Clinical trials: Cerebral Small Vessels Disease
Fabry Disease
Evaluation of Efficacy and Safety of Agalsidase Beta in Heterozygous Females for Fabry Disease
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A.
- Trial name
- HEART (NCT00487630)
- Testing
- recombinant alpha-galactosidase A
- Where
- France
- Volunteers
- 34 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Lupus Erythematosus, Systemic Lupus Erythematosus
Study Evaluating SC291 in Subjects With Severe r/r B-cell Mediated Autoimmune Diseases (GLEAM)
SC291-102 is a Phase 1 study to evaluate SC291 safety and tolerability, preliminary clinical response, cellular kinetics and exploratory assessments for subjects with severe autoimmune diseases.
- Conditions
- Lupus Erythematosus, Systemic Lupus Erythematosus, SLE (Systemic Lupus), Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
- Trial name
- NCT06294236
- Testing
- SC291
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Sana Biotechnology
Cerebral Autosomal Dominant Ateriopathy With Subcortical Infarcts and Leukoencephalopathy
Registry for CADASIL
This study is being done in order to create a registry (list) of people interested in Cerebral Autosomal Dominant Arteriopathy with Subcortical Infarcts and Leukoencephalopathy (CADASIL) research.
- Trial name
- NCT05567744
- Testing
- Registry
- Where
- United States
- Volunteers
- 2,500 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Fabry Disease, Nephropathy
Screening for Fabry Disease in Renal Transplantation
Single centre, prospective pilot study examining the relevance to screen for Fabry disease in a cohort of patients who have undergone renal transplantation for nephropathy of indeterminate cause, vascular nephropathy, diabetic…
- Trial name
- DEFYT (NCT03886714)
- Testing
- Screening for Fabry disease, Screen for α-galactosidase mutation
- Where
- France
- Volunteers
- 592 people
- Sponsor
- University Hospital, Montpellier
Fabry Disease
An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease
This study will evaluate the safety of multiple biweekly intravenous doses of Replagal over 26 weeks in 25 children with Fabry disease and the way in which that agent can improve the health of this patient population.
- Trial name
- NCT00071877
- Testing
- Replagal
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Bladder Cancer, Brain and Central Nervous System Tumors
High-dose ICE With Amifostine
The purpose of the study is to evaluate the combination of amifostine and high dose chemotherapy with blood stem cell support. Amifostine is a druf developed to protect normal tissues against the toxicities of chemotherapy and…
- Conditions
- Bladder Cancer, Brain and Central Nervous System Tumors, Carcinoma of Unknown Primary, Extragonadal Germ Cell Tumor
- Trial name
- NCT00003657
- Testing
- filgrastim, Amifostine, Carboplatin
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- Dana-Farber Cancer Institute
Fabry Disease, Cardiovascular Diseases
the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
The overall objective of this study is to investigate Fabry-related cardiomyopathy and the use of native T1-mapping, coronary microvascular function, cardiac inflammation, and cardiac injury in an effort to improve the ability to…
- Trial name
- RATIFY (NCT06776419)
- Testing
- Cardiac Magnetic Ressonance Imaging, 82Rubidium-positron emission tomography and computer-tomography
- Where
- Denmark
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Caroline Michaela Kistorp
CADASIL
Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL
This study is a single-center, prospective, single-arm, self-controlled clinical trial designed to assess the safety and efficacy of edaravone dexborneol sublingual tablets for blood-brain barrier (BBB) dysfunction in patients…
- Trial name
- NCT07692399
- Testing
- Edaravone Dexborneol Sublingual Tablets
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Huashan Hospital
Intracerebral Hemorrhage, Hemorrhagic Stroke
Outcome Prediction and Recovery After Spontaneous Intracerebral Haemorrhage
Spontaneous intracerebral haemorrhage (ICH) is a severe form of stroke with high mortality and frequent long-term disability. Current tools to predict how an individual patient will recover are limited.
- Trial name
- CR-4-Health (NCT07882784)
- Where
- 3 sites in Austria, Slovenia
- Volunteers
- 300 people
- Sponsor
- University Medical Centre Maribor
Cerebral Small Vessel Diseases, Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy
Retina is a Marker for Cerebrovascular Heath
Cerebral small vessel disease (SVD), present in 80-94% of adults over age 65 years, increases the risk of stroke by 2-fold, and dementia by 2.3-fold. There is currently no treatment to slow SVD progression.
- Conditions
- Cerebral Small Vessel Diseases, Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy, Cerebral Microbleeding, Sporadic White Matter Disease
- Trial name
- NCT04753970
- Testing
- Cilostazol
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Cerebral Small Vessel Diseases, Stroke, Ischemic
Improving Cerebral Blood Flow and Cognition in Patients with Cerebral Small Vessel Disease. the ETLAS-2 Trial
In a randomized controlled trial the feasibility and effect of three months treatment with daily tadalafil, on cerebral blood flow/reactivity and cognition, is investigated in patients with cerebral small vessel disease.
- Trial name
- ETLAS2 (NCT05173896)
- Testing
- Tadalafil 20 MG
- Where
- 2 sites in Denmark
- Volunteers
- 100 people
- Sponsor
- Christina Kruuse
Fabry Disease
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A.
- Trial name
- NCT04046224
- Testing
- ST-920
- Where
- 18 sites in United States, Australia and 5 more
- Volunteers
- 36 people
- Sponsor
- Sangamo Therapeutics
Fabry Disease
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world…
- Trial name
- PEGASO (NCT07109375)
- Testing
- pegunigalsidase alfa
- Where
- 7 sites in Italy
- Volunteers
- 75 people
- Sponsor
- Chiesi Italia
Sepsis, Pre-Eclampsia
Establishment of an ELISA for the Recognition of Procalcitonin Variants in Patients With Hyperprocalcitonemia.
Procalcitonin is a protein consisting of 116 amino-acids which can rapidly rise under inflammatory conditions and sepsis. More than 20 years ago it has been shown that dipeptidylpeptidase-4 (DPP-4) cleaves procalcitonin from the…
- Conditions
- Sepsis, Pre-Eclampsia, Granulomatosis With Polyangiitis, Microscopic Polyangiitis
- Trial name
- NCT05703802
- Testing
- Procalcitonin-variants ELISA-Assay
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- University Hospital Muenster
Fabry Disease, Heart Diseases
Effect of Migalastat on Cardiac Involvement in Fabry Disease
Anderson-Fabry Disease (AFD) is one of the rare lysosomal storage disorders for which a cause - specific therapy is available. Recently, a new specific drug has been marketed, namely Migalastat, a small-molecule pharmacological…
- Trial name
- MAIORA (NCT03838237)
- Testing
- Cardiological evaluation
- Where
- Italy
- Volunteers
- 18 people
- Sponsor
- Ospedale San Donato
Fabry Disease
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
Objective: To explore the safety and tolerability of different doses of EXG110 with Fabre disease
- Trial name
- NCT06539624
- Testing
- EXG110 injection
- Where
- 2 sites in China
- Volunteers
- 12 people
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Fabry Disease
Long Duration Holter ECG in Fabry Disease
The main objective is to assess the occurrence of cardiac arrhythmias and conduction disorders during a three-year follow-up using implantable Holter ECG monitoring in 40 patients with Fabry disease.
- Trial name
- NCT04440254
- Testing
- Implantation (subcutaneous) of a marketed miniaturized Holter ECG recording device
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Cerebral Small Vessel Diseases, Cerebral Small Vessel Ischemic Disease
Cambridge 7 Tesla Cerebral Small Vessel Disease Study
CamSVD is jointly sponsored by the University of Cambridge and Cambridge University Hospitals NHS Foundation Trust. We aim to explore and understand the underlying arterial pathology in Cerebral Small Vessel Disease (SVD) using…
- Conditions
- Cerebral Small Vessel Diseases, Cerebral Small Vessel Ischemic Disease, Lacunar Stroke
- Trial name
- CamSVD (NCT04330222)
- Testing
- 7 Tesla Magnetic Resonance Imaging, Brief Memory and Executive Test
- Where
- United Kingdom
- Volunteers
- 210 people, healthy volunteers welcome
- Sponsor
- University of Cambridge
Non-ischemic Cardiomyopathy, Dilated Cardiomyopathy (DCM)
Multimodal and Multidisciplinary Approach to Optimize Diagnostic, Prognostic, and Therapeutic Management of Patients with Non-ischemic Cardiomyopathies and Arrhythmogenic-inflammatory Phenotypes: a Multicenter, Observational, Retrospective and Prospective Registry Study.
Non-ischemic cardiomyopathies (NICM) represent a heterogeneous group of pathologies characterized by absence of obstructive disease of the epicardial coronary vessels and distinct structural and functional changes of the…
- Conditions
- Non-ischemic Cardiomyopathy, Dilated Cardiomyopathy (DCM), Hypertrophic Cardiomyopathy (HCM), Restrictive Cardiomyopathy
- Trial name
- AINICM (NCT06607471)
- Testing
- Support treatment, cardiac medical treatment, aetiology-specific treatment, device implant, arrhythmia ablation, Support treatment, cardiac medical treatment, aetiology-specific treatment
- Where
- Italy
- Volunteers
- 15,000 people
- Sponsor
- Scientific Institute San Raffaele
ANCA-associated Vasculitis, Granulomatosis With Polyangiitis
Biosimilars of Rituximab in ANCA-associated Vasculitis Compared to the Originator
The goal of this multicentre observational study is to compare the safety and effectiveness of rituximab biosimilars to the originator in Canadian patients with Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis…
- Conditions
- ANCA-associated Vasculitis, Granulomatosis With Polyangiitis, Microscopic Polyangiitis
- Trial name
- BRAVO (NCT05716334)
- Where
- 9 sites in Canada
- Volunteers
- 201 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre