Clinical trials: Newborn
Phenylketonuria
The Effectiveness of Kuvan in Amish PKU Patients
The purpose of this study is to determine if Amish patients with PKU show responsiveness after a high dose, prolonged Saproterin trial.
- Trial name
- NCT02677870
- Testing
- saproterin dihydrochloride, Diet treatment
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- University Hospitals Cleveland Medical Center
Fragile X Syndrome
Trial of Sertraline to Treat Children With Fragile X Syndrome
This study is a control trial of sertraline (Zoloft) in fragile X syndrome children aged 2 years to 5 years 8 months old. The trial is six months long, and each participant will receive a series of tests at both the beginning and…
- Trial name
- NCT01474746
- Testing
- Sertraline
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- Randi J. Hagerman, MD
Fetal Alcohol Syndrome, Attention Deficit Hyperactivity Disorder (ADHD)
Open-Label Study of the Long Term Tolerability and Safety of Atomoxetine in Children With FASD and ADD/ADHD
Determine if atomoxetine is safe and well tolerated by children with fetal alcohol syndrome.
- Trial name
- B4Z-US-050 (NCT00418262)
- Testing
- Atomoxetine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Oklahoma
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Premature Birth
Neonatal Resuscitation - Sustained Inflations
This study compares a fifteen second sustained inflation (SI) to five repeated two - three second 'inflation breaths' during resuscitation at delivery of infants born prematurely.
- Trial name
- NCT02967562
- Testing
- Inflation Breaths, Sustained Inflation
- Where
- United Kingdom
- Volunteers
- 63 people
- Sponsor
- King's College London
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Neonatal Abstinence Syndrome
Donor Human Milk in Neonatal Abstinence Syndrome
This study is designed to develop pilot data on the acceptability and benefit of donor human milk for infants undergoing pharmacologic treatment for NAS.
- Trial name
- DHM&NAS (NCT02182973)
- Testing
- donor human milk
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Neolac Inc dba Medolac Laboratories
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Dilated Cardiomyopathy, Heart Failure
PUFAs and Left Ventricular Function in Heart Failure
The purpose of this study is to test the hypothesis that n-3 PUFAs improve left ventricular systolic function in patients with stable chronic HF secondary to nonischemic dilated cardiomyopathy (NICM).
- Trial name
- CS-PUFA-02 (NCT01223703)
- Testing
- n-3 PUFAs
- Where
- Italy
- Volunteers
- 133 people
- Sponsor
- Università degli Studi di Brescia
Maroteaux-Lamy Syndrome
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic…
- Trial name
- NCT02156674
- Testing
- Naglazyme®
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Premature Birth of Newborn
The Developmental Effects of NICU Positioning
The purpose of this study is to investigate the developmental effects of two different types of positioning used with premature infants in the neonatal intensive care unit.
- Trial name
- NCT01628510
- Testing
- Dandle Roo/Dandle Wrap, Traditional NICU Positioning
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Washington University School of Medicine
Ovarian Cancer, Breast Cancer
A Phase II Single Arm Pilot Study of the Chk1/2 Inhibitor (LY2606368) in BRCA1/2 Mutation Associated Breast or Ovarian Cancer, Triple Negative Breast Cancer, High Grade Serous Ovarian Cancer, and Metastatic Castrate-Resistant Prostate Cancer
Background: All cells go through cycles which allow them to divide. In normal cells, checkpoint kinase 1 (Chk1) and checkpoint kinase 2 (Chk2) (CHEK 2 (Chk1/2) stop cell division at various points to allow any damage to…
- Conditions
- Ovarian Cancer, Breast Cancer, Prostate Cancer
- Trial name
- NCT02203513
- Testing
- LY2606368
- Where
- United States
- Volunteers
- 111 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis, Eczema
A Trial of a Botanical Drug Containing East Indian Sandalwood Oil (EISO) For Treatment of Atopic Dermatitis
This trial will be a double-blind, randomized, placebo-controlled, safety, tolerability and efficacy trial of SAN007 (5% East Indian sandalwood oil in a cream formulation) treatment regimen when administered daily for up to 28…
- Trial name
- NCT02871479
- Testing
- SAN007 5% cream, SAN007 10% cream
- Where
- 5 sites in United States
- Volunteers
- 71 people
- Sponsor
- Santalis Pharmaceuticals, Inc.
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Von Willebrand Diseases, Postpartum Hemorrhage
Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial
This is a single-center randomized phase III clinical trial, the VWD-Woman Trial, in which 20 pregnant subjects with von Willebrand disease (VWD), defined as VWF ristocetin co-factor activity (VWF:RCo) <0.50 IU/ml (historic) and…
- Trial name
- NCT04344860
- Testing
- Recombinant Von Willebrand factor, Tranexamic Acid Injection [Cyklokapron]
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Nicoletta C Machin
Corneal Edema Secondary to Corneal Endothelial Dystrophy
Early Experience With Descemet's Stripping Automated Endothelial Keratoplasty
The purpose of this study is to report the early clinical outcomes in patients undergoing Descemet's Stripping Automated Endothelial Keratoplasty (DSAEK) for corneal edema secondary to corneal endothelial cell dysfunction, and to…
- Trial name
- DSAEK (NCT00744796)
- Testing
- DSAEK, Anterior Segment Optical Coherence Topography (OCT), Best Spectacle Corrected Visual Acuity (BSCVA)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic
Efficacy and Safety of Dupilumab in Participants ≥12 to <18 Years of Age, With Moderate-to-severe Atopic Dermatitis
The primary objective of the study was to demonstrate the efficacy of dupilumab as a monotherapy in participants ≥12 years to <18 years of age with moderate-to-severe atopic dermatitis (AD).
- Conditions
- Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic, Eczema, Skin Diseases, Skin, Diseases Genetic, Genetic
- Trial name
- NCT03054428
- Testing
- Dupilumab
- Where
- 45 sites in United States, Canada
- Volunteers
- 251 people
- Sponsor
- Regeneron Pharmaceuticals
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
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