Clinical trials: Vascular Anomalies
NOMID, CINCA Syndrome
Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease
This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous…
- Trial name
- NCT00770601
- Testing
- Canakinumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Angioedema (HAE)
Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks
The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema…
- Trial name
- NCT01756157
- Testing
- CINRYZE with rHuPH20
- Where
- 23 sites in United States, Germany and 2 more
- Volunteers
- 47 people
- Sponsor
- Shire
Vascular Anomalies
A Study of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies.
- Trial name
- NCT07767877
- Volunteers
- 200 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Angioedema
First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX7353 in healthy subjects, and single and…
- Trial name
- NCT02448264
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Behçet's Disease, Juvenile Psoriatic Arthritis
A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis
The primary objective of this study is to evaluate the long-term safety of apremilast in subjects 2 years of age or older with oral ulcers associated with Behçets disease or 5 years of age or older with active juvenile psoriatic…
- Trial name
- NCT05767047
- Testing
- Apremilast
- Where
- 7 sites in Greece, Israel and 2 more
- Volunteers
- 48 people
- Sponsor
- Amgen
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia and Statin Intolerant
The purpose of this study is to determine if bempedoic acid (ETC-1002) is effective and safe versus placebo in patients with elevated LDL cholesterol and who are statin-intolerant.
- Conditions
- Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Statin Adverse Reaction
- Trial name
- CLEAR Serenity (NCT02988115)
- Testing
- bempedoic acid
- Where
- 67 sites in United States, Canada
- Volunteers
- 345 people
- Sponsor
- Esperion Therapeutics, Inc.
Fabry Disease
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have…
- Trial name
- NCT03614234
- Testing
- pegunigalsidase alfa
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 29 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Fabry Disease
A Study of Patients With Fabry Disease (US Specific)
This is an observational study to evaluate the effects of treatment on long-term effectiveness, safety, and health-related quality of life (HRQOL) in patients with Fabry disease, with a main focus on migalastat.
- Trial name
- NCT06906367
- Testing
- migalastat HCl, ERT
- Where
- 8 sites in United States
- Volunteers
- 450 people
- Sponsor
- Amicus Therapeutics
Hereditary Angioedema
A Relative Bioavailability Study of Two Formulations of BCX7353
This is an open-label, randomized study to investigate the relative bioavailability of two formulations of BCX7353 and to determine if there is a food effect
- Trial name
- NCT03202784
- Testing
- BCX7353
- Where
- United Kingdom
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Fabry Disease
A Study to Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease
The primary purpose of this study was to assess the safety and tolerability of lucerastat in adults with Fabry Disease receiving Enzyme Replacement Therapy (ERT).
- Trial name
- NCT02930655
- Testing
- Lucerastat, Enzyme replacement therapy (ERT)
- Where
- Germany
- Volunteers
- 14 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Fabry Disease
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201…
- Trial name
- NCT04999059
- Testing
- Safety and Efficacy Assessments
- Where
- 3 sites in Australia, Brazil
- Volunteers
- 5 people
- Sponsor
- AVROBIO
Fabry Disease
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on…
- Trial name
- FAVORIT (NCT06880250)
- Where
- 23 sites in Russia
- Volunteers
- 100 people
- Sponsor
- NPO Petrovax
Hypoxic-Ischemic Encephalopathy, Neonatal Asphyxia
Assess Safety and Efficacy of Sovateltide in Hypoxic-ischemic Encephalopathy
Sovateltide (PMZ-1620; IRL-1620) is targeted to be used as a "Treatment for hypoxic-ischemic encephalopathy in neonates," which is a life-threatening condition.
- Conditions
- Hypoxic-Ischemic Encephalopathy, Neonatal Asphyxia, Neonatal Encephalopathy
- Trial name
- NCT05514340
- Testing
- Normal Saline along with standard treatment, Sovateltide along with standard treatment
- Where
- 4 sites in India
- Volunteers
- 40 people
- Sponsor
- Pharmazz, Inc.
Chronic Kidney Disease
Novel Endovascular Access Trial (NEAT)
The objective of this study is to evaluate the safety and efficacy of the FLEX System when used to create an arteriovenous fistula (AVF or AV fistula) percutaneously in patients with Chronic Kidney Disease (CKD) who require…
- Trial name
- NEAT (NCT02036671)
- Testing
- The FLEX System will be used to percutaneously create a fistula in CKD patients who require hemodialysis vascular access
- Where
- 9 sites in Australia, Canada and 1 more
- Volunteers
- 80 people
- Sponsor
- C. R. Bard
Hereditary Angioedema
Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients
The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).
- Trial name
- NCT05691361
- Testing
- ADX-324
- Where
- Australia
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- ADARx Pharmaceuticals, Inc.
Ischemic Stroke
Study to Test Sensitivity and Specificity of Passive Wave Pressure Device in Determining Ischemic Stroke
That the Jan Medical Nautilus NeuroWaveTM system provides significantly higher sensitivity to hyper acute ischemic stroke than does CT.
- Trial name
- NCT01643525
- Testing
- Nautilus NeuroWaveTM System
- Where
- 3 sites in United States
- Volunteers
- 29 people, healthy volunteers welcome
- Sponsor
- Jan Medical, Inc.
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
NALP3 Mutation
Safety, Efficacy, Pharmacokinetics, Pharmacodynamics of ACZ885 in Patients With NALP3 Mutations and Clinical Symptoms
This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are…
- Trial name
- NCT00487708
- Testing
- canakinumab
- Where
- 10 sites in France, Germany and 3 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Cardiovascular Diseases, Cardiovascular Diseases Risk
Cor360: Clinician-Facing Broad-Spectrum Cardiac Phenotyping and Risk Assessment Using COR® Wearable ECG to Inform Outcomes-Oriented Care Pathways
Cor360 is an observational study evaluating whether extended wearable electrocardiogram (ECG) data can support broad-spectrum cardiac phenotyping and risk assessment beyond conventional single-purpose ambulatory rhythm…
- Conditions
- Cardiovascular Diseases, Cardiovascular Diseases Risk, Cardiac Conduction System Disease, Bundle/Fascicular Abnormalities
- Trial name
- Cor360 (NCT07775976)
- Testing
- COR Wearable ECG Monitoring, Cor360 Clinician-Facing Decision-Aid Reporting
- Where
- 2 sites in United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Peerbridge Health, Inc
Hereditary Angioedema (HAE)
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels.
- Trial name
- EXPLORHAE (NCT07218393)
- Testing
- No intervention
- Where
- 7 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Takeda