Clinical trials: Vascular Anomaly, open to volunteers
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
Retinal Disease, Age-Related Macular Degeneration
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.
- Conditions
- Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease
- Trial name
- SCOTS2 (NCT03011541)
- Testing
- Arm 1
- Where
- 4 sites in United States, Austria and 1 more
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
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
Behçet Disease
Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease
The aim of this study is to estimate the efficacy of apremilast compared to placebo in the treatment of oral ulcers in pediatric participants from 2 to < 18 years of age with oral ulcers associated with Behçet's disease (BD)…
- Trial name
- BEAN (NCT04528082)
- Testing
- Apremilast
- Where
- 27 sites in France, Greece and 6 more
- Volunteers
- 60 people
- Sponsor
- Amgen
Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with…
- Trial name
- DIAMOND (NCT07623525)
- Testing
- DIAG723
- Where
- 11 sites in Australia, Canada and 1 more
- Volunteers
- 93 people
- Sponsor
- Diagonal Therapeutics, Inc.
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Fabry Disease
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.
- Trial name
- NCT06270316
- Testing
- AMT-191
- Where
- 10 sites in United States
- Volunteers
- 20 people
- Sponsor
- UniQure Biopharma B.V.
Hereditary Angioedema (HAE)
A Study to Learn About the C1-Inhibitor Function as Diagnosis for Hereditary Angioedema
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
- AHAE (NCT07293364)
- Testing
- Technochrom C1-INH Kit
- Where
- Algeria
- Volunteers
- 514 people
- Sponsor
- Takeda
Intracranial Aneurysm, Brain Arterial Disease
Safety, Performance and Usability of BALT Medical Devices: The EVIDENCE Post Marketing Clinical Follow-up Platform
BALT has designed an electronic platform to continue collecting clinical data as part of the post-marketing clinical follow-up of its devices.
- Conditions
- Intracranial Aneurysm, Brain Arterial Disease, Acute Ischemic Stroke, Peripheral Vascular Diseases
- Trial name
- EVIDENCE (NCT04927156)
- Testing
- BALT medical devices
- Where
- 12 sites in France, Germany and 1 more
- Volunteers
- 10,000 people
- Sponsor
- Balt Extrusion
Hereditary Angioedema
Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL)
This is a Phase 4, prospective, open-label trial to evaluate the safety and effectiveness of sebetralstat when used for STP for a qualifying procedures in patients 12 years of age or older with hereditary angioedema (HAE).
- Trial name
- NCT07654829
- Testing
- KVD900 600 mg
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Arterio-venous Fistula, Stenosis of Arteriovenous Dialysis Fistula
A First-In-Man Study to Assess Safety and Efficacy of Ultra High Pressure PTA Balloon ULTRAVIOLET in Treating A-V Fistula Stenosis
First-In-Man Single Arm Prospective Study to assess the safety and efficacy of ULTRAVIOLET UHP PTA balloon.
- Trial name
- ULTRAVIOLETFIM (NCT07330817)
- Testing
- PTA balloon
- Where
- Italy
- Volunteers
- 35 people
- Sponsor
- Cardionovum GmbH
Heterozygous Familial Hypercholesterolemia (HeFH)
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
A phase 1/2, two-part trial consisting of an open label dose-escalation part and a randomized, double blind, placebo-controlled part to investigate safety, tolerability, pharmacokinetics, and efficacy with EDIT-401 gene editing…
- Trial name
- NCT07894887
- Testing
- Drug: EDIT-401
- Where
- 2 sites in Australia
- Volunteers
- 56 people
- Sponsor
- Editas Medicine, Inc.
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
Myocardial Ischemia, Heart Defects, Congenital
Spectroscopic Assessment of Myocardial Oxygenation Changes in Pediatric Heart Surgery
The goal of this observational study is to investigate the ability of the SpectroCor Tissue Oxygenation Monitor and its accessory SpectroCor Sensor to observe and monitor changes in cardiac oxygen availability in pediatric…
- Conditions
- Myocardial Ischemia, Heart Defects, Congenital, Reperfusion Injury, Myocardial
- Trial name
- NCT07785141
- Where
- Finland
- Volunteers
- 30 people
- Sponsor
- Spectrocor
PIK3CA-Related Overgrowth Spectrum (PROS), PIK3CA-related Vascular Malformations (PRVM)
Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)
This study is a multi-center, open-label, single arm, phase I/II study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of CYH33 in patients with PIK3CA-related overgrowth spectrum (PROS) and…
- Trial name
- NCT06975618
- Testing
- CYH33
- Where
- 15 sites in China, Japan
- Volunteers
- 141 people
- Sponsor
- Haihe Biopharma Co., Ltd.
Rosacea, Vascular Diseases
Vbeam Pro Pulse Dye Laser for the Treatment of Vascular Conditions
This is a clinical study to evaluate the safety and performance of the Vbeam Pro Laser System for the treatment of vascular indications, including rosacea and port wine stain birthmarks.
- Conditions
- Rosacea, Vascular Diseases, Port-Wine Stain
- Trial name
- NCT07290426
- Testing
- Vbeam Pro Laser Treatment
- Where
- Spain
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Candela Corporation
Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer)
An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
This is a Phase Ib/II clinical study. The Phase Ib dose-escalation study aims to evaluate and determine the recommended Phase II dose (RP2D) of TCC1727 in combination with benmelstobart /olaparib /topotecanfor patients with…
- Conditions
- Solid Cancers, NSCLC (Advanced Non-small Cell Lung Cancer), Gastric (Stomach) Cancer, Endometrial Cancer
- Trial name
- NCT07371663
- Testing
- TCC1727 tablet 90mg, TCC1727 tablet 120mg, TCC1727 tablet 160mg
- Where
- 3 sites in China
- Volunteers
- 266 people
- Sponsor
- Beijing Tide Pharmaceutical Co., Ltd
Fabry Disease
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.
- Trial name
- MODERN (NCT06663358)
- Testing
- Pegunigalsidase-alfa
- Where
- 10 sites in United States, Slovenia and 1 more
- Volunteers
- 100 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Arteriovenous Malformation, Gastrointestinal Bleed
Case-Specific Health Care Professional Clinical Survey for Concerto Versa™ Detachable Coils
The purpose of the Case-Specific Health Care Professional (HCP) Clinical Survey for Concerto Versa™ Detachable Coils, also known as the Concerto Versa HCP Clinical Assessment, is to collect early clinical data from HCPs to assess…
- Conditions
- Arteriovenous Malformation, Gastrointestinal Bleed, Gastric Varices, Hemorrhoid
- Trial name
- NCT07639853
- Testing
- Concerto Versa™ Detachable Coils
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medtronic Endovascular
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of…
- Conditions
- Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
- Trial name
- KH176-301 (NCT06451757)
- Testing
- Sonlicromanol
- Where
- 10 sites in United States, Denmark and 5 more
- Volunteers
- 220 people
- Sponsor
- Khondrion BV