524A in adolescents with heterozygous familial…
- Trial name
- NCT01583647
- Testing
- MK-0524A
- Volunteers
- 10 people
- Sponsor
- Merck Sharp & Dohme LLC
Gilles de la Tourette Syndrome, Tics
Repetitive Transcranial Magnetic Stimulation of the Posterior Parietal Cortex in Patients Suffering From Gilles de la Tourette Syndrome
Repetitive transcranial stimulation (rTMS) of the posterior parietal cortex will be applied daily over five days in adult Gilles de la Tourette patients.
- Trial name
- rTMSPPCGT (NCT01043549)
- Testing
- rTMS
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
MPS IV A
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed…
- Trial name
- NCT00884949
- Testing
- BMN 110
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Respiratory Disease of A Newborn
Swaddling and Facilitated Tucking During Nasal CPAP Application
This study was planned to determine the effects of fetal position and swaddling during nasal CPAP application on pain, stress level, comfort and physiological parameters of the newborn.
- Trial name
- NCT07077148
- Testing
- Facilitated tucking, Swaddling
- Where
- Turkey (Türkiye)
- Volunteers
- 114 people
- Sponsor
- Recep Tayyip Erdogan University
Chronic Myeloproliferative Disorders, Leukemia
12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004058
- Testing
- tetradecanoylphorbol acetate
- Where
- United States
- Sponsor
- University of Medicine and Dentistry of New Jersey
Retinitis Pigmentosa, Stem Cell Transplant
Spheroidal Mesenchymal Stem Cells in Retinitis Pigmentosa
Purpose: This prospective clinical case series aimed to evaluate the effect of suprachoroidal implantation of mesenchymal stem cells (MSCs) in the form of spheroids as a stem cell therapy for retinitis pigmentosa (RP) patients…
- Trial name
- NCT05712148
- Testing
- Stem cell implantation
- Where
- Turkey (Türkiye)
- Volunteers
- 15 people
- Sponsor
- Acibadem University
Neurofibromatosis Type 2, Vestibular Schwannoma
WES of NF2-associated in Comparison to Sporadic Vestibular Schwannomas - Correlation With Clinical Data
Whole exome sequencing (WES) of 50 sporadic and 50 Neurofibromatosis Type2 (NF2)-associated vestibularis schwannomas (VS) in children and young adults.
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Acoustic Neuroma
- Trial name
- NF2 (NCT03210285)
- Testing
- Whole exome sequencing
- Where
- Germany
- Volunteers
- 70 people
- Sponsor
- University Hospital Tuebingen
Bronchiolitis
Association of Dysbiosis and Immune Response in Bronchiolitis in Under 12 Months -Old Infants
Acute bronchiolitis is a common disease in children under the age of two, caused mainly by the respiratory syncytial virus (RSV).
- Trial name
- BRONCHOBIOTE (NCT06161285)
- Testing
- Sampling
- Where
- 3 sites in France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Skin Laxity, Wrinkles
Feasibility Study: Evaluation of Ulthera Versus Thermage for Treating the Face and Neck
Up to 20 subjects will be enrolled and randomly assigned to one of two treatment groups. Group A will receive Ultherapy™ on the right side of the face and neck and Thermage on the left side.
- Trial name
- NCT01713985
- Testing
- Ulthera System Treatment, Thermage
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Heart Failure, Dilated Cardiomyopathy
Myocardial Telomere Recapping Study for Dilated Cardiomyopathy
The aim of this study is to determine the safety and feasibility of giving an adeno-associated viral vector expressing a modified telomerase protein (TERT), driven by cardiac troponin T promoter (AAV9-cTnT-modTERT), to 12 dilated…
- Trial name
- MERCURY-DCM (NCT05837143)
- Testing
- JV001
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai East Hospital
Von Willebrand Disease
Recombinant Von Willebrand Factor in Subjects With Severe Von Willebrand Disease Undergoing Surgery
The purpose of the study is to assess the efficacy and safety of recombinant von Willebrand factor (rVWF) with or without ADVATE in major and minor elective surgical procedures in adult patients with hereditary severe von…
- Trial name
- NCT02283268
- Testing
- Recombinant von Willebrand Factor (rVWF)
- Where
- 34 sites in United States, Australia and 11 more
- Volunteers
- 24 people
- Sponsor
- Baxalta now part of Shire
Advanced Stage Solid Tumors
ATRi Transition Rollover Study
The main purpose of the study was to monitor the safety of participant receiving long-term treatment of Berzosertib as monotherapy.
- Trial name
- NCT03309150
- Testing
- Berzosertib
- Where
- United Kingdom
- Volunteers
- 1 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Maternal Death, Neonatal Death
Integrated Community Engagement and Audit Systems
The goal of this implementation research project is to determine the feasibility of establishing and implementing an acceptable and robust audit system with community representation at secondary health facilities to improve…
- Conditions
- Maternal Death, Neonatal Death, Stillbirth
- Trial name
- NCT05640050
- Testing
- Audit Implementation
- Where
- Pakistan
- Volunteers
- 1,000 people
- Sponsor
- Aga Khan University
Dermatitis, Atopic, Dermatitis
A Study of Lebrikizumab (LY3650150) in Combination With Topical Corticosteroids in Japanese Participants With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to evaluate the efficacy and safety of lebrikizumab in combination with a topical corticosteroids in Japanese participants with atopic dermatitis.
- Conditions
- Dermatitis, Atopic, Dermatitis, Eczema, Skin Diseases
- Trial name
- ADhere-J (NCT04760314)
- Testing
- Lebrikizumab, Topical Corticosteroid
- Where
- 37 sites in Japan
- Volunteers
- 286 people
- Sponsor
- Eli Lilly and Company
Choroidal Ischemia, Vitelliform Macular Dystrophy
Sildenafil for Treatment of Choroidal Ischemia
The hypothesis of this study is to determine if there is a benefit afforded by the use of systemic Sildenafil to patients with choroidal and retinal degenerations and dystrophies, such as vitelliform degeneration, dry and…
- Conditions
- Choroidal Ischemia, Vitelliform Macular Dystrophy, Age-related Macular Degeneration, Central Serous Retinopathy
- Trial name
- NCT04356716
- Testing
- Sildenafil, Standard of Care Sildenafil, Ocular Coherence Tomography-Angiography (OCT-A)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Columbia University
Cerebral Palsy, Brain Injuries
The LETS Study: A Longitudinal Evaluation of Transition Services
This project will describe and evaluate the impact of a unique partnership model designed to coordinate transfer of care by formally linking pediatric and adult heath care services.
- Conditions
- Cerebral Palsy, Brain Injuries, Spinal Dysraphism
- Trial name
- LETS (NCT00975338)
- Testing
- LIFEspan, Non-LIFEspan
- Where
- 2 sites in Canada
- Volunteers
- 149 people
- Sponsor
- Holland Bloorview Kids Rehabilitation Hospital
Atopic Dermatitis
Topical YR001 Ointment in Adult With Mild to Moderate Atopic Dermatitis
This study is to evaluate the safety, tolerability, and pharmacokinetics of YR001 topical ointment in adult patients with mild to moderate atopic dermatitis
- Trial name
- NCT06309355
- Testing
- YR001 ointment
- Where
- 4 sites in United States
- Volunteers
- 35 people
- Sponsor
- Hangzhou Yirui Pharmaceutical Technology Co., Ltd
Neonatal Seizure, Neonatal Encephalopathy
A Dose Escalation Study of Levetiracetam in the Treatment of Neonatal Seizures
The main purpose of this study is to determine the maximum safe tolerated dose of LEV in the treatment of neonatal seizures. Our hypothesis is that optimal dosing of Levetiracetam (LEV) to treat neonatal seizures is significantly…
- Conditions
- Neonatal Seizure, Neonatal Encephalopathy, Hypoxic-Ischemic Encephalopathy, Seizure Newborn
- Trial name
- NEOLEV3 (NCT05610085)
- Testing
- Levetiracetam Injection, Phenobarbital Sodium Injection
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 133 people
- Sponsor
- University of California, San Diego
Iron Overload, Hemochromatosis
Confounder-Corrected Quantitative MRI Biomarker of Hepatic Iron Content
The purpose of this multi-site research is to validate a rapid magnetic resonance based confounder-corrected R-2 mapping method as a quantitative imaging biomarker of liver iron concentrations.
- Conditions
- Iron Overload, Hemochromatosis, Hemosiderosis
- Trial name
- NCT02025543
- Testing
- MRI
- Where
- 4 sites in United States
- Volunteers
- 207 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison