Clinical trials: Cerebral Small Vessels Disease
Granulomatosis With Polyangiitis (GPA), Microscopic Polyangiitis (MPA)
Study of IFX-1 to Replace Steroids in Patients With Granulomatosis With Polyangiitis and Microscopic Polyangiitis.
The purpose of the study is to evaluate the efficacy of IFX-1 treatment as replacement for glucocorticoid (GC) therapy in subjects with polyangiitis (GPA) or microscopic polyangiitis (MPA).
- Trial name
- IXchange (NCT03895801)
- Testing
- IFX-1, Glucocorticoid (GC)
- Where
- 76 sites in Belgium, Czechia and 9 more
- Volunteers
- 57 people
- Sponsor
- InflaRx GmbH
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Cerebral Small Vessel Diseases, Stroke, Lacunar
LACunar Intervention (LACI-2) Trial-2
About 35,000 people each year in the UK have a type of stroke, called 'lacunar' or 'small vessel' stroke, which is different to other common types of stroke and for which there is no proven treatment.
- Trial name
- LACI-2 (NCT03451591)
- Testing
- Isosorbide Mononitrate XL (ISMN), Cilostazol, ISMN XL and Cilostazol
- Where
- United Kingdom
- Volunteers
- 363 people
- Sponsor
- University of Edinburgh
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
MELAS Syndrome
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
The purpose of this study is to compare the efficacy of two (2) different doses of idebenone with that of a placebo over a one month period on cerebral lactate concentration as measured by magnetic resonance spectroscopy.
- Trial name
- MELAS (NCT00887562)
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Michio Hirano
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074984
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 82 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Fabry Disease
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to <18 years of age with Fabry disease and amenable gene…
- Trial name
- NCT03500094
- Testing
- Migalastat HCl 150 mg
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 22 people
- Sponsor
- Amicus Therapeutics
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Microscopic Polyangiitis
Rituximab Vasculitis Maintenance Study
Rituximab is now established as an effective drug for anti-neutrophil cytoplasmic antibody (ANCA) vasculitis following major European and US trials reported in 2010. After a time, its effect wears off and the disease can return.
- Conditions
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Microscopic Polyangiitis, Wegener Granulomatosis
- Trial name
- RITAZAREM (NCT01697267)
- Testing
- Rituximab, Azathioprine
- Where
- 38 sites in United States, Australia and 8 more
- Volunteers
- 188 people
- Sponsor
- Cambridge University Hospitals NHS Foundation Trust
Alzheimer's Disease With Vascular Pathology
A Study of the Effects of CY6463 in Participants With Alzheimer's Disease With Vascular Pathology
This study is being conducted to test the safety, tolerability, and pharmacokinetics of the investigational drug CY6463 compared with placebo in individuals who are aged 60 years or older and have Alzheimer's disease (AD) along…
- Trial name
- NCT04798989
- Testing
- CY6463
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- Tisento Therapeutics
Ischemic Stroke, Cerebral Small Vessel Disease
Remote Preconditioning Over Time To Empower Cerebral Tissue
Previous studies in animals and humans has shown that brief periods of reduced blood flow to one organ or tissue in the body can help protect other tissues from subsequent injury caused by reduced blood flow such as a stroke.
- Conditions
- Ischemic Stroke, Cerebral Small Vessel Disease, Cognitive Decline
- Trial name
- REM-PROTECT (NCT02169739)
- Testing
- Ischemic Preconditioning
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, Los Angeles
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Mild Cognitive Impairment
Rehabilitation of Attention in Patients With MCI and Brain Subcortical Vascular Changes Using the APT-II
Background: Subcortical Vascular Dementia (VaD), consequent to deep brain small vessel disease (SVD), is the most frequent form of VaD.
- Trial name
- RehAtt (NCT02033850)
- Testing
- Attention Process Training-II
- Where
- Italy
- Volunteers
- 46 people
- Sponsor
- Azienda Ospedaliero-Universitaria Careggi
Fabry Disease
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and…
- Trial name
- SMILE (NCT05843916)
- Testing
- Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta)
- Where
- 5 sites in Argentina
- Volunteers
- 20 people
- Sponsor
- Bio Sidus SA
MELAS
Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
- Trial name
- NCT04475549
- Testing
- IW-6463 Tablets
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tisento Therapeutics
Fabry Disease
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without…
- Trial name
- FIELD (NCT00701415)
- Testing
- Agalsidase beta
- Where
- 12 sites in United States, Argentina and 7 more
- Volunteers
- 31 people
- Sponsor
- Genzyme, a Sanofi Company