Clinical trials: Vascular Anomaly
Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP)
Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders
The purpose of this study is to assess the safety and efficacy of Immune Globulin Intravenous (Human), 10% (IGIV 10%) in subjects with primary immunodeficiency disorders.
- Conditions
- Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP), Kawasaki Syndrome
- Trial name
- NCT00157079
- Testing
- Immune Globulin Intravenous (Human), 10%
- Where
- 11 sites in United States
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire
Hypertension
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
The purpose of this study is to determine whether 6R-BH4 (sapropterin dihydrochloride) is safe and effective in the treatment of poorly controlled hypertension in the presence or absence of type 2 diabetes.
- Trial name
- NCT00325962
- Testing
- 6R-BH4 (sapropterin dihydrochloride)
- Where
- 24 sites in United States
- Volunteers
- 84 people
- Sponsor
- BioMarin Pharmaceutical
Venous Stenosis, Venous Occlusion
Merit WRAPSODY™ Endoprosthesis for Treatment of Stenosis or Occlusion (WRAP)
The WRAP Study aims to expand and understand the safety and efficacy data on the WRAPSODY Endoprosthesis System in a real-world population.
- Trial name
- NCT05062291
- Testing
- Merit WRAPSODY Endovascular Stent Graft
- Where
- 35 sites in Australia, Brazil and 7 more
- Volunteers
- 500 people
- Sponsor
- Merit Medical Systems, Inc.
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Fabry Disease, Pregnancy
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during…
- Conditions
- Fabry Disease, Pregnancy, Pregnancy Complications
- Trial name
- MOS (NCT06941025)
- Testing
- Not applicable- observational study
- Where
- 5 sites in United States, Germany and 3 more
- Volunteers
- 10 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Tetrology of Fallot, Pulmonary Stenosis
MASA Valve Early Feasibility Study
The MASA Valve Early Feasibility Study (MVEFS) multi-site interventional clinical trial within the United States of America with each center following a common protocol.The objective of the trial is to evaluate the safety and…
- Conditions
- Tetrology of Fallot, Pulmonary Stenosis, Truncus Arteriosus, Transposition of Great Vessels
- Trial name
- MVEFS (NCT05452720)
- Testing
- Surgical Right Ventricular Outflow Tract Reconstruction
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- PECA Labs
Hereditary Angioedema (HAE)
Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema
The primary objective of this study is: assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE The secondary objectives are: evaluate the pharmacokinetic profile of ecallantide in…
- Trial name
- NCT01253382
- Testing
- ecallantide
- Sponsor
- Shire
Hypervascular Tumor and Arteriovenous Malformation
A Multicenter, Open-label Study for E7040 in Japanese Subjects With Hypervascular Tumor and Subjects With Arteriovenous Malformation
To evaluate the efficacy and safety of transcatheter arterial embolization with E7040 in Japanese subjects with hypervascular tumor or arteriovenous malformation
- Trial name
- NCT01677624
- Testing
- E7040
- Where
- 10 sites in Japan
- Volunteers
- 28 people
- Sponsor
- Eisai Co., Ltd.
Von Hippel-Lindau Disease, Malignant Neoplasms
Extension Study for Participants in Studies That Include Belzutifan (MK-6482-043/LITESPARK-043)
Researchers are looking for new ways to treat advanced solid tumors and von Hippel-Lindau (VHL)-related tumors: Advanced means the cancer has spread to other parts of the body (metastatic) or cannot be removed with surgery Solid…
- Trial name
- NCT07405164
- Testing
- Belzutifan, Nivolumab, Lenvatinib
- Where
- 57 sites in United States, Australia and 20 more
- Volunteers
- 450 people
- Sponsor
- Merck Sharp & Dohme LLC
Fabry Disease
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry…
- Trial name
- NCT00140621
- Testing
- Agalsidase beta
- Where
- 7 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Genzyme, a Sanofi Company
Intracranial Arteriovenous Malformations
Efficacy and Safety of the Liquid Embolic System (Tonbridge) for Endovascular Treatment of Cerebrovascular Malformations
The purpose of this study is to verify the efficacy and safety of the Liquid Embolic System (Tonbridge Medical Tech. Co., Ltd. (Suzhou)) for the treatment of cerebrovascular malformations.
- Trial name
- NCT06479343
- Testing
- Liquid Embolic System (Tonbridge), Onyx Liquid Embolic System (Medtronic)
- Where
- 14 sites in China
- Volunteers
- 170 people
- Sponsor
- Ton-Bridge Medical Tech. Co., Ltd
Crohn Disease, Ulcerative Colitis
Expanded Access to Upadacitinib
This is an expanded access program (EAP) for eligible participants. This program is designed to provide access to upadacitinib prior to approval by the local regulatory agency. Availability will depend on territory eligibility.
- Conditions
- Crohn Disease, Ulcerative Colitis, Idiopathic Arthritis (Including sJIA, pJIA, or JPsA), Atopic Dermatitis
- Trial name
- NCT04159597
- Testing
- Upadacitinib
- Sponsor
- AbbVie
Hereditary Angioedema
Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects
The study objective was to describe the pharmacokinetics (PK) of one or two doses of C1 esterase inhibitor (C1INH-nf) in hereditary angioedema (HAE) subjects who were not experiencing an HAE attack.
- Trial name
- NCT00432510
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Shire
Fabry Disease
PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion
Pharmacokinetic/Pharmacodynamic Study of 2 agalsidase beta Formulations in Single Dose Administered to Healthy Volunteers as intravenous infusion, at a concentration of 1 mg/kg
- Trial name
- NCT05343715
- Testing
- Agalsidase beta from Biosidus 1 mg/kg, Fabrazyme (agalsidase beta) 1 mg/kg
- Where
- Argentina
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Bio Sidus SA
Behcet Disease
1 Year of Treatment With Canakinumab in Behçet's Disease Patients With Neurologic or Vascular Involvement
Primary objective of the study was to evaluate the safety and efficacy of canakinumab on the clinical and inflammatory findings of Behced Disease patients with neurologic and vascular involvement.
- Trial name
- Behcet (NCT02756650)
- Testing
- Canakinumab
- Where
- Turkey (Türkiye)
- Volunteers
- 8 people
- Sponsor
- Novartis Pharmaceuticals
Cryptogenic Stroke
Patent Foramen Ovale Closure or Medical Therapy After Stroke - RESPECT Trial
The purpose of this study is to investigate whether percutaneous Patent Foramen Ovale (PFO) closure, using the AMPLATZER PFO Occluder, is superior to current standard of care medical treatment in the prevention of recurrent…
- Trial name
- NCT00465270
- Testing
- AMPLATZER PFO Occluder, Standard of Care - Medical Management
- Where
- 69 sites in United States, Canada
- Volunteers
- 980 people
- Sponsor
- Abbott Medical Devices
Fabry Disease, Anderson Fabry Disease
Treatment-related Benefit and Satisfaction in Fabry Patients. Insight in Patients Expectations and Preferences
In the shared decision-making process, patients should express their expectations and preferences regarding treatment to the physician.
- Trial name
- NCT04043273
- Testing
- Noninterventional characterization of patients expectations and preferences regarding their treatment
- Where
- 15 sites in France
- Volunteers
- 69 people
- Sponsor
- Amicus Therapeutics France SAS
Adenocarcinoma, Adenocystic Carcinoma
The Registry of Oncology Outcomes Associated With Testing and Treatment
This study is to collect and validate regulatory-grade real-world data (RWD) in oncology using the novel, Master Observational Trial construct. This data can be then used in real-world evidence (RWE) generation.
- Conditions
- Adenocarcinoma, Adenocystic Carcinoma, Anal Cancer, Appendix Cancer
- Trial name
- ROOT (NCT04028479)
- Testing
- Biomarker Testing (L), Systemic Treatment (T), Patient Reported Outcomes (P)
- Where
- 2 sites in United States
- Volunteers
- 167 people
- Sponsor
- Taproot Health
Fabry Disease
Evaluate the Safety and Exploratory Efficacy of GC1119
The purpose of this study is to evaluate the safety and exploratory efficacy of GC1119 (recombinant human α-galactosidase A) for enzyme replacement therapy in Fabry disease patients.
- Trial name
- NCT01653444
- Testing
- GC1119
- Where
- 3 sites in South Korea
- Volunteers
- 8 people
- Sponsor
- Green Cross Corporation
Fabry Disease, Cerebrovascular Accident
Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation
New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease.
- Trial name
- sifap2 (NCT00413595)
- Testing
- No intervention
- Where
- 20 sites in Austria, Croatia and 5 more
- Volunteers
- 100 people
- Sponsor
- CENTOGENE GmbH Rostock