Clinical trials: Neonatal Disease
Fabry Disease
A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease
Background It is necessary to find out the difficulties experienced by patients with Fabry disease and their families, and to seek solutions for them.
- Trial name
- NCT05679076
- Where
- South Korea
- Volunteers
- 10 people
- Sponsor
- Yonsei University
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Dermatitis, Atopic, Eczema
A Study of Lebrikizumab in Combination With Topical Corticosteroids in Patients With Atopic Dermatitis (AD) That Are Not Adequately Controlled With or Are Non-eligible for Cyclosporine
The main purpose of this study is to evaluate the efficacy of lebrikizumab compared with placebo in participants not adequately controlled with cyclosporine or for whom cyclosporine is not medically advisable up to Week 16.
- Trial name
- ADvantage (NCT05149313)
- Testing
- Lebrikizumab
- Where
- 48 sites in Austria, Belgium and 6 more
- Volunteers
- 331 people
- Sponsor
- Almirall, S.A.
Intrauterine Retardation
Levels of Endocan in Umbilical Cord Blood Samples of Pregnant Females With Intrauterine Growth Retardation
As a proteoglycan secreted from endothelial cells, expression, and secretion of endocan increases in the endothelium of several tissues secondary to inflammation. It can be regarded as an indicator of endothelial damage.
- Trial name
- NCT04630223
- Testing
- endocan in umbilical cord blood
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Van Bölge Eğitim ve Araştırma Hastanesi
Parkinson's Disease
The Parkinson's Genetic Research Study
The Parkinson's Genetic Research Study (PaGeR), headed by Dr. Cyrus Zabetian, is searching for genes that increase the risk of developing Parkinson's disease (PD) and related disorders.
- Trial name
- PaGeR (NCT01558479)
- Where
- 5 sites in United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- University of Washington
Fabry Disease
Evaluation of Phenotypic Variability in Fabry Disease
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels.
- Trial name
- NCT03145779
- Where
- United States
- Sponsor
- Boston Children's Hospital
Premixed, Sequential
Premixed vs Sequential Injections of Lidocaine and Bupivacaine for Supraclavicular Brachial Plexus Block in Patients Undergoing Arteriovenous Fistula Creation
This study aims to compare premixed and sequential injections of lidocaine and bupivacaine for supraclavicular brachial plexus block in patients undergoing arteriovenous fistula creation.
- Conditions
- Premixed, Sequential, Lidocaine, Bupivacaine
- Trial name
- NCT06570096
- Testing
- Lidocaine 2% then bupivacaine 0.5%, Lidocaine 2% and bupivacaine 0.5%
- Where
- Egypt
- Volunteers
- 100 people
- Sponsor
- Tanta University
Atrial Septal Defect, Patent Foramen Ovale
Canadian Adult Congenital Heart Disease Intervention Registry
The ACHDi Registry study will create a foundational database for adult congenital heart disease interventions. This Pan-Canadian Registry will collect clinical and patient-reported information that will enable the evaluation of…
- Conditions
- Atrial Septal Defect, Patent Foramen Ovale, Tetralogy of Fallot, Fontan
- Trial name
- C-ACHDiR (NCT04288596)
- Testing
- Participants undergoing ACHD intervention
- Where
- Canada
- Volunteers
- 9,000 people
- Sponsor
- University Health Network, Toronto
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Cystic Fibrosis
Comparison of Absorption of Vitamin D in Cystic Fibrosis
The investigators predict that a powder pill form of vitamin D will be more effectively absorbed than an oil form of vitamin D in people diagnosed with Cystic Fibrosis.
- Trial name
- NCT01880346
- Testing
- Vitamin D Powder, Vitamin D Oil
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Emory University
Glioblastoma
A Study of DSP-7888 Dosing Emulsion in Combination With Bevacizumab in Patients With Recurrent or Progressive Glioblastoma Following Initial Therapy
This is an event driven, adaptive design, a randomized, active-controlled, multicenter, open-label, parallel groups, Phase 3 study of DSP-7888 Dosing Emulsion plus Bevacizumab versus Bevacizumab alone in patients with recurrent…
- Trial name
- NCT03149003
- Testing
- DSP-7888 Dosing Emulsion, Bevacizumab
- Where
- 61 sites in United States, Canada and 3 more
- Volunteers
- 221 people
- Sponsor
- Sumitomo Pharma America, Inc.
Abnormalities, Blood Coagulation Disorder
Study of Abnormal Blood Clotting in Children With Stroke
Effective treatment and prevention strategies for childhood stroke and porencephaly can only be developed once the causes are understood.
- Conditions
- Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident
- Trial name
- NCT00001927
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Bronchopulmonary Dysplasia
Mesenchymal Stem Cell Therapy for Bronchopulmonary Dysplasia in Preterm Babies
Bronchopulmonary Dysplasia (BPD) is the most frequent disease related to a premature birth, 15-50% of very low birth newborns (<1500 gr.) will develop BPD.
- Trial name
- NCT02443961
- Testing
- Mesenchymal Stem Cell (MSC) therapy
- Where
- 4 sites in Spain
- Volunteers
- 10 people
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Adult Acute Erythroid Leukemia (M6), Adult Acute Megakaryoblastic Leukemia (M7)
Vorinostat in Treating Patients With Acute Myeloid Leukemia
Vorinostat may stop the growth of cancer cells by blocking some of the enzymes needed for their growth. Giving the drug in different ways may kill more cancer cells.
- Conditions
- Adult Acute Erythroid Leukemia (M6), Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT00305773
- Testing
- vorinostat
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Cancer Institute (NCI)
Heart Septal Defects, Ventricular, Atrioventricular Septal Defect
Aberrations in Carnitine Homeostasis in Congenital Heart Disease With Increased Pulmonary Blood Flow
Infants with congenital heart disease and increased pulmonary blood flow have altered carnitine homeostasis that is associated with clinical outcomes; and L-carnitine treatment will attenuate these alterations and improve…
- Trial name
- L-carn (NCT01825369)
- Testing
- IV L-carnitine
- Where
- United States
- Sponsor
- University of California, San Francisco
Normal Middle Ears., Otitis Media Ears.
Observation of Gas Exchange in the Middle Ear
Observation of fluid behavior over a perforated eardrum. A comparison of the behavior of the gas in normal vs. different kinds of otitis media ears.
- Trial name
- NCT00794105
- Where
- Israel
- Sponsor
- Shaare Zedek Medical Center
Colorectal Cancer, Precancerous Condition
Celecoxib in Preventing Colorectal Cancer in Young Patients With a Genetic Predisposition for Familial Adenomatous Polyposis
RATIONALE: Chemoprevention is the use of certain drugs to keep cancer from forming. The use of celecoxib may keep polyps and colorectal cancer from forming in patients with familial adenomatous polyposis.
- Trial name
- NCT00685568
- Testing
- celecoxib
- Where
- 4 sites in United States
- Volunteers
- 22 people
- Sponsor
- M.D. Anderson Cancer Center
Heart Defects, Congenital, Adolescent
Rehabilitation of Adolescents and Young Adults With Congenital Heart Diseases
The investigators aim to measure the impact of a rehabilitation program in congenital cardiology in terms of health related quality of life.
- Conditions
- Heart Defects, Congenital, Adolescent, Young Adult
- Trial name
- QUALIREHAB (NCT03690518)
- Testing
- Cardiac rehabilitation
- Where
- 17 sites in France
- Volunteers
- 142 people
- Sponsor
- University Hospital, Montpellier
Angelman Syndrome
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
Angelman syndrome (AS) is a rare neurogenetic disorder that affects approximately 1 in 15,000 children - approximately 500,000 people worldwide.
- Trial name
- EYEANGEL (NCT06737718)
- Testing
- Eye tracking, Data collection from patients' medical files, questionnaires
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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