Clinical trials: Clinical Disease and/or Syndrome
Chronic Graft-versus-host-disease
A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received…
- Trial name
- AGAVE-201 (NCT04710576)
- Testing
- Axatilimab
- Where
- 121 sites in United States, Australia and 14 more
- Volunteers
- 296 people
- Sponsor
- Syndax Pharmaceuticals
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
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
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
FSGS
A Study of the Efficacy and Safety of DMX-200 in Patients With FSGS Who Are Receiving an ARB
DMX-200 (repagermanium) is a C-C chemokine receptor type 2 (CCR2) inhibitor that, when administered concurrently with an ARB, is designed to inhibit recruitment of monocytes implicated in the inflammatory chemokine environment of…
- Trial name
- ACTION3 (NCT05183646)
- Testing
- DMX-200
- Where
- 220 sites in United States, Argentina and 19 more
- Volunteers
- 286 people
- Sponsor
- Dimerix Bioscience Pty Ltd
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)
Tuberculosis, Children, Only
Integrating Pediatric TB Services Into Child Healthcare Services in Africa
Under-diagnosis of TB in children is a critical gap to address. The INPUT study is a multinational stepped-wedge cluster-randomized intervention study aiming to assess the effect of integrating TB services into child healthcare…
- Conditions
- Tuberculosis, Children, Only, Diagnosis, Delivery of Health Care
- Trial name
- INPUT (NCT03862261)
- Testing
- Integrated pediatric TB services
- Where
- 12 sites in Cameroon, Kenya
- Volunteers
- 1,715 people
- Sponsor
- Elizabeth Glaser Pediatric AIDS Foundation
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
Patellar Dislocation, Patellar Dislocation, Recurrent
PROMs and Patellar Tracking After MPFL Reconstruction With or Without Tibial Tuberosity Transposition
Patellar dislocations cause pain and functional decline in adolescents, which can be restored by a MPFL reconstruction with or without tibial tuberosity transposition.
- Conditions
- Patellar Dislocation, Patellar Dislocation, Recurrent, Patellofemoral Maltracking
- Trial name
- NCT05547269
- Where
- Netherlands
- Volunteers
- 11 people
- Sponsor
- Radboud University Medical Center
Invasive Group A Beta-Haemolytic Streptococcal Disease
Paediatric European Group A Streptococcal United Study.
Introduction Since 2022 there are reports of a rapid increase in invasive group A streptococcal infections (iGAS) in children in some but not all European countries. Detailed information on this increase is lacking.
- Trial name
- PEGASUS (NCT06058117)
- Testing
- No intervention
- Where
- Netherlands
- Volunteers
- 500 people
- Sponsor
- Noordwest Ziekenhuisgroep
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
Alzheimer Disease
Finding Retinal Biomarkers in Alzheimer's Disease
CSF Alzheimer's disease (AD) biomarkers are the only one that reflect both Aβ and tau pathologies. There is increasing evidence for the presence of AD abnormalities in the retina of AD patients.
- Trial name
- FIREBALZ (NCT05067010)
- Testing
- Detailed ophthalmologic examination
- Where
- France
- Volunteers
- 93 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemangiomas, Vascular Malformations
Diagnosis of Hemangiomas and Vascular Malformations
To develop a non-invasive, simple and affordable, measurement technique to differentiate hemangiomas from subtypes of VMS. The purpose of this study is to determine the best office based diagnositc instrument for differentiating…
- Conditions
- Hemangiomas, Vascular Malformations, Venular Malformations, Venous Malformations
- Trial name
- NCT00577213
- Volunteers
- 56 people
- Sponsor
- University of Arkansas
End Stage Renal Disease
Prognostic Value of Central and BracHial Ambulatory Blood Pressure Monitoring in ERSD Patients Treated With HeMOdialysis
This is a prospective cohort study which aims to explore the significance of brachial and central ambulatory blood pressure monitoring in predicting cardiovascular risk in patients with end stage renal disease who are treated…
- Trial name
- C-HEMO (NCT03306160)
- Testing
- 48-hour blood pressure ambulatory monitoring
- Where
- China
- Volunteers
- 900 people
- Sponsor
- Shanghai 10th People's Hospital
Bicuspid Aortic Valve Disease
Bicuspid Echocardiac Study Team（BEST）
The Chinese Bicuspid Aortic Valve (BAV) Ultrasound imaging cohort study is a Chinese BAV ultrasound imaging cohort study. At present, a retrospective study cohort involving more than 30 hospitals has been established.
- Trial name
- BEST (NCT05656820)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Suzhou Municipal Hospital
Patellar Dislocation, Recurrent, Patellar Dislocation
JUPITER 4.0 - Risk Factors for Failure of Isolated Medial Patellofemoral Ligament Reconstruction
The goal of this observational study is to learn about the outcomes of medial patellofemoral ligament (MPFL) reconstruction for the treatment of recurrent patellar instability.
- Conditions
- Patellar Dislocation, Recurrent, Patellar Dislocation, Patellar Instability, Patellofemoral Dislocation
- Trial name
- NCT06883396
- Testing
- Medial Patellofemoral Ligament (MPFL) Reconstruction
- Where
- 16 sites in United States
- Volunteers
- 850 people
- Sponsor
- Hospital for Special Surgery, New York
Amnesia, Dementia
Magnetic Resonance Imaging for the Study of Patients With Neurological Disorders
This study is divided into two parts. The first part of the study will use MRI technology to view the brain structure of patients with neurological disorders and normal volunteers.
- Conditions
- Amnesia, Dementia, Healthy, Nervous System Disease
- Trial name
- NCT00001362
- Where
- United States
- Volunteers
- 830 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Neuroendocrine Tumors, Progression
Everolimus 5 mg vs 10 mg/Daily for Patients With Neuroendocrine Tumors
Everolimus is approved in many countries to treat patients with advanced/metastatic well-differentiated neuroendocrine tumors (NET), providing median progression-free survival times of approximately 12 months across different…
- Conditions
- Neuroendocrine Tumors, Progression, Neuroendocrine Tumor Grade 1, Neuroendocrine Tumor Grade 2
- Trial name
- EVENET (NCT06472388)
- Testing
- Everolimus 5 MG
- Where
- 2 sites in Brazil
- Volunteers
- 100 people
- Sponsor
- AC Camargo Cancer Center
Microbial Colonization, Neuroblastoma
Investigating the Microbiome and Volatile Organic Compound Profile of Children With Neuroblastoma
Background: Malignant tumors may lead to a catabolic state with loss of muscle and adipose tissue. The full picture of catabolism is termed cachexia and is associated with significant morbidity and mortality of cancer patients.
- Conditions
- Microbial Colonization, Neuroblastoma, Children, Only
- Trial name
- NCT03545542
- Testing
- Initial fecal microbiome, Initial fecal volatile organic compounds, Initial breath volatile organic compounds
- Where
- Austria
- Volunteers
- 20 people
- Sponsor
- Medical University of Graz
Myositis, Dermatomyositis
Environmental Risk Factors for the Anti-synthetase Syndrome
Background: Like other complex diseases, autoimmune diseases are the result of numerous causes, including genetic and environmental factors.
- Conditions
- Myositis, Dermatomyositis, Polymyositis, Juvenile Dermatomyositis
- Trial name
- NCT01276470
- Where
- 8 sites in United States
- Volunteers
- 580 people, healthy volunteers welcome
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
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