Clinical trials: Small Vessel Disease
Fabry Disease
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on…
- Trial name
- FAVORIT (NCT06880250)
- Where
- 23 sites in Russia
- Volunteers
- 100 people
- Sponsor
- NPO Petrovax
Fabry Disease
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to < 12 years of age with Fabry disease and with amenable GLA variants.
- Trial name
- NCT06904261
- Testing
- Migalastat HCl 20 mg
- Where
- 12 sites in United States, Belgium and 4 more
- Volunteers
- 8 people
- Sponsor
- Amicus Therapeutics
Leiomyosarcoma, Liposarcoma
MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma
This study will evaluate the safety and efficacy of MASCT-I combined with Doxorubicin and Ifosfamide for first-line treatment in patients with advanced soft tissue sarcoma.
- Conditions
- Leiomyosarcoma, Liposarcoma, Synovial Sarcoma, Angiosarcoma
- Trial name
- NCT06277154
- Testing
- MASCT-I, Doxorubicin, Ifosfamide
- Where
- China
- Volunteers
- 148 people
- Sponsor
- HRYZ Biotech Co.
Heart Failure, Chronic Heart Failure
Wearable Tissue Oximetry During Arm Cuff Occlusion, Walking, and Standing Tests in Outpatients With Chronic Heart Failure
This prospective, single-center, observational pilot study evaluated an investigational wearable tissue oximeter (NIRSense Argus Oximetry System, also called Envello Core) in adult outpatients with chronic heart failure in New…
- Trial name
- NCT07874386
- Testing
- Argus Oximetry System
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- NIRSense Inc.
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
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
B-cell Malignancy, Chronic Lymphocytic Leukemia
AS-1763 in Patients With Previously Treated CLL/SLL or Non-Hodgkin Lymphoma
This is an open-label, multi-center Phase 1b clinical study of oral AS-1763 (docirbrutinib) in patients with CLL/SLL or B-cell NHL who have failed or are intolerant to ≥2 lines of systemic therapy.
- Conditions
- B-cell Malignancy, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Waldenstrom Macroglobulinemia
- Trial name
- NCT05602363
- Testing
- Docirbrutinib
- Where
- 13 sites in United States
- Volunteers
- 120 people
- Sponsor
- Carna Biosciences, Inc.
Neoplasms
Patient-Reported Outcome Measurement Information System (PROMIS) Physical Function
The objective of this study is to conduct in-person qualitative interviews of subjects who have different tumor types to identify and assess relevant PROMIS physical function items that can be utilized in future studies.
- Trial name
- NCT03351140
- Testing
- Qualitative Interviews, PROMIS Physical Function Items
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- GlaxoSmithKline
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Coronary Artery Disease, Myocardial Ischemia
BIOFLOW-III Austria Satellite Registry
This registry is a clinical post-market evaluation of the Orsiro LESS in subjects requiring coronary revascularization with Drug Eluting Stents (DES).
- Trial name
- NCT01667016
- Where
- 8 sites in Austria
- Volunteers
- 305 people
- Sponsor
- Biotronik Vertriebs-GmbH
Coronary Artery Disease, Drug Eluting Balloon
Stents Versus PAclitaxel Coated Balloons for Revascularization of CompleX and Small Coronary Vessels (SPARX) Trial
The main objective of the SPARX trial is to compare paclitaxel-coated balloons to with contemporary DES in complex and small coronary artery lesions in patients with NSTEACS or CCS; the co-primary objective is to compare two…
- Conditions
- Coronary Artery Disease, Drug Eluting Balloon, Drug Eluting Stent
- Trial name
- SPARX RCT (NCT07397845)
- Testing
- Protégé and Protégé NC, Agent, Drug Eluting Stent
- Where
- Switzerland
- Volunteers
- 1,380 people
- Sponsor
- Translumina GmbH
ANCA Associated Vasculitis (AAV)
Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)
- Trial name
- NCT06868290
- Testing
- Rapcabtagene autoleucel, Active Comparator, Glucocorticoids
- Where
- 39 sites in United States, Brazil and 6 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
PAH, Pulmonary Hypertension
A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients
Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health…
- Conditions
- PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension
- Trial name
- NCT03626688
- Testing
- Ralinepag
- Where
- 209 sites in United States, Argentina and 31 more
- Volunteers
- 687 people
- Sponsor
- United Therapeutics
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire
Critical Limb Ischemia, Peripheral Arterial Disease
Pilot Safety Study of Injectable SIS for Critical Limb Ischemia
This study is intended to collect safety and effectiveness data on the Cook Injectable Small Intestinal Submucosa (SIS)
- Conditions
- Critical Limb Ischemia, Peripheral Arterial Disease, Peripheral Vascular Disease
- Trial name
- CLIC (NCT02145845)
- Testing
- Injectable SIS
- Where
- Czechia
- Volunteers
- 13 people
- Sponsor
- Cook Group Incorporated
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
Safety, PK, PD, and Antitumor Activity of Vecabrutinib (SNS-062) in B Lymphoid Cancers
This is an open-label Phase 1b/2 study in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL)or non hodgkin's lymphoma (NHL) who have failed prior standard of care therapies including a BTK inhibitor…
- Conditions
- Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Lymphoplasmacytoid Lymphoma, Mantle-Cell Lymphoma
- Trial name
- NCT03037645
- Testing
- SNS-062
- Where
- 11 sites in United States
- Volunteers
- 39 people
- Sponsor
- Sunesis Pharmaceuticals
Microscopic Polyangiitis (MPA), Granulomatosis With Polyangiitis (GPA)
Special Drug Use-results Survey for Long-term Use（Avacopan）
The purpose of this survey is evaluating the safety and efficacy of long-term administration of avacopan for Japanese patients with microscopic polyangiitis or granulomatosis with polyangiitis under actual conditions of use.
- Trial name
- NCT06758271
- Testing
- Avacopan
- Where
- Japan
- Volunteers
- 250 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Coronary Stenosis
Assess the Efficacy and Safety of RESTORE Paclitaxel Eluting Balloon Versus RESOLUTE Zotarolimus Eluting Stent for the Treatment of Small Coronary Vessel Disease
The purpose of this study is to evaluate the efficacy and safety of RESTORE Paclitaxel eluting balloon in the treatment of Chinese patients with coronary heart disease and small vessel lesions in comparison with RESOLUTE…
- Trial name
- NCT02946307
- Testing
- Restore DEB（dimeter>2.00 mm）, Resolute DES, Restore DEB（dimeter:2.00 mm）
- Where
- 12 sites in China
- Volunteers
- 240 people
- Sponsor
- ZhuHai Cardionovum Medical Device Co., Ltd.
Non-Small Cell Lung Cancer, Ovarian Cancer
A Study of CART-TnMUC1 in Patients With TnMUC1-Positive Advanced Cancers
Multi-center, open-label, first in human Phase 1 study of the safety, tolerability, feasibility, and preliminary efficacy of the administration of genetically modified autologous T cells (CART-TnMUC1 cells) engineered to express…
- Conditions
- Non-Small Cell Lung Cancer, Ovarian Cancer, Fallopian Tube Cancer, Triple Negative Breast Cancer
- Trial name
- NCT04025216
- Testing
- CART-TnMUC1, Cyclophosphamide, Fludarabine
- Where
- 10 sites in United States
- Volunteers
- 16 people
- Sponsor
- Kite, A Gilead Company
Fabry Disease
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics