Clinical trials: Effect Neonatal
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
Healthy Volunteers
A Study Evaluating Relative Bioavailability of an Oral Suspension of Abrocitinib and Effect of an Acid Reducing Agent on the Bioavailability of Abrocitinib and Assessing the Taste of Abrocitinib Oral Formulations.
This study consists of 2 parts: Part A is to estimate the relative bioavailability of a single 200 mg dose of abrocitinib oral suspension (Test formulation) compared to the commercial abrocitinib tablet (200 mg) (Reference…
- Trial name
- NCT04903093
- Testing
- Abrocitinib tablet, Abrocitinib Suspension F1, Abrocitinib Suspension F2
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Pfizer
Huntington Disease
Effect of Tetrabenazine on Stroop Interference in HD
Tetrabenazine has been shown to improve gating of abnormal visual stimuli and improve postural stability in Huntington disease (HD) patients as measured by computerized dynamic posturography testing.
- Trial name
- NCT01834911
- Testing
- Tetrabenazine withdrawal
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- New York Medical College
Infant Colic
Effects of Acupuncture in the Treatment of Infant Colic
Standardized acupuncture treatment for infant colic is a common treatment in general practice for doctors educated in medical acupuncture.
- Trial name
- NCT00907621
- Testing
- Acupuncture with Seirin 020x15 mm sterile acupuncture needle
- Where
- Norway
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Oslo
Klinefelter Syndrome
Body Composition in Infants With Klinefelter Syndrome and Effects of Testosterone Treatment
This research study in infant males with Klinefelter syndrome (47,XXY) will learn more about body composition (muscle and fat) and male hormones and look at the effect of testosterone shots on body composition.
- Trial name
- NCT02408445
- Testing
- testosterone cypionate 200mg/ml
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Colorado, Denver
Congenital Heart Defect
The Effects of Inhaled Nitric Oxide After Fontan Operation
Pleural effusions continue to be a post-operative complication seen after Fontan operation. This study will examine the use inhaled nitric oxide (iNO) given for 54 hours after Fontan operation to improve fluid balance.
- Trial name
- NCT00945529
- Testing
- inhaled nitric oxide (iNO)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Ellen Spurrier
Sickle Cell Disease (SCD)
Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease
The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle…
- Trial name
- STEADFAST (NCT04053764)
- Testing
- Crizanlizuamb, Standard of Care
- Where
- 24 sites in United States, Brazil and 9 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Obesity, Sedentary
Effects of Obesity and Physical Inactivity on Pregnancy Outcomes
Regular maternal physical activity leads to the delivery of lighter, leaner infants. Higher birth weights and childhood obesity are both strong predictors for adult obesity, suggesting that the impact of maternal physical…
- Trial name
- NCT02039414
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Washington University School of Medicine
Huntington Disease
Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease
This is a double-blind, placebo-controlled 2-period 10-week treatment within-subject crossover study of neflamapimod in early-stage Huntington disease (HD).
- Trial name
- NCT03980938
- Testing
- neflamapimod
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- EIP Pharma Inc
Congenital Hyperinsulinism
Effect of Exendin-(9-39) on Fasting Adaptation and Protein Sensitivity
The purpose of this study is to examine the effects of exendin-(9-39) on fasting blood glucose and protein induced hypoglycemia on subjects with Congenital Hyperinsulinism.
- Trial name
- NCT00897676
- Testing
- Exendin-(9-39)
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Diva De Leon
Sickle Cell Disease, Sickle Cell Nephropathy
Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease
The purpose of this research study is to learn about the effect of the drug, atorvastatin, on blood vessels in patients with sickle cell disease.
- Trial name
- ENDO (NCT01732718)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
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
Hereditary Angioedema (HAE)
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
- Trial name
- NCT02865720
- Testing
- CINRYZE 500 U, CINRYZE 1000 U
- Where
- 10 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Sickle Cell Disease
Fixed Dose Flavonoid Isoquercetin on Thrombo-Inflammatory Biomarkers in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited hemoglobin disorder. People with SCD have an increased chance for getting blood clots.
- Trial name
- NCT04514510
- Testing
- Isoquercetin
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Short Stature
Weekly Zinc Chelate Supplementation on Children's Growth
The purpose of this study is to measure the effect of weekly zinc supplementation on schoolchildren with growth deficit or normal stature.
- Trial name
- APZinc (NCT01911260)
- Testing
- Zinc amino acid chelate
- Volunteers
- 199 people, healthy volunteers welcome
- Sponsor
- Federal University of São Paulo
Neonatal Abstinence Syndrome
Fetal and Infant Effects of Maternal Buprenorphine Treatment
This research will track the longitudinal neurobehavioral development of the buprenorphine-exposed fetus across gestation through 1 month of age in an effort to determine the safety of this medication for use during gestation…
- Trial name
- NCT01561079
- Testing
- Buprenorphine
- Where
- 2 sites in United States
- Volunteers
- 127 people
- Sponsor
- Johns Hopkins University
Leukemia
Prophylactic White Cell Transfusions Versus Therapeutic White Cell Transfusions in Patients With Leukemia
Patients with leukemia often have low white blood cell counts after chemotherapy, which puts them at greater risk for infection.
- Trial name
- NCT01204788
- Testing
- Prophylactic White Cell Transfusion, Therapeutic White Cell Transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- M.D. Anderson Cancer Center
Huntington Disease
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Huntington disease (HD) is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia.
- Trial name
- Reach2HD (NCT01590888)
- Testing
- PBT2
- Where
- 19 sites in United States, Australia
- Volunteers
- 109 people
- Sponsor
- Prana Biotechnology Limited
Infant, Newborn, Infant, Small for Gestational Age
Donor Milk vs. Formula in Extremely Low Birth Weight (ELBW) Infants
The Milk Trial seeks to determine the effect on neurodevelopmental outcomes at age 22-26 months of donor human milk as compared to preterm infant formula as the in-hospital diet for infants whose mothers choose not to provide…
- Conditions
- Infant, Newborn, Infant, Small for Gestational Age, Infant, Extremely Low Birth Weight
- Trial name
- NCT01534481
- Testing
- Donor Milk, Preterm Formula
- Where
- 17 sites in United States
- Volunteers
- 483 people
- Sponsor
- NICHD Neonatal Research Network
Cystic Fibrosis, Mucociliary Clearance Defect
Researching the Effects of Airway Clearance Therapies in Cystic Fibrosis
This is a pilot study investigating the effectiveness and clinical efficacy of airway clearance therapy (ACT) in cystic fibrosis (CF).
- Trial name
- REACT-CF (NCT03078127)
- Testing
- Baseline (Huff-Cough alone), Oscillatory Positive Expiatory Pressure Device, Whole-Body Vibration Platform
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of North Carolina, Chapel Hill