Clinical trials: Physiopathology
Heart Failure
Open-label Extension Study to Evaluate Long-term Safety of Sacubitril/Valsartan in Pediatric Patients With HF
The purpose of this study was to evaluate long-term safety and tolerability and to provide post-trial access to sacubitril/valsartan to eligible participants who successfully completed CLCZ696B2319 (PANORAMA-HF) core study Part 2…
- Trial name
- NCT03785405
- Testing
- sacubitril/valsartan
- Where
- 79 sites in United States, Argentina and 25 more
- Volunteers
- 216 people
- Sponsor
- Novartis Pharmaceuticals
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Friedreich Ataxia
A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
In this study, researchers are learning more about RTA 408, also known as omaveloxolone, BIIB141, or SKYCLARYS®. The main goal of this study is to learn more about the safety of RTA 408 and how it affects physical effort…
- Trial name
- NCT02255435
- Testing
- Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg
- Where
- 11 sites in United States, Australia and 3 more
- Volunteers
- 172 people
- Sponsor
- Biogen
Sudden Cardiac Death, Left Ventricular Dysfunction
LifeVest Safety and Efficacy in Real Life Settings in France
This post-market study is a prospective observational study evaluating the efficacy and safety of the LifeVest in real-life settings.
- Conditions
- Sudden Cardiac Death, Left Ventricular Dysfunction, Cardiac Event, Cardiac Arrythmias
- Trial name
- WEARIT-FR (NCT03319160)
- Testing
- Wearable Cardioverter Defibrillator
- Where
- 2 sites in France
- Volunteers
- 1,164 people
- Sponsor
- Zoll Medical Corporation
Gastroesophageal Reflux Disease
Neonatal Gastro-Esophageal Reflux Disease (GERD) Management Trial
The overall purpose of the investigator's study is to evaluate the causes of and treatment for feeding difficulty in infants with Gastro-esophageal Reflux Disease (GERD). New treatments can be possible only if the cause is known.
- Trial name
- GMT (NCT02486263)
- Testing
- Study Arm - acid suppression plus feeding bundle, Conventional arm - acid suppression only
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- Sudarshan Jadcherla
Spinal Cord Injury
The Effect of FES on Children With Spinal Cord Injury
Regular exercise is strongly recommended to help maintain a healthy lifestyle. Unfortunately, children and young adults with damaged spinal cords may not be able to exercise regularly.
- Trial name
- NCT00291317
- Testing
- RT 300-P FES Cycle
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Children's Specialized Hospital
Mild Persistent Asthma
Response of FeNO, Small Airway Dysfunction and Lung Heterogeneity to 2-week Montelukast Treatment in Asthmatic Children.
This study aims to identify markers to prove rapid improvement of lung function, airway inflammation and bronchodilator response after 2-week LTRA administration.
- Trial name
- NCT01581710
- Volunteers
- 32 people
- Sponsor
- CHA University
Psychological, Sexual Dysfunction
The Impact of Chronic Adolescent Skin Conditions on Sexual Health
Background: Skin conditions are common in young people and are known to adversely affect emotional well-being and quality of life in a range of ways, including negative impact on intimate relationships.
- Conditions
- Psychological, Sexual Dysfunction, Adolescent Development, Interaction, Social
- Trial name
- NCT06397664
- Where
- United Kingdom
- Volunteers
- 91 people
- Sponsor
- Oxford University Hospitals NHS Trust
Sturge-Weber Syndrome
Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
The purpose of this study is to better understand the utility of cannabidiol (CBD/ Epidiolex) for improving the treatment of cognitive impairments in Sturge-Weber syndrome (SWS).
- Trial name
- NCT04447846
- Testing
- Cannabidiol
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Anne Comi, MD
Chronic Inflammation, Chronic Pain
Gulf War Illness Inflammation Reduction Trial
The primary objective of this clinical trial is to determine if treatment with an anti-inflammatory drug (delayed-release prednisone) improves the health-related quality of life (HRQOL) of veterans with Gulf War Illness (GWI).
- Conditions
- Chronic Inflammation, Chronic Pain, Fatigue, Cognitive Impairment
- Trial name
- GWIIRT (NCT02506192)
- Testing
- Modified-Release Prednisone
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Minneapolis Veterans Affairs Medical Center
ADHD, Attention Deficit Hyperactivity Disorder
Study of Medication Patch to Treat Children Ages 6-12 With ADHD
The purpose of this study is to investigate the effectiveness of a medication skin patch called Methylphenidate Transdermal System (MTS). We will compare the MTS medicated patch to a placebo patch.
- Trial name
- NCT00586157
- Testing
- Methylphenidate Transdermal System
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Massachusetts General Hospital
Meningococcal Disease
Safety and Immunogenicity of Novartis Meningococcal B Vaccine When Administered to Immunocompromised Children and Adolescents Compared to Healthy Subjects.
The study aims at evaluating the safety and immunogenicity of rMenB+OMV NZ when administered to subjects from 2 to 17 years of age with increased risk of meningococcal disease because either of primary or secondary complement…
- Trial name
- NCT02141516
- Testing
- rMenB+OMV
- Where
- 20 sites in Italy, Poland and 3 more
- Volunteers
- 239 people, healthy volunteers welcome
- Sponsor
- Novartis
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Congenital Heart Disease, Bradycardia Sinus
Specialized Pacing for Patients With Congenital Heart Disease
The closed-loop stimulation (CLS) algorithm is a novel sensor-based technology that relies on the change in myocardial systolic impedance for modulation of the heart rate during physical and emotional stress.3 The pacing…
- Trial name
- NCT03361189
- Testing
- Closed loop stimulation, Standard rate response
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, Los Angeles
Duchenne Muscular Dystrophy (DMD)
Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
Idebenone is a synthetic analogue of coenzyme Q10 and is a powerful antioxidant and essential constituent of the process of energy production on the cellular level.
- Trial name
- DELPHI (NCT00654784)
- Testing
- idebenone
- Where
- Belgium
- Volunteers
- 21 people
- Sponsor
- Santhera Pharmaceuticals
Phenylketonuria
Phenylketonuria, Oxidative Stress, and BH4
The purpose of this study is to see how tetrahydrobiopterin therapy (BH4; also known as sapropterin dihydrochloride or Kuvan) affects measures of oxidative stress and endothelial function in patients with Phenylketonuria (PKU).
- Trial name
- NCT01395394
- Testing
- Kuvan, Meal Challenge
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Emory University
Heart Failure, Congestive, Ventricular Dysfunction
Thoratec HeartMate II Left Ventricular Assist System (LVAS) for Bridge to Cardiac Transplantation
The purpose of this study is to determine the safety and effectiveness of the Thoratec HeartMate II Left Ventricular Assist System (LVAS) as a bridge to cardiac transplantation in end-stage heart failure patients who are listed…
- Conditions
- Heart Failure, Congestive, Ventricular Dysfunction, Cardiomyopathies
- Trial name
- NCT00121472
- Testing
- Thoratec HeartMate II Left Ventricular Assist System (LVAS)
- Where
- 36 sites in United States, Canada
- Volunteers
- 194 people
- Sponsor
- Abbott Medical Devices
Autism, Cognition Disorder
The Effect of Donepezil (Aricept(Registered Trademark)) on REM Sleep in Children With Autism
This study will test whether donepezil (Aricept(Registered Trademark)), a drug that is approved by the Food and Drug Administration to treat Alzheimer's disease, can increase rapid eye movement (REM) sleep in children with autism…
- Trial name
- NCT00695136
- Testing
- Donepezil hydrochloride, Increase REM sleep percentage
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- National Institute of Mental Health (NIMH)
Down Syndrome
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
The purpose of this study is to determine whether donepezil HCl is effective and safe in improving cognitive dysfunction exhibited by children and adolescents with Down syndrome (DS).
- Trial name
- NCT00570128
- Testing
- Donepezil HCl
- Where
- 31 sites in United States
- Volunteers
- 129 people
- Sponsor
- Eisai Inc.
Stunting, Environmental Enteric Dysfunction
Safety, Acceptability, and Feasibility of Enterade®
This is a randomized, double-blinded, placebo-controlled pilot study to determine the safety, acceptability, and feasible pediatric dosage/tolerability of enterade® solution, an amino acid-based oral rehydration solution…
- Trial name
- SAFE (NCT03782272)
- Testing
- Enterade
- Where
- Kenya
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- PATH