Clinical trials: Vascular Malformation
Fabry Disease
Neuropathic Pain and Fabry Disease
Fabry disease is a rare X-linked lysosomal storage disorder. The mutations result in a deficiency of the lysosomal enzyme α-galactosidase causing accumulation of glycosphingolipids in the vascular endothelial cells and many other…
- Trial name
- NCT00168974
- Where
- Denmark
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Danish Pain Research Center
Down Syndrome, Cardiovascular Risk Factor
Vascular Health and Risk Factors in Children With Down Syndrome
This is a prospective, multicenter, cross-sectional study to evaluate prevalence of vascular risk factors in children with Down Syndrome and to determine the association between vascular disease risk factors and objective markers…
- Conditions
- Down Syndrome, Cardiovascular Risk Factor, Atherosclerosis
- Trial name
- NCT04735874
- Testing
- Evaluate vascular disease risk factors in children with Down Syndrome, Determine the associations between cardiovascular disease risk factors and markers of early atherosclerosis in children with Down Syndrome
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- University of Utah
Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia
Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease
LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy.
- Conditions
- Atherosclerotic Cardiovascular Disease, Heterozygous Familial Hypercholesterolemia, Hypercholesterolemia
- Trial name
- NCT06112327
- Where
- 3 sites in New Zealand, United Kingdom
- Volunteers
- 116 people
- Sponsor
- Verve Therapeutics, Inc.
Chronic Kidney Disease
A Study of Vonapanitase (PRT-201) Administered Immediately After Radiocephalic Arteriovenous Fistula(AVF) Creation in Patients With Chronic Kidney Disease (CKD) (PATENCY-2)
This research study is designed to assess the safety and effectiveness of an experimental drug called vonapanitase (PRT-201) in patients both receiving or expecting to receive hemodialysis who have chronic kidney disease and who…
- Trial name
- NCT02414841
- Testing
- Vonapanitase
- Where
- 41 sites in United States, Canada
- Volunteers
- 696 people
- Sponsor
- Proteon Therapeutics
Hypertension, Congestive Cardiomyopathy
The Acute Effects of the Angiotensin-converting Enzyme Inhibitor Enalaprilat on Flow Distribution
The primary objective of this study is to study the acute effects of angiotensin-converting enzyme inhibitor (ACEI) on systemic, pulmonary and cerebral blood flow in post bidirectional cavopulmonary connection (BCPC) patients.
- Trial name
- NCT00741156
- Testing
- Enalaprilat
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- The Hospital for Sick Children
Attention Deficit Disorder With Hyperactivity, Asthma
Study of an Intervention to Improve Problem List Accuracy and Use
The aim of this study is to identify patients with problem list gaps and intervene to correct these gaps by creating clinical decision support interventions that alert providers to likely problem list gaps and offer clinicians…
- Conditions
- Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer
- Trial name
- MAPLE (NCT01105923)
- Testing
- MAPLE
- Where
- United States
- Volunteers
- 140 people
- Sponsor
- Brigham and Women's Hospital
Pseudoxanthoma Elasticum
The Prevention of Systemic Ectopic Mineralization in Pseudoxanthoma Elasticum
The goal of this randomized clinical trial is to assess the effect of etidronate on ectopic calcification in relatively young patients with Pseudoxanthoma elasticum.
- Trial name
- TEMP-PREVENT (NCT05832580)
- Testing
- Etidronate
- Where
- Netherlands
- Volunteers
- 76 people
- Sponsor
- UMC Utrecht
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Background: Pyrimidine and purine metabolism disorders (DPPMs) affect how the body metabolizes chemicals called pyrimidines and purines.
- Conditions
- AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of
- Trial name
- NCT06092346
- Where
- United States
- Volunteers
- 999 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Pulmonary Hypertension
Anticoagulation For Pulmonary Hypertension in Sickle Cell Disease
Sickle cell disease (SCD) is often referred to as a hypercoagulable state. However, the contribution of coagulation activation to the pathogenesis of SCD remains uncertain.
- Trial name
- NCT01036802
- Testing
- Warfarin
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Angioedema (HAE)
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE).
- Trial name
- APeX-1 (NCT02870972)
- Testing
- BCX7353
- Where
- 24 sites in Australia, Austria and 9 more
- Volunteers
- 75 people
- Sponsor
- BioCryst Pharmaceuticals
Neurologic Disorder, Stroke
Home Based Tele-exercise for People With Chronic Neurological Impairments
To assess the impact of a 12-week virtual seated physical intervention on cardiovascular health and wellness in people with chronic neurological impairments (CNI).
- Conditions
- Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy
- Trial name
- Telex (NCT04564495)
- Testing
- Seated exercise
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Burke Medical Research Institute
Fabry Disease
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
This is a Phase II, open-label study designed to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of AL01211 in male subjects with classic Fabry disease who have never received any treatment (eg.
- Trial name
- NCT06114329
- Testing
- AL01211
- Where
- 6 sites in China
- Volunteers
- 16 people
- Sponsor
- AceLink Therapeutics, Inc.
Congenital Disorders
Effects of Beta-adrenergic in Adults w/Transposition of Great Arteries on Systemic Ventricular Function
The purpose of this chart review study is to examine the effects of beta -adrenergic blocking agents on systemic ventricular dimensions, systemic atrioventricular valve function and exercise tolerance in patients with…
- Trial name
- NCT00313352
- Where
- United States
- Volunteers
- 110 people
- Sponsor
- Emory University
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant
RATIONALE: Rasburicase may be an effective treatment for graft-versus-host disease caused by a donor stem cell transplant. PURPOSE: This clinical trial is studying how well rasburicase works in preventing graft-versus-host…
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00513474
- Testing
- busulfan, cyclophosphamide, cyclosporin-A
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Massachusetts General Hospital
Arteriovenous Malformation, Schwannoma
Hippocampal Radiation Exposure and Memory
Neurocognitive impairment as a result of gamma knife radiosurgery has not been well studied and is poorly understood. Radiosurgery to the base of skull for the treatment of benign and malignant disorders may consequently impair…
- Conditions
- Arteriovenous Malformation, Schwannoma, Trigeminal Neuralgia
- Trial name
- NCT00603694
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- University Health Network, Toronto
Coronary Disease
Platelet Activation Markers in Pediatric Cardiac Surgery
In this study the focus will be on correlating the levels of platelet activation markers (proteins that are released when blood cells are activated)to the duration of cardiopulmonary bypass, temperature during cardiopulmonary…
- Trial name
- NCT00450151
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Weill Medical College of Cornell University
Hereditary Haemorrhagic Telangiectasia, PFO - Patent Foramen Ovale
Non-Invasive Detection of Pulmonary Right-to-Left Shunts Using the SONAS Ultrasound Device
The goal of this clinical trial is to learn how well the SONAS ultrasound device can detect right-to-left shunts of adults with hereditary hemorrhagic telangiectasia (HHT) who are already scheduled for a contrast bubble…
- Conditions
- Hereditary Haemorrhagic Telangiectasia, PFO - Patent Foramen Ovale, Pulmonary Arteriovenous Malformation
- Trial name
- SONAS (NCT07414446)
- Testing
- Transcranial Ultrasound Device
- Where
- Netherlands
- Volunteers
- 40 people
- Sponsor
- St. Antonius Hospital
Hemodialysis Access
Safety of Vascugel Treatment After Creation of Arteriovenous Access for Hemodialysis Use
Vascugel™ is safe when placed at the anastomotic site at the time of surgery during creation of an AV graft or fistula for hemodialysis access.
- Trial name
- NCT00479180
- Testing
- Vascugel
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Pervasis Therapeutics, Inc
Hereditary Angioedema (HAE)
Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema
Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of…
- Trial name
- NCT02052141
- Testing
- CINRYZE 500, CINRYZE 1000
- Where
- 8 sites in United States, Germany and 3 more
- Volunteers
- 12 people
- Sponsor
- Shire
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