Clinical trials: Cerebral Small Vessel Diseases
Fabry Disease
Safety Study of Replagal® Therapy in Children With Fabry Disease
The purpose of this study is to assess the safety of Replagal in children with Fabry disease who who have not previously been treated with enzyme replacement therapy (ERT).
- Trial name
- NCT01363492
- Testing
- Replagal (agalsidase alfa)
- Where
- 5 sites in United States
- Volunteers
- 15 people
- Sponsor
- Shire
Cancer
Cyproheptadine in Preventing Weight Loss in Children Receiving Chemotherapy for Cancer
RATIONALE: Cyproheptadine hydrochloride may prevent weight loss caused by cancer or cancer treatment. It is not yet known whether cyproheptadine is more effective than a placebo in preventing weight loss in young patients…
- Trial name
- NCT01132547
- Testing
- cyproheptadine hydrochloride
- Where
- 14 sites in United States
- Volunteers
- 22 people
- Sponsor
- University of South Florida
Fabry Disease
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
The objective of CLI-06657AA1-04 (formerly PB-102-F60) was to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry…
- Trial name
- NCT03566017
- Testing
- pegunigalsidase alfa
- Where
- 30 sites in United States, Australia and 11 more
- Volunteers
- 97 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the long-term safety of GZ/SAR402671 in adult male participants with Fabry disease who previously completed study ACT13739 (NCT02489344).
- Trial name
- NCT02489344
- Testing
- GZ/SAR402671
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 8 people
- Sponsor
- Genzyme, a Sanofi Company
Vasculitis
Belimumab in Remission of VASculitis
The purpose of this study is to evaluate the efficacy and safety of belimumab, in combination with azathioprine, for the maintenance of remission following a standard induction regimen in patients with Wegener's granulomatosis or…
- Trial name
- BREVAS (NCT01663623)
- Testing
- Belimumab 10 mg/kg, Azathioprine
- Where
- 89 sites in United States, Australia and 18 more
- Volunteers
- 106 people
- Sponsor
- Human Genome Sciences Inc., a GSK Company
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Fabry Disease
Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients
This study aimed to evaluate blood oxygenation at the optic nerve head in relation with visual field losses observed in many Fabry patients. Data collected allowed to evaluate if there is a link between these two entities.
- Trial name
- NCT02023086
- Testing
- Contrast sensitivity measurement, Slit Lamp assessment and intra-ocular pressure measurement, Visual field testing
- Where
- Canada
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Université de Montréal
Granulomatosis With Polyangiitis, Microscopic Polyangiitis
Pilot Study of Short-Course Glucocorticoids and Rituximab for Treatment of ANCA-Associated Vasculitis
The purpose of this pilot study is to test whether an 8-week course of glucocorticoids, combined with rituximab, is effective in treating ANCA-associated vasculitis.
- Trial name
- SCOUT (NCT02169219)
- Testing
- Glucocorticoids, Rituximab
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Massachusetts General Hospital
Depression, Stress, Psychological
Rural Dementia Caregiver Project
Rural caregivers face challenges of geographic isolation and lower health care access and quality. Many rural dementia caregivers experience serious health consequences due to caregiving responsibilities that can limit their…
- Conditions
- Depression, Stress, Psychological, Self Efficacy, Loneliness
- Trial name
- NCT04428112
- Testing
- Building Better Caregivers Workshop, Attention Control
- Where
- United States
- Volunteers
- 418 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Fabry Disease, Lysosomal Storage Diseases
A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme.
- Trial name
- NCT04455230
- Testing
- FLT190
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
Fabry Disease
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
- Trial name
- NCT04049760
- Testing
- migalastat HCl 150 mg
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
- Trial name
- NCT01304277
- Testing
- agalsidase alfa
- Where
- 5 sites in Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease.
- Trial name
- NCT01298141
- Testing
- agalsidase alfa
- Where
- 12 sites in Canada
- Volunteers
- 171 people
- Sponsor
- Shire
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
Fabry Disease
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
- Trial name
- MODIFY (NCT03425539)
- Testing
- Lucerastat
- Where
- 49 sites in United States, Australia and 12 more
- Volunteers
- 118 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Fabry's Disease
Evaluation of the Gastrointestinal Manifestation of Fabry's Disease
Patients will undergo a SmartPill test to gain additional understanding of Fabry disease manifestation via motility abnormalities in order to improve symptom targeted therapy.
- Trial name
- NCT02798458
- Testing
- Smartpill, Endoscopic Mucosal Resection
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Massachusetts General Hospital
Hand Function, Stroke
TheraBracelet Phase I
This project aims to test a new technology that may improve hand function impaired from a stroke, thereby improving independence and quality of life.
- Conditions
- Hand Function, Stroke, Upper Extremity, Hemiparesis
- Trial name
- NCT03318341
- Testing
- Real Stimulation
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- TheraBracelet, Inc.
Aging, Arterial Stiffness
Inorganic Nitrate Supplementation on Cerebrovascular Aging and Arterial Stiffness Study
This study investigates the effect of dietary inorganic nitrate supplementation on 1) large elastic artery stiffness and hemodynamics and 2) cerebrovascular function in middle-aged and older adults.
- Conditions
- Aging, Arterial Stiffness, Endothelial Dysfunction, Hypertension
- Trial name
- INCA (NCT03617302)
- Testing
- Experimental: Nitrate-containing
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Gary L. Pierce, PhD
Granulomatosis With Polyangiitis (Wegener's) (GPA), Microscopic Polyangiitis (MPA)
Plasma Exchange and Glucocorticoids for Treatment of Anti-Neutrophil Cytoplasm Antibody (ANCA) - Associated Vasculitis
The purpose of this study is to determine whether plasma exchange as well as immunosuppressive therapy are effective in reducing death and end-stage renal disease (ESRD).
- Trial name
- PEXIVAS (NCT00987389)
- Testing
- Plasma Exchange, No Plasma Exchange, Glucocorticoids [Standard Dose]
- Where
- 98 sites in United States, Australia and 13 more
- Volunteers
- 704 people
- Sponsor
- University of Pennsylvania
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire