Clinical trials: Newborn Diseases
Liver Post-transplant Patients
Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
Lysosomal Acid Lipase (LAL) deficiency is a rare, autosomal recessive storage disease linked to decrease enzymatic activity of LAL, responsible for intracellular accumulation of cholesterol esters and triglycerides.
- Trial name
- LALTH-2 (NCT02851550)
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Hospices Civils de Lyon
Severe Combined Immunodeficiency, Atypical
Generalized Neonatal Screening of Severe Combined Immunodeficiencies
Severe combined Immunodeficiencies ( SCID ) are a group of inherited diseases of the immune system by characterised profound abnormalities of T cell development .
- Trial name
- DEPISTREC (NCT02244450)
- Testing
- SCID screening
- Where
- 51 sites in France
- Volunteers
- 190,539 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Hemophilia A
Factor IX as Adjunctive Therapy to Emicizumab (EMIX)
This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab)…
- Trial name
- NCT05281718
- Testing
- Factor IX
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Tulane University
Spinal Cord Injury, Neurogenic Detrusor Overactivity
Is the Nerve Growth Factor (NGF) a Useful Biomarker in Neurogenic Bladder Dysfunction After Spinal Cord Injury?
Spinal cord injury (SCI) almost always affects bladder function as well. As a result of this bladder dysfunction, individuals with SCI have to undergo regular invasive examination of their bladder function (urodynamic…
- Conditions
- Spinal Cord Injury, Neurogenic Detrusor Overactivity, Neurogenic Lower Urinary Tract Dysfunction
- Trial name
- NCT02138149
- Where
- Switzerland
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Swiss Paraplegic Research, Nottwil
Pediatric ALL
GlideScope Spectrum Video Laryngoscope Blades and Stylet for Use in Neonate & Pre-Adolescent Populations
The overall purpose of this study is to assess the functionality \& impact of the use of the newly designed GlideScope Spectrum pediatric video laryngoscope and stylet for the orotracheal intubation of neonates and pediatric…
- Trial name
- NCT04836338
- Testing
- orotracheal intubation
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Verathon
Cystic Fibrosis, Quality of Life
The Effects of Massage Therapy on QOL in Youth/Young Adults With Cystic Fibrosis
The purpose of this study is to describe the effects of massage therapy on quality of life (QOL) in youth and young adults (ages 8 to 21 years) with cystic fibrosis (CF).
- Trial name
- NCT01729585
- Testing
- Massage Therapy
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Graves Ophthalmopathy
Sirolimus for Graves' Orbitopathy (GO)
Graves' Orbitopathy (GO) is a disabling and disfiguring condition associated with Graves' Disease, due to autoimmunity against antigens expressed by the thyroid and orbital tissues, and resulting in orbital fibroblast…
- Trial name
- SIRGO (NCT04598815)
- Testing
- Sirolimus
- Where
- Italy
- Volunteers
- 54 people
- Sponsor
- University of Pisa
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Phenylketonuria
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
Sapropterin dihydrochloride (subsequently referred to as sapropterin) (Kuvan®) was approved by the FDA for the treatment of hyperphenylalaninemia in 2007.
- Trial name
- NCT00789568
- Testing
- sapropterin dihydrochloride, Moxifloxacin
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Disease
Biotin-RBC Transfusion in SCD
This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion…
- Trial name
- NCT04426591
- Testing
- Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Marianne Yee
Teeth Absent, Tooth-loss
Peri-implant Tissues on the Galimplant Anti-rotational Slim Abutment.
A dental implant is an alloplastic material surgically inserted in a residual bone ridge, mainly with a prosthetic foundation. Nowadays the most widely used implants and with a higher degree of scientific evidence are the…
- Conditions
- Teeth Absent, Tooth-loss, Tooth Extraction, Dental Implant
- Trial name
- NCT05118815
- Testing
- Slim abutment, Standard abutment
- Where
- Spain
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- University of Santiago de Compostela
Congenital Myotonia
Relations Between Myotonia and Fitness
Investigators aimed to investigate whether training can increase fitness levels in patients with myotonia, and thereby reduce the symptom of myotonia.
- Trial name
- NCT02161835
- Testing
- Training
- Where
- Denmark
- Volunteers
- 9 people
- Sponsor
- Grete Andersen, MD
Cytomegalovirus Infections, Breast Feeding
Biochip for HCMV Detection in Breast Milk
Human cytomegalovirus (HCMV) is the leading cause of neonatal viral infection and can have a significant impact on the neurosensory development of newborns and especially preterm infants.
- Conditions
- Cytomegalovirus Infections, Breast Feeding, Premature Birth of Newborn
- Trial name
- VIRUMILK (NCT02840825)
- Where
- France
- Volunteers
- 2 people
- Sponsor
- Centre Hospitalier Universitaire de Besancon
Psuedomas Infection, Cystic Fibrosis
The Role of Bacteria and Genetic Variations in Cystic Fibrosis
This study will examine 1) the role of hereditary factors in cystic fibrosis; i.e., the relationship of the disease to specific gene variations, and 2) the role of bacterial products involved in lung infections substances…
- Trial name
- NCT00043225
- Where
- 4 sites in United States
- Volunteers
- 76 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy
Exploratory Study of NS-065/NCNP-01 in DMD
This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT02081625
- Testing
- NS-065/NCNP-01
- Where
- Japan
- Volunteers
- 10 people
- Sponsor
- National Center of Neurology and Psychiatry, Japan
Anemia, Sickle Cell
Transfusion Treatment in Patients With SCD
The "National Transfusion Treatment Survey in patients with sickle cell disease (SCD)" is a prospective longitudinal systemic study that was created in order to evaluate the therapeutic approach, mainly transfusional, in patients…
- Trial name
- NCT03397017
- Testing
- Transfusion
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Società Italiana Talassemie ed Emoglobinopatie
Corneal Dystrophy, Macular Dystrophy
The Efficacy of Hyperemesis Gravidarum on Macular Thickness, Corneal Thickness and Intraocular Pressure in Pregnancy
Aim Physiological changes in intraocular pressure as well as in the cornea and macula may occur during pregnancy. In the literature, there are limited data on ocular findings in hyperemesis gravidarum.
- Conditions
- Corneal Dystrophy, Macular Dystrophy, Hyperemesis Gravidarum - Severe
- Trial name
- NCT05927740
- Testing
- Optic coherence tomography
- Where
- Turkey (Türkiye)
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Batman Training and Research Hospital
Hypoplastic Left Heart Syndrome
Prophylactic Peritoneal Dialysis Decreases Time to Achieve a Negative Fluid Balance After the Norwood Procedure
Babies born with hypoplastic left heart syndrome (HLHS) have three separate, complex heart surgeries before they turn three years of age. The first surgery typically happens in the first two weeks of life.
- Trial name
- NCT01215240
- Testing
- Peritoneal dialysis
- Where
- Canada
- Volunteers
- 22 people
- Sponsor
- University of Alberta
Acquired Haemophilia
Turkish Acquired Haemophilia Registry
Turkish Acquired Haemophilia Registry is a database in which information about clinical features and therapeutic management of acquired haemophilia is collected in Turkey.This is a multicentre, retrospective and prospective…
- Trial name
- EHEM (NCT01403740)
- Testing
- rFVIIIa, aPCC, FVIII, DDAVP, Azathioprine, Rituximab, Cyclosporin
- Where
- Turkey (Türkiye)
- Volunteers
- 250 people
- Sponsor
- Turkish Society of Hematology
Patent Ductus Arteriosus
Patent Ductus Arteriosus and Splanchnic Oxygenation at First Feed
Patent ductus arteriosus (PDA) is common in preterm infants. In the presence of a large PDA, significant systemic to pulmonary shunting occurs, which may results in pulmonary hyperperfusion and systemic hypoperfusion.
- Trial name
- NCT03723889
- Volunteers
- 50 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
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