Clinical trials: Vascular Anomalies
Hemophilia A
Study Evaluating The Safety Of Xyntha In Usual Care Settings
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year…
- Trial name
- NCT00765726
- Testing
- Moroctocog alfa(AF-CC), Blood draw for laboratory testing
- Where
- 6 sites in United States, New Zealand
- Volunteers
- 12 people
- Sponsor
- Pfizer
Aneurysm, AVM, Dural Arteriovenous Fistula, Glioma, Meningioma, Metastasis, Bypass
Intraoperative Laser Speckle Contrast Imaging of Cerebral Blood Flow
The purpose of the proposed study is to investigate the usefulness of laser speckle contrast imaging (LSCI) compared to indocyanine green angiography (ICGA).
- Trial name
- NCT05305378
- Testing
- Laser speckle contrast imaging (LSCI)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Dynamic Light
Cryopyrin-Associated Periodic Syndromes (CAPS)
HL2351 CAPS Phase II Study
This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).
- Trial name
- NCT02853084
- Testing
- HL2351
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Handok Inc.
Fabry Disease, Fabry Disease, Cardiac Variant
Long-Term Follow-up of Subjects Who Were Treated With ST-920
Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol.
- Trial name
- NCT05039866
- Testing
- ST-920
- Where
- 13 sites in United States, Australia and 3 more
- Volunteers
- 32 people
- Sponsor
- Sangamo Therapeutics
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
Fabry Disease
Open-Label Phase 3 Long-Term Safety Study of Migalastat
This was a long-term, open-label study of migalastat (123 milligrams [mg] of migalastat [equivalent to 150 mg of migalastat hydrochloride]) (migalastat) in participants with Fabry disease who completed treatment in a previous…
- Trial name
- AT1001-041 (NCT01458119)
- Testing
- migalastat hydrochloride
- Where
- 25 sites in United States, Argentina and 11 more
- Volunteers
- 85 people
- 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
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00304512
- Testing
- migalastat HCl
- Where
- 6 sites in United States, Australia and 4 more
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Procedural Sedation
A Safety Study of AQUAVAN® (Fospropofol Disodium) Injection for Sedation During Minor Surgical Procedures.
Very often patients receive medications before a diagnostic, therapeutic, or surgical procedure to help them relax, keep them calm, and to relieve them from pain. This is called procedural sedation.
- Trial name
- NCT00327392
- Testing
- AQUAVAN® (fospropofol disodium) Injection
- Where
- 15 sites in United States
- Volunteers
- 123 people
- Sponsor
- Eisai Inc.
Hereditary Angioedema, HAE
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
This is a phase 3, multicenter, 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.
- Trial name
- APeX-2 (NCT03485911)
- Testing
- BCX7353 capsules
- Where
- 47 sites in United States, Austria and 9 more
- Volunteers
- 121 people
- Sponsor
- BioCryst Pharmaceuticals
Chronic Kidney Diseases, Diabetes
KidneYou - Innovative Digital Therapy
The aim of the study is to assess the improvement of health in patients affected by CKD when they are exposed to non-pharmacological treatment strategies as nutritional program (NP), physical activity program (PA) and mindfulness…
- Conditions
- Chronic Kidney Diseases, Diabetes, Arterial Hypertension, Chronic Glomerulonephritis
- Trial name
- NCT05286632
- Testing
- KidneYou APP
- Where
- Italy
- Volunteers
- 210 people
- Sponsor
- Advice Pharma Group srl
Chronic Kidney Diseases, End Stage Renal Disease
SAVE-FistulaS: the SelfWrap-Assisted ArterioVEnous Fistulas Study
This is a prospective, randomized, multi-center clinical trial for chronic kidney disease (CKD) patients referred for creation of a new arteriovenous fistula (AVF) in order to assess the safety and effectiveness of SelfWrap, a…
- Conditions
- Chronic Kidney Diseases, End Stage Renal Disease, Arteriovenous Fistula, Hemodialysis Access Failure
- Trial name
- SAVE-FistulaS (NCT06001827)
- Testing
- SelfWrap Bioabsorbable Perivascular Wrap, Untreated AVF Control
- Where
- 28 sites in United States
- Volunteers
- 600 people
- Sponsor
- VenoStent
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
The goal of this clinical trial is to evaluate the long-term safety and tolerability of zagociguat in patients with MELAS who completed study medication treatment in the lead-in study TIS6463-203.
- Trial name
- NCT06961344
- Testing
- zagociguat 15mg
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 37 people
- Sponsor
- Tisento Therapeutics
Heart Failure, Ischemic Heart Failure
Endocardial Delivery for Myocardial Regeneration Using Allogeneic iPSC-derived Cardiomyocyte Spheroids for HF With Systolic Dysfunction (EMERALD Study)
The purpose of this clinical study is to evaluate the safety and efficacy of endocardial delivery of HS-001 CS into severe heart failure patients with reduced ejection fraction for 26 weeks after transplantation.
- Conditions
- Heart Failure, Ischemic Heart Failure, Ischemic Heart Disease, Dilated Cardiomyopathy (DCM)
- Trial name
- EMERALD (NCT07347197)
- Testing
- HS-005
- Where
- 5 sites in Japan
- Volunteers
- 14 people
- Sponsor
- Heartseed Inc.
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America
The main aim of this study is to compare the HAE attack rate before and after lanadelumab treatment was started in persons with Hereditary Angioedeme Type I or II. Data from participants will be collected for at least 24 months.
- Trial name
- EMPOWER (NCT03845400)
- Where
- 29 sites in United States, Canada and 1 more
- Volunteers
- 168 people
- Sponsor
- Shire
Mitochondrial Diseases, Mitochondrial Myopathies
The KHENERGY Study
Mitochondrial Diseases are rare, progressive, multi-system, often-early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS
- Trial name
- KHENERGY (NCT02909400)
- Testing
- KH176
- Where
- Netherlands
- Volunteers
- 20 people
- Sponsor
- Khondrion BV
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Hereditary Angioedema
A Single and Multiple Doses Safety, Tolerability, Pharmacokinetics and Food Effect Study of KVD824 in Healthy Volunteers
This is a 3 part phase 1, randomized, double-blind, placebo-controlled, study of the safety, tolerability, and pharmacokinetics of KVD824 following administration of single and multiple ascending oral doses; followed by a…
- Trial name
- NCT05178355
- Testing
- KVD824
- Where
- United Kingdom
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Fabry Disease
A Study of Replagal in Treatment-naïve Adults With Fabry Disease
In this study, adults with Fabry Disease who have not had any treatment for this condition will be treated with Replagal. The main aim of the study is to check if Replagal improves kidney function and heart structure of…
- Trial name
- NCT04840667
- Testing
- REPLAGAL
- Where
- 28 sites in Canada, Finland and 6 more
- Volunteers
- 17 people
- Sponsor
- Shire