Clinical trials: Vascular Anomalies
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.
Von Hippel Lindau Disease
MSD Belzutifan PAS
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice.
- Trial name
- NCT06554730
- Testing
- Belzutifan
- Sponsor
- Merck Sharp & Dohme LLC
Fabry Disease
A 24-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) in participants with Fabry disease.
- Trial name
- NCT00283933
- Testing
- migalastat HCl
- Where
- 2 sites in France, United Kingdom
- Volunteers
- 5 people
- Sponsor
- Amicus Therapeutics
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.
Cerebral Arteriovenous Malformation
Treatment of Cerebral Arteriovenous Malformations With SQUID Liquid Embolic Agent (CHOICE)
The objective of this study is to further establish that SQUID, an alternative liquid embolic agent with specific properties, is a safe and effective alternative in bAVM endovascular treatment strategy available to date.
- Trial name
- CHOICE (NCT03413852)
- Testing
- SQUID non-adhesive liquid embolic agent
- Where
- 19 sites in Belgium, Denmark and 3 more
- Volunteers
- 111 people
- Sponsor
- Embo-Flüssigkeiten A.G.
Hereditary Angioedema (HAE)
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
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.
- Trial name
- NCT07263685
- Testing
- No intervention
- Where
- Saudi Arabia
- Volunteers
- 50 people
- Sponsor
- Takeda
Hereditary Angioedema
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Trial name
- NCT06262399
- Where
- 7 sites in Australia, France and 4 more
- Volunteers
- 100 people
- Sponsor
- Intellia Therapeutics
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
Hereditary Angioedema
C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to determine the safety and efficacy of C1INH-nf for the treatment of acute HAE attacks.
- Trial name
- NCT00289211
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 37 sites in United States
- Volunteers
- 83 people
- Sponsor
- Shire
Pulmonary Hypertension
Efficacy and Safety of Bosentan in Sickle Cell Disease (SCD) Patients Diagnosed With Pulmonary Hypertension (PH)
The study will assess the effect of bosentan on pulmonary vascular resistance and exercise capacity in Sickle Cell Disease (SCD) patients diagnosed with Pulmonary Hypertension.
- Trial name
- ASSET-2 (NCT00313196)
- Testing
- bosentan
- Volunteers
- 12 people
- Sponsor
- Actelion
Behçet's Syndrome
A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease
The main objective of this study is to evaluate the efficacy and safety of apremilast in the treatment of oral ulcers in adults with active Behçet's disease (BD).
- Trial name
- NCT02307513
- Testing
- Apremilast
- Where
- 63 sites in United States, France and 8 more
- Volunteers
- 207 people
- Sponsor
- Amgen
Fabry Disease
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
BIO-AGA-Fase III-001 is a Phase III, prospective, multicenter, open-label, single-group, baseline-controlled, switch over clinical trial to evaluate the efficacy and safety of AGA BETA BS in patients with FD already treated and…
- Trial name
- SMILE (NCT05843916)
- Testing
- Recombinant human alpha galactosidase A (agalsidase beta), Recombinant human alpha-galactosidase A (agalsidase beta)
- Where
- 5 sites in Argentina
- Volunteers
- 20 people
- Sponsor
- Bio Sidus SA
Hereditary Angioedema (HAE)
A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)
The main aim of this study is to evaluate the safety of lanadelumab in Chinese participants with HAE. Participants will be treated with lanadelumab for 26 weeks.
- Trial name
- NCT05460325
- Testing
- Lanadelumab
- Where
- 4 sites in China
- Volunteers
- 20 people
- Sponsor
- Takeda
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Hairy Cell Leukemia
US Post-Marketing Safety Study of Moxetumomab Pasudotox-tdfk (LUMOXITI)
This study is being conducted to satisfy a post-marketing requirement (PMR) to provide evidence characterizing 1) the safety of moxetumomab pasudotox-tdfk in patients who are 65 years of age and older and/or 2) the safety of…
- Trial name
- PROXY (NCT04125290)
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- AstraZeneca
Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This is a Phase 3, multicenter, 3-part study, with 2 randomized, double-blind, placebo-controlled parts and an open-label extension part, to evaluate the efficacy and safety of orally administered deucrictibant XR tablet for…
- Trial name
- CREAATE (NCT07266805)
- Testing
- Deucrictibant
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 48 people
- Sponsor
- Pharvaris Netherlands B.V.
Meningioma, Tumor, Brain
MiDura-Study (Neuro-Patch in Duraplasty)
The aim of this study is to collect systematically and proactively data regarding the performance of Neuro-Patch, like complications and handling, under daily clinical practice when used as intended by the manufacturer
- Conditions
- Meningioma, Tumor, Brain, Chiari Malformation, Vascular Malformation
- Trial name
- MiDura (NCT04189172)
- Where
- 4 sites in France, Germany
- Volunteers
- 328 people
- Sponsor
- Aesculap AG
Fabry Disease
Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function
This study will assess the safety, tolerability, and pharmacokinetics (PK) study of a single dose of 150 mg AT1001 (migalastat HCl, GR181413A) administered orally to healthy subjects with normal renal function and to subjects…
- Trial name
- NCT01730469
- Testing
- AT1001 150 mg
- Where
- 4 sites in United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Amicus Therapeutics
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
Hemophilia A
A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion.
- Trial name
- GENEr8-LTE (NCT05768386)
- Where
- 41 sites in United States, Australia and 10 more
- Volunteers
- 172 people
- Sponsor
- BioMarin Pharmaceutical