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248 recruiting in MO
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Comparison of the Outcomes of Single vs Multiple Arterial Grafts in Women

Heart Diseases Β· Coronary Artery Disease Β· Coronary Artery Bypass Grafting

The central hypothesis of ROMA:Women is that the use of multiple arterial grafting (MAG) will improve clinical outcomes and quality of life (QOL) compared to single arterial grafting (SAG). The specific aims of ROMA:Women are: Aim 1: Determine the impact of MAG vs SAG on major adverse cardiac and cerebrovascular events in women undergoing coronary artery bypass grafting (CABG). The investigators will compare major adverse cardiac and cerebrovascular events (death, stroke, non-procedural myocardial infarction, repeat revascularization, and hospital readmission for acute coronary syndrome or heart failure) in a cohort of 2,300 women randomized 1:1 to MAG or SAG. Differences by important clinical and surgical subgroups (patients younger or older than 70 years, diabetics, racial and ethnic minorities, on vs off pump CABG, type of arterial grafts used) will also be evaluated. The women enrolled in the ongoing ROMA trial (anticipated to be approximately 690) will be included in ROMA:Women, increasing efficiency and reducing enrollment time. Hypothesis 1.0. MAG will reduce the incidence of major adverse cardiac and cerebrovascular events. Hypothesis 1.1. The improvement with MAG will be consistent across key subgroups. Aim 2: Determine the impact of MAG vs SAG on generic and disease-specific QOL, physical and mental health symptoms in women undergoing CABG. The investigators will compare generic (SF-12, EQ-5D) and disease-specific (Seattle Angina Questionnaire) QOL and physical and mental health symptoms (PROMIS-29) in a sub-cohort of 500 women randomized 1:1 to MAG or SAG (including those enrolled in ROMA:QOL). Differences by important subgroups (as defined above) will also be evaluated. Hypothesis 2.0. MAG will improve generic and disease-specific QOL compared to SAG. Hypothesis 2.1. MAG will improve physical and mental health symptoms compared to SAG. Hypothesis 2.2. The improvement with MAG will be consistent across key subgroups.

St Louis, MO18+ yrsWomen
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A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Neutropenia

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

St Louis, MO12+ yrsAll genders
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A Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy

Non-obstructive Hypertrophic Cardiomyopathy

FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxstat compared to placebo on top of Standard of Care in patients with symptomatic nHCM

St Louis, MO18+ yrsAll genders
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TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to: * Evaluate the efficacy of nucresiran compared to placebo on reducing all-cause mortality and cardiovascular (CV) events * Evaluate the efficacy of nucresiran compared to placebo on additional assessments of CV events and/or death * Evaluate the efficacy of nucresiran compared to placebo on patient-reported health status and health-related quality of life

St Louis, MO18–85 yrsAll genders
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Study of BB-031 in Acute Ischemic Stroke Patients (RAISE)

Acute Ischemic Stroke

The purpose of this study is to evaluate the safety and tolerability of ascending doses (Part A) and selected doses (Part B) of BB-031 in acute ischemic stroke patients presenting within 24 hours of stroke onset. Participants will be randomized to receive one dose of either the investigational drug or placebo and will be followed for 90 days. A total of 228 patients are planned in this study.

St Louis, MO18+ yrsAll genders
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Proximal Internal Carotid Artery Acute Stroke Secondary to Tandem or Local Occlusion Thrombectomy Trial

Acute Ischemic Stroke

The primary objective is to establish the efficacy of intra-arterial (IA) mechanical thrombectomy (MT) with extracranial proximal carotid artery acute stenting versus non-stenting approaches in patients with acute ischemic stroke (AIS) from intracranial vessel occlusion (IVO) in the anterior circulation and have a proximal carotid occlusive disease (occlusion or severe stenosis).

St Louis, MO18–79 yrsAll genders
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THRIVE- THerapeutic IntravasculaR Ultrasound (TIVUSβ„’) REnal Denervation System Versus Sham for the Adjunctive Treatment of Hypertension

Hypertension

The primary objective of the THRIVE Pivotal study is to demonstrate the adjunctive effectiveness and the safety of the TIVUS system in: 1. subjects with uncontrolled hypertension (HTN) receiving 0 - 2 anti-hypertensive drugs of different classes in whom the anti-hypertensive medications will be stopped for a 4-week wash-out period before RDN/Sham procedure and during 2 months after procedure. 2. subjects with controlled hypertension receiving 1 - 2 anti-hypertensive drugs of different classes and who accept to be off-medications for a 4-week wash-out period before RDN/Sham procedure and 2 months after the procedure

Kansas City, MO22–75 yrsAll genders
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A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia

Hypoxic-Ischemic Encephalopathy

Hypoxic-ischemic encephalopathy (HIE) affects approximately 4,000 to 12,000 persons annually in the United States. Mortality from HIE has been reported up to 60%, with at least 25% of survivors left with significant neurocognitive disability. Despite this vital unmet medical need, no pharmacological adjunct or alternative therapy has proven beneficial in improving outcomes in neonatal HIE. RLS-0071 is a novel peptide being developed for the treatment of neonatal HIE. This study is designed to evaluate the safety and tolerability of RLS-0071 in the treatment of newborns with moderate or severe HIE.

St Louis, MOAll agesAll genders
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Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE) Β· Hereditary Angioedema - Type 1 Β· Hereditary Angioedema - Type 2

Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with Type I and Type II hereditary angioedema (HAE). The study will also include pharmacodynamic (PD), pharmacokinetic (PK), and health-related quality of life (HRQoL) measurements.

St Louis, MO18+ yrsAll genders
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Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)

Alagille Syndrome Β· Progressive Familial Intrahepatic Cholestasis

The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) treated with Livmarli.

Kansas City, MOAll agesAll genders
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Percutaneous Intervention Versus Observational Trial of Arterial Ductus in Low Weight Infants

Ductus Arteriosus, Patent

Patent Ductus Arteriosus is a developmental condition commonly observed among preterm infants. It is a condition where the opening between the two major blood vessels leading from the heart fail to close after birth. In the womb, the opening (ductus arteriosus) is the normal part of the circulatory system of the baby, but is expected to close at full term birth. If the opening is tiny, the condition can be self-limiting. If not, medications/surgery are options for treatment. There are two ways to treat patent ductus arteriosus - one is through closure of the opening with an FDA approved device called PICCOLO, the other is through supportive management (medications). No randomized controlled trials have been done previously to see if one of better than the other. Through our PIVOTAL study, the investigators aim to determine is one is indeed better than the other - if it is found that the percutaneous closure with PICCOLO is better, then it would immediately lead to a new standard of care. If not, then the investigators avoid an invasive costly procedure going forward.

St Louis, MO0–0 yrsAll genders
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Recruiting

Optimizing Reperfusion to Improve Outcomes and Neurologic Function

Acute Ischemic Stroke

The goal of this study is to evaluate the safety and efficacy of JX10 versus placebo in participants with Acute Ischemic Stroke (AIS) who present for care within 4.5 to 24 hours. The main question the study aims to answer are: 1. JX10 improves functional outcomes as measured by the modified Rankin Scale score when compared with placebo following AIS. 2. Risk of symptomatic intracranial hemorrhage of JX10 in participants with AIS. During Part 1, participants will be randomized to JX 10 (1mg/kg, 3 mg/kg) or placebo. During Part 2, participants will receive JX10 (optimal dose chosen from Part 1) or placebo.

St Louis, MO18–90 yrsAll genders
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Conquer-AF Protocol for Redo Ablation Procedures in Recurrent Paroxysmal and Persistent Atrial Fibrillation Using the Sphere-9 Catheter and Affera Ablation System (Conquer-AF)

Atrial Fibrillation (AF) Β· Persistent Atrial Fibrillation Β· Paroxysmal Atrial Fibrillation (PAF)

Conquer-AF is a prospective, multi-center, interventional, non-randomized clinical study that will enroll up to 400 subjects at up to 30 sites across the United States, Europe, and Australia. Subjects diagnosed with symptomatic recurrent paroxysmal or persistent atrial fibrillation after a single prior ablation procedure will be enrolled in this study. Each subject will undergo a study index redo ablation procedure using the Sphere-9 Catheter and Affera Ablation System and will be followed for 12 months. The expected total study duration (from time of first enrollment to the exit of last enrolled subject) is approximately 2 years.

Kansas City, MO18+ yrsAll genders
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Bleeding Patterns in Sequential and Continuous Progesterone Supplementation in Adolescents With Turner Syndrome

Turner Syndrome Β· Primary Ovarian Insufficiency (Poi)

This is a single-site open label non-randomized study comparing effects of sequential versus continuous use of progesterone supplementation amongst Turner Syndrome (TS) patients with primary ovarian insufficiency (POI) prescribed hormone replacement therapy (HRT).

Kansas City, MO12–20 yrsWomen
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Comprehensive HHT Outcomes Registry of the United States (CHORUS)

Hereditary Hemorrhagic Telangiectasia Β· Arteriovenous Malformations Β· Telangiectasia

The Comprehensive HHT Outcomes Registry of the United States (CHORUS) is an observational registry of patients diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT). The purpose of this study is to better understand HHT, the symptoms and complications it causes, and the impact the disease has on people's lives. The investigators will collect long-term information about the participant, allowing us to understand how the disease changes over time, and what factors can influence those changes. Ultimately, this should help improve treatments for the disease. Another important goal of the study is to provide a way to contact people to participate in future clinical trials and other research. The registry will be a centralized resource for recruitment for clinical trials. People in the registry will not be obligated to join any of these additional studies, but if interested, can agree to be contacted if they may be eligible for a study. Participants will: * Be asked to provide permission to collect information from their medical records, including things like demographic information, diagnosis information, family history, test results, treatment information, symptoms, complications, lifestyle and other relevant medical information. * Be asked study-related questions by phone or at a clinic visit. * Be asked study-related questions every year after enrollment for up to 10 years or until the study ends. A member of the study team will communicate with participants by phone or at clinic visits to collect information regarding any changes to their health over the previous year/s including new test results, treatment information, symptoms, and complications from HHT.

St Louis, MOAll agesAll genders
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