Clinical trials: Vascular Anomaly
Heart Failure, Coronary Artery Disease
COVID-19 Testing in Underserved and Vulnerable Populations
As part of National Institutes of Health Rapid Acceleration of Diagnostics-Underserved Populations (RADx-UP) program, the goal of the RADxUP study is to develop, test, and evaluate a rapid, scalable capacity building project to…
- Conditions
- Heart Failure, Coronary Artery Disease, Cancer, Chronic Kidney Diseases
- Trial name
- NCT05315908
- Testing
- Community outreach method
- Where
- United States
- Volunteers
- 9,120 people
- Sponsor
- Jesse Nodora
Sickle Cell Anemia, Sickle Cell-Beta0-Thalassemia
The Effect of Rivaroxaban in Sickle Cell Disease
The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD)…
- Trial name
- NCT02072668
- Testing
- rivaroxaban
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of North Carolina, Chapel Hill
Hereditary Angioedema, Hereditary Angioedema Type I
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II
This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE).
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- HAE CHAPTER-1 (NCT05047185)
- Testing
- Deucrictibant low dose, Deucrictibant high dose
- Where
- 19 sites in United States, Austria and 7 more
- Volunteers
- 34 people
- Sponsor
- Pharvaris Netherlands B.V.
Hemophilia A, Gene Therapy
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
This study is being conducted by BioMarin Pharmaceutical Inc. as an open label, single dose study to determine the safety of valoctocogene roxaparvovec (an Adenovirus-Associated Virus (AAV) based gene therapy vector) in severe…
- Conditions
- Hemophilia A, Gene Therapy, Clotting Disorders, Blood Disorder
- Trial name
- GENEr8-AAV5+ (NCT03520712)
- Testing
- Valoctocogene Roxaparvovec
- Where
- 9 sites in South Africa, South Korea and 2 more
- Volunteers
- 3 people
- Sponsor
- BioMarin Pharmaceutical
Cerebral Palsy, Perinatal Stroke
Single-session tDCS in Cerebral Palsy
The goal of this study is to characterize individual responses to a single application of transcranial direct current stimulation (tDCS) in children with unilateral cerebral palsy (UCP), and to test which electrode configuration…
- Conditions
- Cerebral Palsy, Perinatal Stroke, Periventricular Leukomalacia
- Trial name
- NCT03635775
- Testing
- Active tDCS
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
Angiofibromas, Tuberous Sclerosis
Topical Rapamycin to Erase Angiofibromas in TSC
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous…
- Trial name
- Treatment (NCT01526356)
- Testing
- Rapamycin
- Where
- 10 sites in United States, Australia
- Volunteers
- 179 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hemophilia A
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement…
- Trial name
- GENEr8-2 (NCT03392974)
- Testing
- Valoctocogene Roxaparvovec
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- BioMarin Pharmaceutical
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Foramen Ovale, Patent, Echocardiography
Long-term Outcomes After Percutaneous Closure of PFO
International medical centre Medicor from Slovenia has reported 363 successful percutaneous closures of patent foramen ovale (PFO) from October 2006 till October 2023.
- Conditions
- Foramen Ovale, Patent, Echocardiography, Embolic Stroke, Atrial Fibrillation
- Trial name
- NCT05558774
- Where
- Slovenia
- Volunteers
- 364 people
- Sponsor
- MC Medicor
Dyslipidemias, High Cholesterol
Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies
This study will be a placebo-controlled, double-blind, randomized, phase 3 study in participants with underlying heterozygous familial hypercholesterolemia (HeFH) and/or ASCVD to evaluate the efficacy, safety, and tolerability of…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROADWAY (NCT05142722)
- Testing
- Obicetrapib
- Where
- 181 sites in United States, China and 6 more
- Volunteers
- 2,530 people
- Sponsor
- NewAmsterdam Pharma
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
CCM, Cavernoma
CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness
Brain Cavernous Angiomas with Symptomatic Hemorrhage (CASH) are rare, but they exact a heavy burden of neurologic disability from recurrent bleeding, for which there is no proven therapy.
- Conditions
- CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1
- Trial name
- NCT03652181
- Where
- United States
- Volunteers
- 123 people
- Sponsor
- University of Chicago
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme.
- Trial name
- NCT00081497
- Testing
- agalsidase beta
- Where
- 25 sites in United States, Canada and 4 more
- Volunteers
- 67 people
- Sponsor
- Genzyme, a Sanofi Company
Von Hippel-Lindau Syndrome
TKI 258 in Von Hippel-Lindau Syndrome (VHL)
The goal of this clinical research study is to learn if dovitinib can safely be given to patients who have VHL with a measurable hemangioblastoma (tumor of the central nervous system).
- Trial name
- NCT01266070
- Testing
- Dovitinib
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- M.D. Anderson Cancer Center
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
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Plerixafor and Filgrastim For Mobilization of Donor Peripheral Blood Stem Cells Before A Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
RATIONALE: Giving chemotherapy and total-body irradiation (TBI) before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01076270
- Testing
- plerixafor, filgrastim, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Fred Hutchinson Cancer Center
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain
RATIONALE: An Opioid Titration Order Sheet that allows healthcare providers to adjust the dose and schedule of pain medication may help improve pain treatment for patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00666211
- Testing
- educational intervention, Titrated pain management, questionnaire administration
- Where
- 11 sites in United States
- Volunteers
- 98 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Hematologic Disease, Hemophilia A
BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)
The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule…
- Trial name
- NCT00586521
- Testing
- Kogenate (BAY14-2222)
- Where
- 11 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- Bayer
Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01302860
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals