Clinical trials: Cerebral Small Vessels Disease
ESRD, Fabry Disease
Kidney Information Network for Disease Research and Education
In this study, Investigators will conduct a prospective cohort study of dialysis patients by collecting research-quality information on patient characteristics, comorbid diseases and laboratory markers used in routine practice…
- Conditions
- ESRD, Fabry Disease, Rare Diseases, Kidney Diseases
- Trial name
- KINDRED (NCT03321604)
- Where
- United States
- Volunteers
- 338 people
- Sponsor
- Massachusetts General Hospital
Fabry Disease
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat)…
- Trial name
- CARAT (NCT05280548)
- Testing
- Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828)
- Where
- 54 sites in United States, Austria and 17 more
- Volunteers
- 104 people
- Sponsor
- Sanofi
Microscopic Polyangiitis (MPA), Granulomatosis With Polyangiitis (GPA)
Special Drug Use-results Survey for Long-term Use（Avacopan）
The purpose of this survey is evaluating the safety and efficacy of long-term administration of avacopan for Japanese patients with microscopic polyangiitis or granulomatosis with polyangiitis under actual conditions of use.
- Trial name
- NCT06758271
- Testing
- Avacopan
- Where
- Japan
- Volunteers
- 250 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Fabry Disease
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
- Trial name
- MODERN (NCT06663358)
- Testing
- Pegunigalsidase-alfa
- Where
- 10 sites in United States, Slovenia and 1 more
- Volunteers
- 100 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Chronic Pain in the Extremities, Chronic Abdominal Pain
Screening for Fabry Disease in a Pediatric Population at Risk
The purpose of this study is to assess the frequency of Fabry disease in children with chronic abdominal pain or chronic pain in the extremities
- Conditions
- Chronic Pain in the Extremities, Chronic Abdominal Pain, Fabry Disease
- Trial name
- NCT02152189
- Testing
- Screening for Fabry disease
- Where
- Germany
- Volunteers
- 1,422 people
- Sponsor
- University Hospital Heidelberg
Fabry Disease
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase…
- Trial name
- BRIGHT (NCT03180840)
- Testing
- Pegunigalsidase alfa
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Protalix
Atherosclerotic Carotid Disease, Atheroma
Effects Of Atorvastatin On Macrophage Activity And Plaque Inflammation Using Magnetic Resonance Imaging
A new way of scanning narrowing in the arteries (main blood vessels) caused by fatty deposits known as plaques has been developed. Heart attacks and strokes occur when plaques become inflamed, depending on the artery affected.
- Conditions
- Atherosclerotic Carotid Disease, Atheroma, Atherosclerosis
- Trial name
- NCT00368589
- Testing
- atorvastatin
- Where
- United Kingdom
- Volunteers
- 47 people
- Sponsor
- GlaxoSmithKline
MELAS Syndrome, Ketogenic Dieting
Effectiveness of Ketogenic Diet in MELAS Syndrome
The goal of this clinical trial is to evaluate the effectiveness of ketogenic diet in patients with MELAS syndrome. The main questions it aims to answer are: Clarify the curative effects of ketogenic diet in the treatment of…
- Trial name
- NCT06013397
- Testing
- Ketogenic diet
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Acute Ischemic Stroke, Haemorrhagic Stroke
Translational Immunodiagnostics in Stroke (TrImS)
In adult patients presenting to emergency departments within 24 hours of symptom onset with suspected acute stroke, we aim: 1. to identify early brain- and pathology-specific circulating, whole blood, plasma and serum panorOmic…
- Conditions
- Acute Ischemic Stroke, Haemorrhagic Stroke, Transient Ischemic Attacks, Cardioembolic Stroke
- Trial name
- TrImS (NCT05300997)
- Testing
- Biomarker blood draw and saliva collection
- Where
- China
- Volunteers
- 650 people, healthy volunteers welcome
- Sponsor
- The University of Hong Kong
Fabry Disease
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
- Trial name
- NCT01476163
- Testing
- migalastat HCl 150 mg, migalastat HCl 20 mg
- Sponsor
- Amicus Therapeutics
Fabry's Disease
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume.
- Trial name
- SHORTEN (NCT06019728)
- Testing
- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sanofi
Left Ventricular Hypertrophy
Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy
The purpose of this study is to determine the prevalence of Fabry mutations in patients with left ventricular hypertrophy (moderate to severe), as measured by echocardiography.This study is a screening study
- Trial name
- NCT01198899
- Testing
- blood sampling
- Where
- 9 sites in Belgium
- Volunteers
- 540 people
- Sponsor
- University Hospital, Ghent
ANCA Associated Systemic Vasculitis Including Wegener's, Granulomatosis and Microscopic Polyangiitis and
IMPROVE: Mycophenolate Mofetil Versus Azathioprine for Maintenance Therapy in ANCA Associated Systemic Vasculitis
The aim of IMPROVE is to define the optimal maintenance therapy for ANCA-associated vasculitides (AASV) by comparing the AZA (standard regimen) with MMF in terms of efficacy, i.e. in preventing relapses.
- Conditions
- ANCA Associated Systemic Vasculitis Including Wegener's, Granulomatosis and Microscopic Polyangiitis and, Renal Limited Vasculitis
- Trial name
- NCT00307645
- Testing
- Cyclophosphamide, Mycophenolate mofetil, Azathioprine
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 160 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Small for Gestational Age at Delivery, Hypoxic-Ischemic Encephalopathy
Umbilical Cord Abnormalities in the Prediction of Adverse Pregnancy Outcomes
The objective of our study is to investigate the association of umbilical cord abnormalities with adverse pregnancy outcomes. The umbilical cord parameters that will be investigated as part of this study include the umbilical…
- Conditions
- Small for Gestational Age at Delivery, Hypoxic-Ischemic Encephalopathy, Stillbirth, Admission to Neonatal Unit
- Trial name
- ULOOP (NCT05901688)
- Testing
- Umbilical cord measurements & observations
- Where
- United Kingdom
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Medway NHS Foundation Trust
Fabry Disease
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male…
- Trial name
- NCT03737214
- Testing
- Lucerastat
- Where
- 42 sites in United States, Australia and 11 more
- Volunteers
- 107 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Cadasil, Migraine
Safety and Efficacy of Fremanezumab for Migraine in Adult CADASIL
Researchers are trying to find out more about the side effects of fremanezumab when treating patients with Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) for migraine…
- Trial name
- NCT04334408
- Testing
- Fremanezumab
- Sponsor
- Mayo Clinic
Cerebrovascular Disease Small Vessel
Study On the Expression Level and Clinical Significance of VISTA in Patients With Vascular Cognitive Impairment
1. Explore the expression level of immune checkpoint VISTA in peripheral blood and cerebrospinal fluid of patients with cardiovascular risk factors; 2.
- Trial name
- NCT06342661
- Testing
- Neuropsychological testing
- Where
- China
- Volunteers
- 400 people
- Sponsor
- First Affiliated Hospital of Harbin Medical University
Small Vessel Cerebrovascular Disease
Development of a Global Scale Assessing Impairment in Cerebral Small Vessel Diseases
In daily practice, several scales are used to evaluate patients with small vessel diseases of the brain (SVD). However, these scales exclude key symptoms such as apathy and mood disorders observed in SVD.
- Trial name
- SMACS (NCT04170673)
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Fabry Disease, Amyloidosis
Substudy (NCT04456582): Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Two-dimensional Shear Wave Elastography) in Patients With Amyloidosis and Fabry Disease.
Introduction: Heart failure with preserved systolic function encompasses several different diseases, but which have diastolic dysfunction and its components in common: myocardial stiffness and altered relaxation.
- Trial name
- FABRY (NCT04893889)
- Testing
- Shear-wave elastography
- Where
- Brazil
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo General Hospital
Alzheimer's Disease, Small Vessel Disease White Matter Hyperintensities Subtype
Amyloid and Glucose PET Imaging in Alzheimer and Vascular Cognitive Impairment Patients With Significant White Matter Disease
The prevalence of both Alzheimer's Disease (AD) and stroke doubles each decade over 65 years old. Both are major causes of dementia, currently estimated to affect 46 million people worldwide.
- Conditions
- Alzheimer's Disease, Small Vessel Disease White Matter Hyperintensities Subtype, Vascular Cognitive Impairment
- Trial name
- MITNEC C6 (NCT02330510)
- Where
- 8 sites in Canada
- Volunteers
- 80 people
- Sponsor
- Dr. Sandra E Black