Clinical trials: Cerebral Small Vessel Disease
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
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis, Fabry Disease
Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer.
The aim of the present study is to compare intraocular pressure (IOP) values assessed with Ocular Response Analyzer to the classical gold standard of IOP measurement, to Goldmann applanation tonometry by mucopolysacchyridosis…
- Trial name
- MPSORA (NCT01695161)
- Where
- Germany
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Johannes Gutenberg University Mainz
Anaplastic Astrocytoma, Anaplastic Ependymoma
SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors
Approximately 90% of children with malignant brain tumors that have recurred or relapsed after receiving conventional therapy will die of disease.
- Conditions
- Anaplastic Astrocytoma, Anaplastic Ependymoma, Anaplastic Ganglioglioma, Anaplastic Meningioma
- Trial name
- NCT03434262
- Testing
- Gemcitabine, ribociclib, sonidegib
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- St. Jude Children's Research Hospital
Childhood Burkitt Lymphoma, Childhood Central Nervous System Choriocarcinoma
Vorinostat and Bortezomib in Treating Young Patients With Refractory or Recurrent Solid Tumors, Including Central Nervous System Tumors and Lymphoma
This phase I trial is studying the side effects and best dose of vorinostat when given together with bortezomib in treating young patients with refractory or recurrent solid tumors, including CNS tumors and lymphoma.
- Conditions
- Childhood Burkitt Lymphoma, Childhood Central Nervous System Choriocarcinoma, Childhood Central Nervous System Germ Cell Tumor, Childhood Central Nervous System Germinoma
- Trial name
- NCT00994500
- Testing
- vorinostat, bortezomib, pharmacological study
- Where
- 6 sites in United States
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Fabry Disease
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
The primary objective of this study was to compare the effect of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) versus placebo on kidney globotriaosylceramide (GL-3).
- Trial name
- NCT00925301
- Testing
- migalastat hydrochloride
- Where
- 28 sites in United States, Argentina and 11 more
- Volunteers
- 67 people
- Sponsor
- Amicus Therapeutics
Intracerebral Hemorrhage, CAA - Cerebral Amyloid Angiopathy
MRI-markers to Monitor Small Vessel Disease Dynamics in the Prognosis of Small Vessel Disease-associated, Cerebrovascular Events
This is a nested cohort study in the PRO-SVD cohort. Small vessel disease is a chronic disease and is thought to progress over time.
- Conditions
- Intracerebral Hemorrhage, CAA - Cerebral Amyloid Angiopathy, Cerebral Small Vessel Diseases
- Trial name
- MRI-PRO-SVD (NCT05773235)
- Testing
- Combined 3- and 7 Tesla-MRI
- Where
- Switzerland
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Fabry Disease
Enzyme Replacement Therapy in Fabry Disease
Fabry disease is an X-linked rare metabolic disease, caused by a deficient activity of the hydrolase α-Galactosidase A, and characterized by a progressive and systematic deposition of glycosphingolipids in many organs.
- Trial name
- NCT00149318
- Testing
- Biochemical analyses.
- Where
- Italy
- Volunteers
- 2 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Cataract, Vitreous Hemorrhage
Study on Eye-Brain Cross-Organ Mapping and Systemic Disease Association Based on Multimodal Big Data
This project integrates multimodal eye data (CFP, OCT, OCTA) from 50,000 cases, brain MR data from 150,000 cases, and ICD diagnoses, medical orders, and test results from an eye-brain paired cohort of 8,000 cases to construct an…
- Conditions
- Cataract, Vitreous Hemorrhage, Diabetic Eye Diseases, Cerebral Small Vessel Disease
- Trial name
- NCT07541651
- Testing
- No interventions were involved in this study.
- Volunteers
- 208,000 people, healthy volunteers welcome
- Sponsor
- Beijing Friendship Hospital
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
Fabry Disease, Healthy
PET Scans in Normal Volunteers and Patients With Fabry Disease
This study will determine the rate of sugar metabolism in the brain of patients with Fabry disease, a genetic disease of abnormal lipid metabolism.
- Trial name
- NCT00005111
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Fabry Disease
To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Versus Continuing on Agalsidase Alfa (Replagal) in Male Patients With Classic Fabry Disease
Primary Objective: To assess reduction of plasma lyso-GL3 level after switch to agalsidase beta from agalsidase alfa Secondary Objectives: To assess reduction of kidney podocyte GL3 content after switch to agalsidase beta from…
- Trial name
- BCLEAR1 (NCT04143958)
- Testing
- agalsidase beta (GZ419828), agalsidase alfa
- Sponsor
- Sanofi
COL4A1\2, COL4A1-Related Brain Small Vessel Disease With Haemorrhage
COL4A1COL4A2: Study of Pathological Conditions Involving Multiple Organs Caused by Mutations in the COL4A1 and COL4A2 Genes
This observational and diagnostic study aims to better understand the clinical features and biological mechanisms associated with mutations in the COL4A1 and COL4A2 genes, which are known to cause a rare inherited small-vessel…
- Trial name
- NCT07374913
- Testing
- Ophtalmological and cardiological screening
- Where
- Italy
- Volunteers
- 120 people
- Sponsor
- Meyer Children's Hospital IRCCS
Brain and Central Nervous System Tumors, Lymphoma
Intrathecal Rituximab in Treating Patients With Recurrent CNS Lymphoma
RATIONALE: Monoclonal antibodies, such as rituximab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Trial name
- NCT00416923
- Testing
- rituximab
- Volunteers
- 10 people
- Sponsor
- University of California, San Francisco
Fabry Disease
Data Collection in Women With Fabry Disease
This protocol will collect information needed to design a clinical study for the symptoms and problems of women with Fabry disease, an inherited metabolic disorder.
- Trial name
- NCT00030134
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Ischemic Stroke Hospitalized
Fabry : National Initiative of Screening
Fabry disease is a genetic disease due to an enzymatic deficit. A screening of this disease allows patients to benefit from an enzyme replacement therapy and prevent the occurrence of life threatening manifestations such as an…
- Trial name
- FIND (NCT00484549)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 889 people
- Sponsor
- University Hospital, Clermont-Ferrand
Cerebral Amyloid Angiopathy, Small Vessel Cerebrovascular Disease
Prognosis of Cerebral Small Vessel Disease
Prognosis of small vessel disease (SVD) depends on the underlying type of SVD and index manifestation. The aim of this prospective, observational cohort study is to determine the risk of different outcome events among patients…
- Conditions
- Cerebral Amyloid Angiopathy, Small Vessel Cerebrovascular Disease, Cadasil, CAA - Cerebral Amyloid Angiopathy
- Trial name
- PRO-SVD (NCT05734378)
- Where
- Switzerland
- Volunteers
- 900 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Mitochondrial Disorders, Mitochondrial Disease
Tissue Sample Study for Mitochondrial Disorders
The investigators are studying patients with undefined mitochondrial diseases to identify genetic mutations in nuclear or mitochondrial Deoxyribonucleic Acid (DNA).
- Conditions
- Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer
- Trial name
- NCT01803906
- Where
- United States
- Volunteers
- 6,900 people, healthy volunteers welcome
- Sponsor
- Columbia University
Pain, Fabry's Disease
Prevalence of Fabry's Disease in a Population of Patients With Chronic Pains
Fabry disease (FD) is a rare X-linked multisytemic lysosomal disorder caused by alpha-galactosidase deficiency. Globotriaosylcéramide (Gb3) deposits are observed in almost all tissues examined.
- Trial name
- DOUFAB (NCT01178164)
- Testing
- Blood sampling for biological and genetic analysis
- Where
- France
- Volunteers
- 137 people
- Sponsor
- University Hospital, Bordeaux
Fabry Disease
Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)
This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition.
- Trial name
- NCT04639999
- Testing
- Echocardiography
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- Yonsei University