Clinical trials: Neonatal Outcomes
Hunter Syndrome
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV)…
- Trial name
- NCT01506141
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- Takeda
Bicuspid Aortic Valve, Thoracic Aortic Aneurysm
The Effect of Losartan in Bicuspid Aortic Valve Patients
The specific aims of this study are to: Establish baseline levels of circulating MMP-2 and -9 , TIMP-1and- 2 and TGFB levels in individuals with bicuspid aortic valve and ascending aortic or aortic sinus measurements >40mm.
- Trial name
- NCT01390181
- Testing
- Cozaar
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Atopic Dermatitis
ASPIRE: PROs & Caregiver Burden in Children With Atopic Dermatitis
This is an open-label, randomized, cross-sectional study to monitor the effects of crisaborole and tacrolimus 0.03% on patient-reported outcomes and caregiver burden in children (ages 2 to 15 years, inclusive) with ≤ moderate…
- Trial name
- NCT03645057
- Testing
- Crisaborole, Tacrolimus 0.03% Ointment
- Where
- United States
- Volunteers
- 92 people
- Sponsor
- University of Rochester
Gaucher Disease
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care.
- Trial name
- SWITCH (NCT04718779)
- Testing
- Digital Engagement Application (GD App), No Intervention
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Takeda
Bronchopulmonary Dysplasia
Analysis of BPD in Premature Infants With Typical Imaging Changes
Bronchopulmonary dysplasia (BPD) is a common chronic respiratory disease in preterm infants. The increase in the survival rate of premature babies following the improvement of perinatal treatment and care has caused an increase…
- Trial name
- NCT04163822
- Testing
- no intervention
- Where
- China
- Volunteers
- 256 people
- Sponsor
- Wang Jianhui
Cerebral Palsy, Hypoxia-Ischemia, Brain
The TOBY Children Study
The aim of this study is to determine the efficacy of therapeutic hypothermia following perinatal asphyxia on neurological and neuropsychological outcomes and also to assess academic attainment and any additional health, societal…
- Trial name
- NCT01092637
- Where
- United Kingdom
- Volunteers
- 280 people
- Sponsor
- Imperial College London
Sickle Cell Disease
A Study to Learn About Sickle Cell Disease In Adult Patients
The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
- Trial name
- NCT05407805
- Testing
- Electronic Diary
- Where
- 4 sites in United States
- Volunteers
- 98 people
- Sponsor
- Pfizer
Infant Mortality, Preterm Birth
Antenatal Micronutrient Supplementation and Infant Survival
The purpose of this community-based randomized trial is to examine whether a daily antenatal and postnatal multiple micronutrient supplement given to women will enhance newborn and infant survival and health and other birth…
- Conditions
- Infant Mortality, Preterm Birth, Low Birth Weight, Neonatal Mortality
- Trial name
- JiVitA-3 (NCT00860470)
- Testing
- Iron (27 mg) - folic acid (600 ug), Multiple micronutrient
- Where
- 2 sites in United States, Bangladesh
- Volunteers
- 44,567 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
Infant, Newborn, Infant, Low Birth Weight
Laparotomy vs. Drainage for Infants With Necrotizing Enterocolitis
This study will compare the effectiveness of two surgical procedures -laparotomy versus drainage - commonly used to treat necrotizing enterocolitis (NEC) or isolated intestinal perforations (IP) in extremely low birth weight…
- Conditions
- Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature
- Trial name
- NEST (NCT01029353)
- Testing
- Laparotomy, Drainage
- Where
- 22 sites in United States
- Volunteers
- 529 people
- Sponsor
- NICHD Neonatal Research Network
Prematurity, Neurodevelopmental Disorder
Effect of Early L-Carnitine Supplementation on Neurodevelopmental Outcomes in Very Preterm Infants
Preterm infants are vulnerable to brain injury, nutritional deficiencies and poor early growth which places them at increased risk for developmental problems later in life.
- Conditions
- Prematurity, Neurodevelopmental Disorder, Carnitine Deficiency
- Trial name
- NCT01783041
- Testing
- L-carnitine, 5% Dextrose
- Where
- 2 sites in United States
- Volunteers
- 144 people
- Sponsor
- Montefiore Medical Center
Type 1 Diabetes, Pregnancy, High Risk
Intravenous Insulin vs Subcutaneous Insulin Infusion in Intrapartum Management of Type 1 Diabetes Mellitus
The purpose of this study is to perform a randomized trial to investigate if intrapartum insulin delivery mechanisms reduces adverse outcomes associated with type 1 diabetes in pregnancy.
- Trial name
- NCT04599075
- Testing
- Insulin
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Gianna Wilkie
Gout
Allopurinol Outcome Study
This is a study of allopurinol in gout patients with hyperuricemia that will evaluate the safety and serum urate (sUA) lowering capability of allopurinol as a urate lowering therapy (ULT) for up to six months.
- Trial name
- LASSO (NCT01391325)
- Testing
- Allopurinol
- Where
- 173 sites in United States, Australia and 5 more
- Volunteers
- 1,735 people
- Sponsor
- Ardea Biosciences, Inc.
Fragile X Syndrome
Study of SPG601 in Adult Men With Fragile X Syndrome
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
- Trial name
- NCT06413537
- Testing
- SPG601
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Spinogenix
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
Growth and Development Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen.
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Trial name
- NCT00486889
- Testing
- alglucosidase alfa
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Cystic Fibrosis (CF)
: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva.
- Trial name
- NCT03445793
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Jewish Health
Infant, Newborn, Infant, Small for Gestational Age
Hydrocortisone for BPD
The Hydrocortisone and Extubation study will test the safety and efficacy of a 10 day course of hydrocortisone for infants who are less than 30 weeks estimated gestational age and who are intubated at 14-28 days of life.
- Conditions
- Infant, Newborn, Infant, Small for Gestational Age, Infant, Very Low Birth Weight, Infant, Premature
- Trial name
- NCT01353313
- Testing
- Hydrocortisone
- Where
- 20 sites in United States
- Volunteers
- 800 people
- Sponsor
- NICHD Neonatal Research Network
Clubfoot
Outcomes of Compliance With Brace Wear in Clubfoot
It has been shown that compliance with brace wear can significantly improve the treatment outcome of idiopathic clubfoot deformities.
- Trial name
- NCT01481324
- Where
- United States
- Volunteers
- 67 people
- Sponsor
- Shriners Hospitals for Children