Clinical trials: Vascular Malformation
Pseudoxanthoma Elasticum
A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
This study was designed to evaluate the safety, tolerability, pharmacodynamics (PD) of DS-1211b, and pharmacokinetics (PK) in individuals with Pseudoxanthoma elasticum (PXE).
- Trial name
- NCT05569252
- Testing
- DS-1211b
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 65 people
- Sponsor
- Daiichi Sankyo
Fabry Disease
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Primary Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.
- Trial name
- NCT02228460
- Testing
- GZ/SAR402671
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
Pulmonary Hypertension, Bronchopulmonary Dysplasia
Inhaled Nitric Oxide for Pulmonary Hypertension and Bronchopulmonary Dysplasia
Inhaled nitric oxide (iNO) is an effective treatment for pulmonary hypertension (PH) in term and near-term infants. Preterm infants are at risk for early PH that is associated with high risk for bronchopulmonary dysplasia or…
- Trial name
- NCT03576885
- Testing
- inhaled nitric oxide
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- AdventHealth
Cardiovascular Diseases, Cardiovascular Abnormalities
The Effects of the Dietary Supplement CardioFlex Q10 on Reducing Cardiovascular Disease Risk Factors in Adults
Cardiovascular diseases (CVD), primarily heart disease and stroke, are the leading causes of death and prescription drug use in Canada.
- Conditions
- Cardiovascular Diseases, Cardiovascular Abnormalities, Heart Attack, Stroke
- Trial name
- NCT03826914
- Testing
- CardioFlex Q10
- Where
- Canada
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- University of Manitoba
Hereditary Angioedema
EASSI - Evaluation of the Safety of Self-Administration With Icatibant
This study is being conducted to explore the clinical safety, local tolerability, convenience and effectiveness of self-treatment of hereditary angioedema (HAE) attacks with subcutaneous injections of icatibant.
- Trial name
- EASSI (NCT00997204)
- Testing
- Icatibant
- Where
- 26 sites in Argentina, Austria and 8 more
- Volunteers
- 151 people
- Sponsor
- Shire
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire
Port Wine Stains
Combined Photodynamic and Pulsed Dye Laser Treatment of Port Wine Stains
This research study was designed to determine the effect on port wine stains (PWS) of liposomal benzoporphyrin derivative monoacid (BPD-MA) termed verteporfin by intravenous (IV) infusion for photodynamic therapy (PDT) or…
- Trial name
- PDT/PDL (NCT00556946)
- Testing
- Combined Photodynamic & Pulsed Dye Laser Treatment
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of California, Irvine
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
Behcet Syndrome
A Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in the Treatment of Behçet Disease
The purpose of this study is to assess whether Apremilast is safe and effective in the treatment of patients with Behcet Disease.
- Trial name
- NCT00866359
- Testing
- Apremilast (CC-10004)
- Where
- 7 sites in United States, Turkey (Türkiye)
- Volunteers
- 111 people
- Sponsor
- Amgen
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup.
- Conditions
- Neuroblastoma, Medulloblastoma, Glioma, Ependymoma
- Trial name
- NCT02162732
- Testing
- Guided Therapy
- Where
- 19 sites in United States, Lebanon
- Volunteers
- 186 people
- Sponsor
- Giselle Sholler
Prematurity, Bronchopulmonary Dysplasia
Inhaled Nitric Oxide and Neuroprotection in Premature Infants
The purpose of this study is to determine whether inhaled nitric oxide improves the neurological outcome for premature infants.
- Conditions
- Prematurity, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage, Periventricular Leukomalacia
- Trial name
- NOVA2 (NCT00515281)
- Testing
- inhaled nitric oxide, oxygen
- Where
- United States
- Volunteers
- 273 people
- Sponsor
- University of Chicago
Hereditary Angioedema
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities…
- Trial name
- NCT00292981
- Testing
- C1 Esterase Inhibitor
- Where
- 14 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Hereditary Angioedema
Subcutaneous Treatment With Icatibant for Acute Attacks of Hereditary Angioedema (HAE)
Primary Outcome Measures: The primary endpoint was the time to onset of symptom relief of the first attack in the double blind phase.
- Trial name
- FAST2 (NCT00500656)
- Testing
- Icatibant, Tranexamic Acid
- Where
- Italy
- Volunteers
- 85 people
- Sponsor
- Shire
Pseudoxanthoma Elasticum
Magnesium Supplements In The Treatment Of Pseudoxanthoma Elasticum (PXE)
The purpose of this study is to evaluate the effectiveness of magnesium oxide supplements on the reversal of calcium deposits in the skin, and the yellow bumps and folds of skin in subjects with pseudoxanthoma elasticum (PXE).
- Trial name
- NCT01525875
- Testing
- Magnesium Oxide
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Mark Lebwohl
End Stage Renal Failure, Hemodialysis Access Failure
Outcomes for One-stage and Two-stage Brachial Basilic Arteriovenous Fistulas
An arteriovenous (AV) fistula is essential for patients with kidney failure on dialysis. There is no clear data on the best method to create a new brachial basilic AV fistula (BBAVF), so the choice between a single procedure and…
- Conditions
- End Stage Renal Failure, Hemodialysis Access Failure, Arterio-venous Fistula
- Trial name
- NCT04376567
- Testing
- One stage approach
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Tze-Woei Tan
Sickle Cell Disease
Cardiovascular Complications of Sickle Cell Disease
In this research study, we are using heart imaging exams and blood testing, in order to gain an improved understanding of the pulmonary (lung) hypertension and cardiovascular (heart) complications that often occur in sickle cell…
- Trial name
- NCT01044901
- Testing
- MRI, Transthoracic Echocardiography, tonometry, EKG
- Where
- United States
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- University of Chicago
Breast Cancer, Chronic Myeloproliferative Disorders
Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, granisetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients treated with chemotherapy.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00293384
- Testing
- Aprepitant, Cyclophosphamide, Dexamethasone
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Chronic Myeloproliferative Disorders, Leukemia
Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor bone marrow transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00134004
- Testing
- cyclophosphamide, fludarabine phosphate, mycophenolate mofetil
- Where
- 3 sites in United States
- Volunteers
- 210 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Behcet Disease
1 Year of Treatment With Canakinumab in Behçet's Disease Patients With Neurologic or Vascular Involvement
Primary objective of the study was to evaluate the safety and efficacy of canakinumab on the clinical and inflammatory findings of Behced Disease patients with neurologic and vascular involvement.
- Trial name
- Behcet (NCT02756650)
- Testing
- Canakinumab
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Angioedema (HAE)
Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema
The objective of this study is to evaluate the formation of antibodies, the occurence of allergic reactions, and the risk of hypercoagulability and hypocoagulability in patients treated with KALBITOR (ecallantide).
- Trial name
- NCT01059526
- Testing
- ecallantide
- Where
- 40 sites in United States
- Volunteers
- 81 people
- Sponsor
- Shire