Clinical trials: Newborn Diseases
Neonatal Hyperbilirubinemia
Home Phototherapy for Term Newborns With Icterus
The study is designed as a randomised controlled multicenter study.The primary aim is to investigate if home phototherapy improves parent-child bonding compared to if treatment is performed at the hospital.
- Trial name
- NCT03536078
- Testing
- home phototherapy
- Where
- 5 sites in Sweden
- Volunteers
- 147 people
- Sponsor
- Region Örebro County
Surgical Infants on NG/NJ/J/G-tube Feeds, Tracheoesophageal Fistula
A Physiological Study to Determine the Enteral Threonine Requirements in Infants Aged 1 to 6 Months
This is the 5th in a series of physiological studies to determine the amino acid requirements of infants. There have been 4 studies to determine tyrosine, methionine, threonine and lysine requirements in infants when they are fed…
- Conditions
- Surgical Infants on NG/NJ/J/G-tube Feeds, Tracheoesophageal Fistula, Pierre-Robin Syndrome, Diaphragmatic Hernia
- Trial name
- INFORALTHR (NCT02364843)
- Testing
- Amino Acid: Threonine
- Where
- Canada
- Volunteers
- 2 people
- Sponsor
- The Hospital for Sick Children
Neonatal Seizure, Epilepsy
Evaluating the Role of Inflammation in Neonatal Epileptogenesis
The purpose of this study evaluate the relationship between inflammation and epilepsy in neonates with seizures after birth.
- Conditions
- Neonatal Seizure, Epilepsy, Seizures, Inflammatory Response
- Trial name
- NSR-RISE (NCT04259125)
- Testing
- Blood draw, Survey
- Where
- 4 sites in United States
- Volunteers
- 72 people
- Sponsor
- University of California, San Francisco
Muscular Dystrophy, Duchenne
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033189
- Testing
- Coenzyme Q10
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Lung Disease, Pulmonary Disease
NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease
- Conditions
- Lung Disease, Pulmonary Disease, AATD, Alpha-1 Antitrypsin Deficiency
- Trial name
- AATD (NCT06622668)
- Testing
- Biological NTLA-3001
- Where
- New Zealand
- Sponsor
- Intellia Therapeutics
Cornelia de Lange Syndrome
Behavioral Assessment and Treatment of Problem Behavior in Children With Cornelia de Lange Syndrome
The goals of this clinical trial are to identify factors associated with the development of problem behavior in Cornelia de Lange syndrome (CdLS) and to develop an effective behavioral assessment and treatment model for problem…
- Trial name
- NCT05829668
- Testing
- ABA-based functional analysis and treatment
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Deafness, Kidney Diseases
Endolymphatic Sac Tumors in a Population of Patients With Von Hippel-Lindau Disease:The Natural History and Pathobiology, and a Prospective Non-Randomized Clinical Trial of Hearing Preservation Surgery in Patients With Early Stage Endolymphatic Sac Tumors
The von Hippel Lindau (VHL) gene has recently been identified as the genetic defect resulting in a syndrome of multiple neoplasias.
- Conditions
- Deafness, Kidney Diseases, Kidney Neoplasms, Neoplasms
- Trial name
- NCT00001668
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Gene Mutations, Pseudoxanthoma Elasticum
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
The purpose of this study (Study INZ701-304 [ADAPT]) is to assess the long-term safety of INZ-701 in patients with ENPP1 Deficiency or ABCC6 Deficiency who have received INZ-701 in an existing clinical study and choose to…
- Conditions
- Gene Mutations, Pseudoxanthoma Elasticum, Arterial Calcification, Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
- Trial name
- NCT06462547
- Testing
- INZ-701
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 22 people
- Sponsor
- Inozyme Pharma
Skin Laxity
Rollover Study to Evaluate Histological Results of Radiofrequency Device Treatments on the Flanks
The purpose of this study is to evaluate histological results of the treatments with the TempSure Firm handpiece on the flanks performed in the CYN20-FIRM-LIPO study.
- Trial name
- NCT04881149
- Testing
- TempSure treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Cynosure, Inc.
Hypothalamic Obesity
A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO)
This is a long-term, open-label extension study of bivamelagon in participants with Hypothalamic Obesity (HO) who have transitioned from the index study, LG-MCCL005. The study will last up to 2 years.
- Trial name
- NCT07156578
- Testing
- Bivamelagon
- Where
- 9 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Thyroid Eye Disease
A Phase III Study of KHN939 in Chinese Patients With Moderate-to-Severe Active Thyroid Eye Disease
This is a multicenter, randomized, double-masked, placebo-controlled Phase 3 study to evaluate the efficacy, safety, immunogenicity, and population pharmacokinetic (PopPK) characteristics of KHN939 in Chinese patients with…
- Trial name
- Halo-1 (NCT07720635)
- Testing
- KHN939
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Beijing Kanghong Biological Medicine Co., Ltd.
Duchenne Muscular Dystrophy
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
While it has been known for many years that corticosteroid use benefits boys with Duchenne Muscular dystrophy (DMD), most clinicians do not consider treating until after age 3 or 4 years of age.
- Trial name
- NCT02167217
- Testing
- Prednisolone
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- Washington University School of Medicine
Hyaline Membrane Disease, Preterm Infant
Evaluation of the Neonatal Autonomic Stress During Intubations Under Propofol in a Population of Premature Infants Under 33 w'GA
Hyaline membrane disease is one of the leading causes of morbidity and mortality in premature newborns in industrialized countries.
- Trial name
- PROPOSURF (NCT03721640)
- Testing
- electrocardiogram
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Skin Laxity
Clinical Assessment of Skin Tightening and Contour Change of Submental Tissue Using Bipolar Radiofrequency Microneedling
The purpose of this study is to evaluate the safety and efficacy of bipolar fractional radiofrequency treatment via use of the Profound System to achieve skin tightening and contour change in lax submental (beneath the chin)…
- Trial name
- NCT04477187
- Testing
- Dermal Handpiece
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Alopecia Areata, Depressive Episode
The Epidemiology, Management, and the Associated Burden of Related Conditions in Alopecia Areata
This study series consists of four related studies and aims to explore and describe many important elements of alopecia areata over three key areas: (1) the current epidemiology of alopecia areata, (2) the prevalence and…
- Conditions
- Alopecia Areata, Depressive Episode, Recurrent Depressive Disorder, Anxiety Disorders
- Trial name
- NCT04239521
- Testing
- Exposure of interest (studies 2 & 3).
- Where
- United Kingdom
- Volunteers
- 51,955 people
- Sponsor
- Momentum Data
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme.
- Trial name
- NCT00196716
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 4 sites in Czechia, Estonia and 2 more
- Volunteers
- 21 people
- Sponsor
- Genzyme, a Sanofi Company
Alström Syndrome
Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed a preceding ProMetic-sponsored Alström Syndrome study with…
- Trial name
- NCT03184584
- Testing
- PBI-4050
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- Liminal BioSciences Ltd.
Electronic Fetal Monitoring, Fetal Hypoxia
Oxygen for Intrauterine Resuscitation of Category II Fetal Heart Tracings
Maternal oxygen administration for concerning fetal heart rate tracing (FHT) patterns is common practice on Labor and Delivery units in the United States.
- Conditions
- Electronic Fetal Monitoring, Fetal Hypoxia, Fetal-Placental Circulation
- Trial name
- O2C2 (NCT02741284)
- Testing
- Room air, 10L Oxygen by nonrebreather mask
- Where
- United States
- Volunteers
- 114 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Retinopathy of Prematurity, Mydriasis
The Effects of Mydriatic Eye Drops in Retinopathy of Prematurity Examinations
Retinopathy of prematurity (ROP) is a retinal disorder of preterm neonates and a potential cause of blindness. As early diagnosis and treatment preserve vision, very low birth weight infants must be screened for ROP.
- Conditions
- Retinopathy of Prematurity, Mydriasis, Neonatal Disease, Analgesic Adverse Reaction
- Trial name
- NCT05880433
- Testing
- NIRS(INVOS 5100; Covidien Somanetics, Troy, MI
- Where
- Turkey (Türkiye)
- Volunteers
- 62 people
- Sponsor
- Istanbul Training and Research Hospital
Neonatal Jaundice From Other Specified Causes, ABO Incompatibility
The Role of Breastmilk and Serum Exosomes in Neonatal Jaundice Due to ABO Incompatibility
Neonatal jaundice is a major global health issue that can lead to serious complications and even death if not promptly treated. ABO incompatibility is a common cause of pathological neonatal jaundice, but there are currently no…
- Conditions
- Neonatal Jaundice From Other Specified Causes, ABO Incompatibility, ABO Hemolytic Disease
- Trial name
- NCT06502847
- Testing
- Exosome profile study
- Where
- Turkey (Türkiye)
- Volunteers
- 338 people
- Sponsor
- Kanuni Sultan Suleyman Training and Research Hospital
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.