Clinical trials: Newborn Diseases
MELAS Syndrome
L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome
MELAS patients suffer from exercise intolerance, weakness, poor vision or blindness, poor growth, developmental delay, and deafness.
- Trial name
- NCT01603446
- Testing
- L-Arginine
- Where
- Canada
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- The Hospital for Sick Children
Fontan Procedure, Hypoplastic Left Heart Syndrome
Impact of Vitamin C on Endothelial Function and Exercise Capacity in Fontan-Palliated Patients
In this study, investigators will evaluate the effect of vitamin C on endothelial function, exercise tolerance and quality of life in patients with a single ventricle who have been palliated to Fontan physiology.
- Conditions
- Fontan Procedure, Hypoplastic Left Heart Syndrome, Tricuspid Atresia
- Trial name
- NCT00974025
- Testing
- Vitamin C
- Where
- United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Noonan Syndrome
Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's Syndrome
1. Clinical Objective : To improve the growth of these children 2. Genetic objective : A study of the genetics of the syndrome
- Trial name
- NCT00452725
- Testing
- MAXOMAT ®, biosynthetic growth hormone
- Where
- France
- Volunteers
- 36 people
- Sponsor
- Sanofi
Hemophilia A, Factor VIII (FVIII)
TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies
This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they…
- Trial name
- NCT07692217
- Testing
- Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center
Language Impairment, Down Syndrome
Phenotypic Specific Communication Intervention for Children With Down Syndrome
Children with Down syndrome (DS) have greater difficulty acquiring expressive language than is predicted by their general cognitive abilities and language comprehension (Miller, 1999).
- Trial name
- Merck (NCT02158390)
- Testing
- Jasper-EMT with words and AAC
- Where
- 2 sites in United States
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Skin Laxity
An Evaluation of the Erchonia Laser As a Non-Invasive Treatment to Improve the Appearance of Skin Laxity
The purpose of this clinical study is to determine the effectiveness of the Erchonia® GVS in improving the appearance of skin laxity on the abdominal region.
- Trial name
- NCT06941610
- Testing
- Erchonia GVS Laser
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Erchonia Corporation
Stillbirth and Neonatal Mortality, Perinatal Mortality
Perinatal Mortality Outcomes Through Household Infection Pathways
The goal of this trial is to learn if replacing household soil floors with concrete floors can prevent deaths of infants around the time of birth, including stillbirths and deaths in the first month of life in rural Bangladesh.
- Conditions
- Stillbirth and Neonatal Mortality, Perinatal Mortality, Infant Mortality, Child Mortality
- Trial name
- POTH (NCT07620613)
- Testing
- Concrete household floor
- Where
- Bangladesh
- Volunteers
- 196 people, healthy volunteers welcome
- Sponsor
- Stanford University
Retinopathy of Prematurity
Time Outside Target Oxygen Saturation Range in Preterm Infants and Long Term Outcomes and Preterm Infants
The purpose of this study is to determine if there is a relationship between time spent out the targeted oxygen saturation range in preterm infants is associated with long-term outcomes such as Retinopathy of Prematurity.
- Trial name
- SAT01ROP (NCT00845624)
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Dermatitis, Atopic
Study to Evaluate the Response to Treatment and Safety of 0.03% Tacrolimus (FK506) Ointment Administered in Pediatric Patients With Moderate to Severe Atopic Dermatitis
Objective of this study is to further assess the response to treatment and safety of 0.03% tacrolimus (FK506) ointment when used for 6 months in pediatric patients with moderate to severe atopic dermatitis, known to be responsive…
- Trial name
- NCT00691262
- Testing
- tacrolimus ointment
- Where
- 9 sites in Italy
- Volunteers
- 166 people
- Sponsor
- Astellas Pharma Inc
Cystic Fibrosis
The Relationship Between CFTR Gene Mutations and Exercise Capacity
This study will explore the effect of mutation severity on exercise capacity. Through a better understanding of the association between mutation severity and exercise capacity, clinicians will be more able to predict a given…
- Trial name
- NCT05157646
- Testing
- Fitness tracker
- Where
- United States
- Sponsor
- Virginia Commonwealth University
Multiple Sclerosis
Herbal Therapy for Subcutaneous Injection Site Reactions in Multiple Sclerosis
The purpose of this study is to determine if an herbal over-the-counter cream can decrease skin site reactions in multiple sclerosis patients who currently take either Betaseron, Copaxone or Rebif as their subcutaneous medication…
- Trial name
- MSSkin (NCT00972062)
- Testing
- Bach's Rescue Remedy Cream
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of North Carolina, Charlotte
One Lung Ventilation OLV, Esophageal Atresia
The Effect of Dexmedetomidine on Oxygen During One Lung Ventilation in Pediatric Surgery.
The children who will undergo OLV (one lung ventilation) through general anesthesia will be divided into two groups: The first will be intravenous infusion of dexmedetomidine at 0.4 mcg / kg / hour, and the second will be…
- Conditions
- One Lung Ventilation OLV, Esophageal Atresia, Hydrocystoma, Pneumonectomy
- Trial name
- RCT (NCT04932746)
- Testing
- Dexmedetomidine
- Where
- Syria
- Volunteers
- 110 people
- Sponsor
- Damascus University
Cognitive Impairment, Hereditary Spastic Paraplegia
Studying Cognition in SPG4
Comparing the cognitive levels of patients with SPG4 mutations to healthy controls.
- Trial name
- NCT03104088
- Testing
- CANTAB, MOCA
- Where
- Germany
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital Tuebingen
Cystic Fibrosis
A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del…
- Trial name
- NCT03525574
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 110 sites in United States, Australia and 11 more
- Volunteers
- 507 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Brain Tumor, Brain Pathology
Can we Use Resting-state fMRI and CSD Fiber Tractography for Presurgical Mapping?
This prospective study aims to investigate and validate the use of resting-state functional MRI (rs-fMRI) and high angular resolution diffusion imaging (HARDI), specifically constrained spherical deconvolution (CSD) tractography…
- Conditions
- Brain Tumor, Brain Pathology, Epilepsy, Vascular Malformations, Brain
- Trial name
- NCT06040580
- Where
- Belgium
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Down Syndrome
Egcg, a dyrk1a Inhibitor as Therapeutic Tool for Reversing Cognitive Deficits in Down Syndrome Individuals.
There is a mounting evidence of the modulation properties of the major catechin in green tea, epigallocatechin-3-gallate (EGCG), on dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) gene overexpression in the…
- Trial name
- NCT01394796
- Testing
- Epigallocatechin-3-gallate (EGCG)
- Volunteers
- 31 people
- Sponsor
- Parc de Salut Mar
Cystic Fibrosis
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the…
- Trial name
- NCT03447262
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 100 sites in United States, Australia and 9 more
- Volunteers
- 484 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Stillbirth
Revealed Versus Concealed Cerebroplacental Ratio
The purpose of this study is to evaluate the role of an integrated strategy at selecting fetuses for delivery at term based on a combination of fetal biometry and cerebroplacental ratio (CPR) to reduce stillbirth rate and adverse…
- Trial name
- NCT02907242
- Testing
- Revealment
- Where
- 9 sites in Chile, Czechia and 4 more
- Volunteers
- 11,582 people, healthy volunteers welcome
- Sponsor
- Hospital Clinic of Barcelona
Alpha1-Antitrypsin Deficiency
An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein.
- Trial name
- NCT05899673
- Testing
- Fazirsiran Injection
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 31 people
- Sponsor
- Takeda
Factor X Deficiency
A Study Investigating Treatment Factor X in People With Factor X Deficiency
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg. The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding…
- Trial name
- NCT00930176
- Testing
- Human Coagulation FACTOR X
- Where
- 14 sites in United States, Germany and 3 more
- Volunteers
- 16 people
- Sponsor
- Bio Products Laboratory
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