Clinical trials: Neonatal Disorder
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Intellectual Disabilities, Congenital Anomaly
Genome Medical Sequencing for Gene Discovery
Background: A number of rare inherited diseases affect only a few patients, and the genetic causes of these conditions remain unknown.
- Conditions
- Intellectual Disabilities, Congenital Anomaly, Rare Disorders
- Trial name
- NCT01087320
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Homozygous Familial Hypercholesterolemia
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to <18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and…
- Trial name
- SPRUCE (NCT07473843)
- Testing
- Zodasiran
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 10 people
- Sponsor
- Arrowhead Pharmaceuticals
Retinopathy of Prematurity (ROP), Bronchopulmonary Dysplasia (BPD)
The Correlation Between Red Cell Transfusion and Complications of Prematurity
The aim of this clinical trial is to learn if there is a correlation between the erythrocyte transfusion in the early neonatal period in premature infants and early and late complications of prematurity.
- Conditions
- Retinopathy of Prematurity (ROP), Bronchopulmonary Dysplasia (BPD), Enterocolitis, Necrotizing, Intraventricular Hemorrhage Neonatal
- Trial name
- NCT07123948
- Testing
- Wechsler Intelligence Test, performed by educated psychologists
- Where
- Croatia
- Volunteers
- 128 people
- Sponsor
- Clinical Hospital Center Rijeka
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Prematurity; Extreme, Feeding Disorder Neonatal
Early Protein Supplementation in Extremely Preterm Infants Fed Human Milk
The central hypothesis of this clinical trial is that, in extremely preterm infants, protein-enriched human milk diets compared to usual human milk diets during the first 2 weeks after birth increase fat-free mass (FFM)-for-age Z…
- Conditions
- Prematurity; Extreme, Feeding Disorder Neonatal, Breast Milk Expression, Growth Failure
- Trial name
- NCT04325308
- Testing
- Protein-enriched human milk diet, Usual human milk diet
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- University of Alabama at Birmingham
Congenital Heart Disease, Fetal Heart Disorder
Perinatal Cardiology: Bridging Obstetric and Neonatal Care in CHD
This prospective observational study aims to evaluate the agreement between prenatal fetal echocardiographic diagnoses and postnatal diagnoses of congenital heart disease, and to assess the impact of prenatal diagnosis status on…
- Conditions
- Congenital Heart Disease, Fetal Heart Disorder, Neonatal Disease, Fetal Disease
- Trial name
- NCT07820163
- Volunteers
- 115 people
- Sponsor
- Assiut University
HIV/AIDS, Pregnancy Related
Measuring Adverse Pregnancy and Newborn Congenital Outcomes
The purpose of this study is to develop a pharmacovigilance (PV) surveillance program to monitor adverse pregnancy and infant outcomes, including the presence of congenital abnormalities, among HIV-positive and HIV-negative women…
- Conditions
- HIV/AIDS, Pregnancy Related, Congenital Disorders, Newborn Morbidity
- Trial name
- MANGO (NCT04405700)
- Testing
- Pharmacovigilance surveillance program
- Where
- Kenya
- Volunteers
- 2,800 people, healthy volunteers welcome
- Sponsor
- Indiana University
Neonatal Morbidity and Mortality, Intrauterine Growth Disorders (IUGR and Macrosomia)
Analysis on the Health of Newborns in Three Hospitals, 2008-2024
The objective of this observational study is to analyze the factors associated with neonatal morbidity and mortality in three hospitals in Quito, Ecuador, from January 2008 to June 2024.
- Conditions
- Neonatal Morbidity and Mortality, Intrauterine Growth Disorders (IUGR and Macrosomia), Prematurity Complications, Neonatal Infections
- Trial name
- NCT06758622
- Where
- Ecuador
- Volunteers
- 40,000 people
- Sponsor
- HOSPITAL GINECO OBSTETRICO ISIDRO AYORA
Rett Syndrome
Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome
The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.
- Trial name
- ASPIRE (NCT07480564)
- Testing
- TSHA-102
- Where
- 3 sites in United States
- Volunteers
- 4 people
- Sponsor
- Taysha Gene Therapies, Inc.
Neonatal Opioid Withdrawal, Neonatal Opioid Withdrawal Syndrome
Cerebral Physiology of NOWS
This study will use a new device to measure blood flow and oxygen levels in the brains of newborn infants who have had exposure to opioid medications in the womb, compared to newborns who have not had any exposure.
- Conditions
- Neonatal Opioid Withdrawal, Neonatal Opioid Withdrawal Syndrome, Opioid Use Disorder, Intrauterine Exposure
- Trial name
- NIRS for NOWS (NCT07610746)
- Testing
- FD-NIRS and DCS
- Where
- United States
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Indiana University
Premature Birth, Neonatal Hypoglycemia
Higher-Dose Dexamethasone Improves Breathing and Shortens NICU Stay in Preterm Babies
The goal of this observational study was to compare two doses of dexamethasone given to pregnant women at risk of preterm birth. Dexamethasone is a medicine used before birth to help a baby's lungs mature.
- Conditions
- Premature Birth, Neonatal Hypoglycemia, Maternal Infection During Pregnancy, Neurodevelopmental Disorder of Foetus
- Trial name
- NCT07765329
- Testing
- Dexamethasone 12 mg Every 12 Hours, Dexamethasone 6 mg Every 12 Hours
- Where
- Indonesia
- Volunteers
- 200 people
- Sponsor
- Stella Maris Women and Children Hospital
Nutrition Disorders, Gastrointestinal Disease
Review of Infant Oral Feeding and Skills
This study evaluates the infant's feeding skill level at discharge from the neonatal intensive care unit. The goal is to determine whether the ability to "full feed by volume" implies "full skill development" for infant oral…
- Conditions
- Nutrition Disorders, Gastrointestinal Disease, Deglutition Disorder, Feeding Difficulties
- Trial name
- RIOS (NCT06736743)
- Testing
- SMART Tool for Feeding Skill Assessment
- Where
- United States
- Volunteers
- 108 people
- Sponsor
- Wake Forest University Health Sciences
Rett Syndrome
A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)
The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit/risk profile based on the…
- Trial name
- NCT05606614
- Testing
- TSHA-102
- Where
- 9 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Taysha Gene Therapies, Inc.
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Fanconi Anemia, Anemia, Hypoplastic, Congenital
Prenatal Transplantation for Fetuses With Fanconi Anemia
The investigators aim to evaluate the safety and efficacy of in utero hematopoietic stem cell transplantation (IUHSCT) for the treatment of fetuses diagnosed with Fanconi anemia (FA) during pregnancy.
- Conditions
- Fanconi Anemia, Anemia, Hypoplastic, Congenital, Congenital Bone Marrow Failure Syndromes, Bone Marrow Failure Disorders
- Trial name
- NCT07408583
- Testing
- IUHSCT for FA-affected fetuses
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Agnieszka Czechowicz
Microvillus Inclusion Disease, Congenital Disorders
Ascending Doses of Crofelemer Powder for Oral Solution in Pediatric Microvillus Inclusion Disease (MVID)
A study that will evaluate the safety, tolerability and preliminary efficacy of multiple ascending doses of crofelemer, compared to placebo, using a randomized cross-over design within each dose level, when administered to…
- Conditions
- Microvillus Inclusion Disease, Congenital Disorders, Rare Diseases
- Trial name
- NCT06721871
- Testing
- Crofelemer Powder for Oral Solution
- Where
- 3 sites in United States, Italy and 1 more
- Volunteers
- 6 people
- Sponsor
- Napo Pharmaceuticals, Inc.
Feeding Disorder Neonatal
Feasibility of Aerodigestive Stimulation Therapy Trial
The goal of this study is to demonstrate safety, feasibility, and efficacy of a novel pharyngo-esophageal stimulation technique in restoring aerodigestive and swallowing functions in select infants at-risk for chronic gavage tube…
- Trial name
- FAST (NCT06118697)
- Testing
- Esophageal stimulation with high resolution esophageal manometry and nutritive oral feeding therapy
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Sudarshan Jadcherla
Congenital Disorders, Cerebral Desaturation
International Study of Cerebral Oxygenation and Electrical Activity During Major Neonatal Surgery
The goal of this observational study is to determine the incidence of perioperative cerebral desaturation in neonates undergoing surgery for congenital malformations. The main questions it aims to answer are: 1.
- Conditions
- Congenital Disorders, Cerebral Desaturation, Neonatal Surgery
- Trial name
- NCT05673499
- Testing
- NIRS/EEG monitoring
- Where
- 15 sites in United States, Australia and 5 more
- Volunteers
- 900 people
- Sponsor
- Children's Hospital of Philadelphia
Necrotising Enterocolitis Neonatal, Nutrition Disorder, Child
Long-term Nutritional Impact of Necrotizing Enterocolitis in Premature Newborns.
With premature newborn increase survival, the risk of serious neonatal morbidity, such as necrotizing enterocolitis (NEC), also increased.
- Conditions
- Necrotising Enterocolitis Neonatal, Nutrition Disorder, Child, Oral Disorders
- Trial name
- PremGrowth (NCT07490834)
- Testing
- Observation of related perinatal and postnatal factors
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Central Hospital, Nancy, France