Clinical trials: Vascular Anomalies
Embolic Stroke of Undetermined Source, Transient Ischemic Attack
RoBotic TCD Ultrasound BubbLe Study Compared to Transthoracic Echocardiography for Detection of Right to Left Shunt
This study is a multi-center, prospective, single-arm, non-significant risk (NSR) device study in which up to 150 evaluable subjects with suspicion of embolic stroke of undetermined source (ESUS) will be evaluated with NB-IS TCD…
- Conditions
- Embolic Stroke of Undetermined Source, Transient Ischemic Attack, Right-To-Left Atrial Shunt, Patent Foramen Ovale
- Trial name
- BUBL (NCT04604015)
- Testing
- NeuralBot Investigational System, Transthoracic Echocardiography (TTE)
- Where
- 7 sites in United States
- Volunteers
- 154 people
- Sponsor
- NeuraSignal Inc dba NovaSignal
Hereditary Angioedema (HAE)
A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home
The main aim of this study is to describe the treatment patterns, characteristics and outcomes of people with HAE who are currently receiving icatibant in the homecare setting in the United Kingdom (UK).
- Trial name
- NCT05489640
- Testing
- No Intervention
- Where
- United Kingdom
- Volunteers
- 85 people
- Sponsor
- Takeda
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
Atherosclerosis, Hypercholesterolemia
Effect of Ezetimibe Plus Simvastatin Versus Simvastatin Alone on Atherosclerosis in the Carotid Artery (ENHANCE)(P02578)
The purpose of the study is to determine whether ezetimibe plus simvastatin will be more effective than simvastatin alone in preventing progression of atherosclerosis of the inner layer of the carotid artery.
- Conditions
- Atherosclerosis, Hypercholesterolemia, Hyperlipoproteinemia Type II
- Trial name
- ENHANCE (NCT00552097)
- Testing
- ezetimibe (plus simvastatin)
- Volunteers
- 720 people
- Sponsor
- Organon and Co
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Evaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular Risk
The purpose of this study is to see if bemedoic acid (ETC-1002) is effective versus placebo in patients with high cardiovascular risk and elevated LDL cholesterol not adequately controlled by their current therapy.
- Trial name
- CLEAR Wisdom (NCT02991118)
- Testing
- bempedoic acid
- Where
- 2 sites in United States
- Volunteers
- 779 people
- Sponsor
- Esperion Therapeutics, Inc.
Hereditary Angioedema
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Type I or II Hereditary Angioedema (HAE) adolescent and adult post-trial and…
- Trial name
- NCT06628713
- Testing
- Sebetralstat
- Where
- United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema (HAE)
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single subcutaneous (SC) administration of icatibant in…
- Trial name
- NCT01386658
- Testing
- icatibant
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Shire
Fabry Disease
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
This program allows physicians to request permission from Amicus Therapeutics (Amicus) for treatment access to migalastat hydrochloride (HCl) for specific patients with Fabry disease. Treatment is open label.
- Trial name
- NCT01476163
- Testing
- migalastat HCl 150 mg, migalastat HCl 20 mg
- Sponsor
- Amicus Therapeutics
Fabry's Disease
A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme
This Phase 4 study will evaluate the safety and tolerability of Fabrazyme at current approved dose with increases in the infusion rate and reduced infusion volume.
- Trial name
- SHORTEN (NCT06019728)
- Testing
- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine
- Where
- 5 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sanofi
Hereditary Angioedema, HAE
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in…
- Trial name
- APeX-J (NCT03873116)
- Testing
- BCX7353 capsules
- Where
- 10 sites in Japan
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Hemorrhagic Telangiectasia
A Trial to Assess the Efficacy and Safety of Engasertib in Participants With Moderate to Severe Hereditary Hemorrhagic Telangiectasia (HHT)
The primary objective of this trial is to assess the efficacy of engasertib 40 mg once daily (QD) in reducing the frequency of epistaxis compared to placebo QD during 28 weeks of double-blind treatment in participants with…
- Trial name
- NCT07743671
- Testing
- Engasertib
- Where
- 4 sites in United States
- Volunteers
- 240 people
- Sponsor
- Vaderis Therapeutics AG
Fabry Disease
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male…
- Trial name
- NCT03737214
- Testing
- Lucerastat
- Where
- 42 sites in United States, Australia and 11 more
- Volunteers
- 107 people
- Sponsor
- Idorsia Pharmaceuticals Ltd.
Vascular Ehlers-Danlos Syndrome
Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome
This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.
- Trial name
- DiSCOVER (NCT05432466)
- Testing
- ACER-002 (celiprolol) 200 mg BID
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Acer Therapeutics Inc.
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05139810
- Testing
- Donidalorsen
- Where
- 51 sites in United States, Belgium and 12 more
- Volunteers
- 91 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Fabry Disease
Sophisticated Assessment of Disease Burden in Patients With Fabry Disease
To detect early signs of cardiac and metabolic alterations as well as to evaluate the progression of cardiac and metabolic impairments in mildly affected patients with Fabry Disease using high sensitive diagnostic methods.
- Trial name
- SOPHIA (NCT01210196)
- Testing
- MRI
- Where
- 11 sites in Belgium, Czechia and 4 more
- Volunteers
- 36 people
- Sponsor
- Shire
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring
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 provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study
- Conditions
- Cryopyrin-associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT01576367
- Testing
- ACZ885
- Where
- 12 sites in Belgium, Canada and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Angioedema
A Single Dose Safety, Tolerability, Pharmacokinetic and Food Effect Study of KVD900 (Sebetralstat) in Healthy Volunteers
A safety, tolerability, pharmacokinetic and food effect study of KVD900 in healthy volunteers.
- Trial name
- NCT04349800
- Testing
- KVD900
- Where
- United Kingdom
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Venous Stenosis, Venous Occlusion
WRAP North America
The goal of this observational study is to investigate the safety and efficacy of the WRAPSODY CIE in a real-world North American population.
- Trial name
- NCT06807099
- Testing
- Merit WRAPSODY® Cell Impermeable Endoprosthesis (WRAPSODY CIE)
- Where
- 16 sites in United States, Canada
- Volunteers
- 250 people
- Sponsor
- Merit Medical Systems, Inc.
Fabry Disease
A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects
GR181413A/AT1001 (migalastat hydrochloride) is a low molecular weight iminosugar, an analog of the terminal galactose group that is cleaved from the substrate GL-3.
- Trial name
- NCT01853852
- Testing
- GR181413A/AT1001 solution, GR181413A/AT1001 capsule, Potable water
- Where
- Australia
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics