111 Clinical Trials for Various Conditions
Interstitial lung disease (ILD) is a lung condition resulting in inflammation and stiffening of the lung, often associated with connective tissue diseases (CTDs). ILD causes reduction in lung volume, shortness of breath, cough and fatigue therefore has high impact on quality of life and is also the leading cause of death in participants with these conditions. The study will assess whether treatment of CTD-ILD participants with belimumab in addition to standard therapy will result in the stabilization and/or improvement of lung function and improve symptoms associated with ILD with an acceptable safety profile.
Lung Diseases, Interstitial
Background: Elastic fibers affect parts of the body that stretch repetitively, such as the skin, blood vessels, and lungs. Researchers want to use medical and research tests in patients with already identified changes in a set of connective tissue genes to better understand diseases related to elastic fibers. Objective: To learn more about the impact of underlying changes in a set of connective tissue genes on people s overall health. Eligibility: People ages 2-100 who have had a gene test that identified a change in a gene that affects elastic fibers. Initial emphasis will be placed on people with changes in ELN, LOX, MFAP4, FBLN5 and EFEMP2. Design: Participants will visit the NIH for several days to perform tests that will help researchers learn more about how changes in connective tissue genes affect a person s health. They will also have clinicians take their medical history and physical exam. Optional tests include giving blood samples, hair strand, urine, and/or saliva samples. They may have a cheek swab or skin biopsy. Their genes may be studied. Their cells may be grown in a laboratory. Participants may have photographs taken of the face and body. They will receive exams by a medical team with experience in connective tissue disease. They may have a dental exam or eye exams and with photography. During the eye exam, pictures may be taken of the blood vessels in the eyes. If this occurs, they will get dye through an intravenous (IV) line in an arm vein. They will also have medical tests to check the health of tissues that may be affected by these connective tissue genes. Participants may have lung function tests. They may have a six-minute walk test. They may complete a treadmill or bike stress test. Their heart s electrical activity may be recorded. Participants may have X-rays and ultrasounds. They may have a DEXA scan to measure bone density. They may have CT, MRI or other imaging scans. Some of these tests require the participant to get a contrast fluid via IV. Participants may have a skin elasticity test using a suction cup that pulls lightly on their skin. Participants may wear blood pressure cuffs while probes are placed on their skin. The tests will be chosen for each individual based on their specific gene change and no person is expected to complete all tests. Participation will last 3-5 days.
Abnormal Elastogenesis, Vascular Diseases, Connective Tissue Diseases
Researchers are trying to find out more about the safety of a new treatment, Allogeneic (coming from a healthy donor) Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs) which is still experimental, for Interstitial Lung Disease (ILD) associated with Connective Tissue Disorder (CTD).
Interstitial Lung Disease, Connective Tissue Diseases
This study assesses the safety and efficacy of bardoxolone methyl relative to placebo in patients with connective tissue disease-associated pulmonary arterial hypertension to determine the recommended dose range and evaluate the change from baseline in 6-minute walk distance (6MWD) following 24 weeks of study participation.
Connective Tissue Disease-Associated Pulmonary Arterial Hypertension
This registry has been established to gain a better understanding of the clinical and emotional presentations of patients with undifferentiated connective tissue disease.
Undifferentiated Connective Tissue Disease
The purpose of this study was to evaluate the use of the dietary supplement Natural Eggshell Membrane (NEM) for the treatment of joint and connective tissue discomfort \& pain.
Osteoarthritis, Fibromyalgia
The purpose of this study was to evaluate the use of the dietary supplement Natural Eggshell Membrane (NEM) for the treatment of joint and connective tissue discomfort, pain, and flexibility.
Osteoarthritis, Fibromyalgia
Systemic lupus erythematosus (SLE) and mixed connective tissue disease (MCTD) are long-term autoimmune diseases in which the immune system attacks parts of the body. The abnormal immune reaction causes inflammation of and damage to various body parts and can affect joints, skin, kidneys, heart, lungs, blood vessels, and the brain. SLE and MCTD often affect young women, especially black and Hispanic women, and there is no known cure. Knowing more about SLE and MCTD will help in developing new and effective treatments. The purpose of this study is to characterize immune system abnormalities, genetic components, and disease progression in people with SLE and MCTD.
Mixed Connective Tissue Disease (MCTD), Systemic Lupus Erythematosus (SLE)
To explore the hypothesis that leg ulcers are associated with hypercoagulable states, the CLUE study will evaluate patients with connective tissue disease associated leg ulcers, to identify risk factors (especially hypercoagulability and immunologic characteristics), characterize pathogenesis, predict response to therapy, and assess the impact of lower extremity ulcers on quality of life.
Connective Tissue Diseases, Blood Coagulation Disorders, Leg Ulcers, Rheumatoid Arthritis, Systemic Lupus Erythematosus, Systemic Scleroderma, Mixed Connective Tissue Disease
This study will define the major genetic risk and protective factors for idiopathic inflammatory myopathies (IIM), a group of immune disorders affecting connective tissues such as muscles. It will also identify new environmental risk factors for IIM and identify immune responses in myositis and related diseases. There are many forms of IIMs, and the causes of these diseases are unknown. However, scientists suspect that they result when people with some genetic factors that predispose them-that is, put them at greater risk-are exposed to certain environmental triggers. Some of those triggers include food, drugs, biologics (such as a vaccine to prevent disease), medical devices and occupational exposures. Patients, including children under 18, who had a diagnosis of myositis, a related autoimmune disease, or a rheumatic disease, as well as their blood relatives, and control subjects who were in good health have already been recruited for this study. The evaluation consisted of one outpatient visit to the patient's doctor, who will obtain a medical history and conduct a physician examination. Patients spent 20 to 30 minutes to answer written questions. There was a blood collection of about 6 tablespoons. If there was a major change in patients' medical conditions, they were asked to return for a second outpatient evaluation to determine whether any of the blood tests or antibodies, which show an immune response, had changed. Blood samples collected will be used only for laboratory research studies. The samples have been identified by a code, and all other identifying information have been removed. During the study, researchers will explore possible environmental risk factors, including studies of infectious and non-infectious agents. They will analyze the blood for genetic markers and test for certain antibodies. Laboratory results will be evaluated as they relate to the signs, symptoms, and severity of patients' illnesses. That would help researchers to better understand patterns of the diseases and the outcomes for patients. This study will not have a direct benefit for patients. However, results from the study can be made available to patients' doctors for use in appropriate care. Also, it is hoped that information gained can help other people in the future.
Autoimmune/Connective Tissue Diseases, Idiopathic Inflammatory Myopathies, Polymyositis, Dermatomyositis
Background: - Heritable disorders of connective tissue are genetic conditions that can affect the skin and other parts of the body. They are related to mutations in genes that are responsible for building tissues. The symptoms differ among disorders. Researchers want to study which genes may be responsible for different disorders. They will be performing a long-term (up to 10 years) study and a study that requires a single visit. These studies will look at how these disorders affect the body and what genes may cause these conditions. Objectives: - To perform one-time and long-term studies of people who have heritable disorders of connective tissue. Eligibility: - Individuals at least 2 years of age who have or may have a heritable disorder of connective tissue. Design: * Participants will be screened with a physical exam, medical history, and blood samples. * Participants will be on one of two parts of this study. The longitudinal arm will require long-term study over about 10 years. The mutational analysis arm will involve a single visit. * Longitudinal arm participants must be at least 12 years of age. They will have study visits at regular intervals for up to 10 years. The tests given at these visits may include all or some of the following: * Blood, saliva, urine, and skin samples * Heart and lung function tests * Magnetic resonance imaging scans of the neck, chest, spine, and abdomen * Other imaging studies such as x-rays, bone density scans, and ultrasounds * Questionnaires about sleep, pain, and quality of life * Photographs of affected areas. * Mutational analysis arm participants will have a single study visit. They will provide blood and saliva samples. They will provide tissue from a skin biopsy. They will also let the researchers take photos of any affected body parts. They will complete questionnaires about sleep, pain, and quality of life.
Ehlers-Danlos Syndrome, Stickler Syndrome, Marfan Syndrome
This study offers evaluation and treatment of patients with a suspected connective tissue disorder. The protocol is not designed to test new treatments; rather, patients receive standard care. The study is designed to: 1) allow NICHD's staff to learn more about connective tissue disorders, 2) train physicians in the evaluation and treatment of these disorders; and 3) establish a pool of patients who may be eligible for other NICHD protocols for connective tissue disorders. (Participants in this protocol will not be required to join another study; the decision will be voluntary.) Patients of all ages with a suspected connective tissue disorder and their unaffected family members may be eligible for this study. Participants undergo diagnostic procedures that may include a medical history, physical examination, X-ray studies, eye examinations, and blood drawing, as well as other specialized tests, when needed. Additional tests may include: * Blood test for DNA genetic analysis * Skin biopsy: Removal of a small piece of tissue for microscopic examination. The area of skin selected for the biopsy is numbed and a small circle of skin, usually from the upper arm, is removed with a surgical cookie cutter-like instrument. * Magnetic resonance imaging (MRI): This test uses a strong magnetic field and radio waves to show structural and chemical changes in tissues. The patient lies on a table that slides into a narrow cylinder containing a magnetic field. Ear plugs are worn to muffle loud knocking and thumping sounds that occur with electrical switching of the magnetic fields. * Computed tomography (CT) scans: This test allows the doctor to view the organs inside the body in small sections. The patient lies in a doughnut-like machine. Scanning can be done from different angles, allowing a three dimensional picture of the part of the body being studied. It may be done with or without injection of a contrast material. * Referral to appropriate sub-specialists when potential complications are found.
Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
The purposes of this study are to identify the genes responsible for inherited connective tissue disorders and learn about the range of medical problems they cause. It will investigate whether specific gene changes cause specific medical problems and will establish diagnostic criteria (signs and symptoms) for the individual syndromes. Children and adults with a known or suspected inherited connective tissue disorder (Marfan, Ehlers-Danlos or Stickler syndrome, or other closely related disorders) and their family members may be eligible for this study. Patients enrolled in the study will have a medical history, physical examination and blood tests, as well as other procedures that may include: * Echocardiogram (ultrasound of the heart) * X-rays and other imaging studies, such as magnetic resonance imaging (MRI) or computerized tomography (CT) scans * Lung function studies * Urine tests * Skin biopsy (removal of a small piece of tissue, under local anesthetic, for microscopic examination) * Examination by various specialists (e.g., in ophthalmology, gastroenterology, rehabilitation medicine) as needed * Questionnaires regarding chronic pain and fatigue, quality of life, and the impact of the connective tissue disorder on the patient and family. (Patients who wish to enroll but cannot travel to NIH may have a more limited participation, including review of medical records, telephone interview regarding personal and family history, and collection of a specimen (blood, skin biopsy, or other) for genetic testing. Patients will be notified of genetic testing results that show a change responsible for their connective tissue disorder. If they wish, the information will also be sent to their local health care provider, along with recommendations for additional tests or treatment options. No treatment is offered as part of this study. Participating family members who do not themselves have a connective tissue disorder will provide a small blood sample for gene testing and be interviewed by telephone about their personal and family health history. Those whose blood test results show a gene change associated with a connective tissue disorder will be invited to NIH for a discussion of the findings or referred to a genetic center in their area.
Connective Tissue Disease, Dissecting Aneurysm, Ehlers Danlos Syndrome, Marfan Syndrome, Nail Patella Syndrome
This study will examine the possible relationship between silicone implants or injections and the connective tissue diseases scleroderma and myositis. It will explore whether certain factors in the blood or the immune system or other factors are involved in the development of these diseases following silicone implantation or injection. Men and women 18 years of age and older who meet the following criteria may be eligible for this study: Group 1-Patients who have had silicone implants or injections and who later developed scleroderma or myositis Group 2-Patients with scleroderma or myositis who have not had silicone implants or injections Group 3-Healthy volunteers who have had silicone implants or injections and did not develop symptoms or other medical features of connective tissue disorders. Participants will have a thorough history and physical examination, blood and urine tests, chest X-ray and lung function tests. In addition, patients will complete a questionnaire about their procedure (including information such as the types of implanted devices and injections, reason for the procedure, post-operative complications, other illnesses or medical conditions present before and after the procedure, etc.).
Autoimmune Diseases, Connective Tissue Diseases, Scleroderma, Circumscribed, Scleroderma, Systemic
The purpose of this study is to determine the ability of Vascana (0.9% nitroglycerin topical cream) to treat and prevent the symptoms experienced by subject's with Raynaud's Phenomenon. The symptoms of this disease include pain, tingling, and numbness in the fingers of the affected hand or hands.
Raynaud's Phenomenon Secondary to Connective Tissue Disease
Background: - Behcet's disease (BD) is an autoimmune disease where the immune system attacks the body. People with BD may develop oral or genital ulcers, skin problems, and eye disease. Most drugs used to treat BD suppress the immune system, but they are not always helpful and may have side effects. A new drug, anakinra, may be able to treat BD with fewer side effects. Because it has not been studied in people with BD, anakinra is considered an experimental treatment. Objectives: - To test whether anakinra can be a safe and effective treatment for Behcet s disease. Eligibility: - People who have Behcet's disease with ongoing oral or genital ulcers for at least one month, or three or more flares of eye disease in the past 6 months. Design: * Participants will be screened with a physical exam and medical history. They will also have blood and urine tests. They will be divided into two groups: those with oral or genital ulcers and those with eye disease. * All participants will keep a diary of symptoms for a month before starting the study drug. * Participants with oral or genital ulcers will receive daily injections of anakinra for 3 to 6 months. Treatment will be monitored with frequent blood draws and daily diaries. Those who improve but do not have a full response to the drug may receive a higher dose. Those who improve after 6 months may have an extra 6 months on either anakinra or placebo to study the differences in response. * Participants with eye disease will receive anakinra for up to 12 months. Treatment will be monitored with frequent blood draws, daily diaries, and regular eye exams. * All participants will have a final study visit 1 month after stopping the study drug.
Autoimmune Connective Tissue Disorder, Immune System Diseases
The specific objective of this study is to perform a small, open-label study to assess the safety and efficacy of intralesional, subcutaneous injection of STS on calcinosis symptoms and lesion size in systemic sclerosis (SSc), mixed connective tissue disease (MCTD) and dermatomyositis (DM) patients. Injection will be guided by ultrasound, lesion size assessed by ultrasound, and symptom burden by patient-reported outcome measures.
Systemic Sclerosis (SSc), Dermatomyositis, Mixed Connective Tissue Disease (MCTD), Calcinosis
The overall study objectives outlined in this study are to derive 129Xe MRI pulmonary vascular biomarker signatures that differentiate common subtypes of PAH and to determine the ability of 129Xe MRI to longitudinally monitor disease progression and response to therapy in PAH, with the aid of additional assessments, such as labs, echocardiography, and six-minute walk distance (6MWD).
Pulmonary Arterial Hypertension, Idiopathic Pulmonary Arterial Hypertension, Pulmonary Arterial Hypertension Associated with Connective Tissue Disease (Disorder), Connective Tissue Diseases
Social determinants of health (SDoH), defined by the World Health Organization as "the conditions in which people are born, grow, work, live and age and the wider set of forces and systems shaping the conditions of daily life" are estimated to be responsible for nearly 90 percent of a person's health outcomes. SDoH are key contributors to racial, ethnic and socioeconomic disparities in care healthcare access and health outcomes. The goal of this clinical trial is to identify patients with inflammatory arthritis or with a systemic rheumatic condition with arthritis who may respond to the simplest and least expensive intervention to address their SDoH-related needs- a tailored list of resources, those who benefit from a community-based resource specialist to help address specific needs, and those who require a nurse-trained navigator to help both coordinate the services provided by the community-based specialist, and their medical and mental health care and needs. The main questions the clinical trial aims to answer are: 1. To test the efficacy of a rheumatology clinic-based nurse navigator and community resource specialist to reduce appointment no-shows and same-day cancellations in patients with systemic rheumatic conditions with arthritis. 2. To examine the cost-effectiveness of each of the different study interventions for individuals with systemic rheumatic conditions with arthritis with SDoH-related needs using questionnaires and cost-related care metrics. Participants will be randomly assigned to 1 of 3 arms. In Arm 1, patients will receive a cultivated list of resources related to the needs that patients indicate on the social determinants of health questionnaire. Arm 1 is the control arm which receives the current standard of care. In Arm 2, patients will receive the assistance of a community resource specialist (CRS) - an individual without formal medical training with community-based expertise. In Arm 3, patients will receive the assistance of a nurse patient navigator with additional systemic rheumatic condition-specific training who will work with the CRS. After 6 months, patients who do not respond to Arm 1 will move to Arm 2. Patients who do not respond to Arm 2, will move to Arm 3. Patients who do not respond to Arm 3 will remain in Arm 3. Patients who respond to any arm will graduate the program at 6 months. The patients who do not respond be in their new arm for 6 months. At 12 months, all patients remaining in the study will graduate.
Rheumatoid Arthritis, Palindromic Arthritis, Juvenile Rheumatoid Arthritis, Ankylosing Spondylitis, Sacroiliitis, Psoriatic Arthritis, Mixed Connective Tissue Disease, Lupus, Enteropathic Arthropathies, Systemic Sclerosis, Sjogren's Syndrome, Sicca Syndrome, Inflammatory Arthritis, Undifferentiated Connective Tissue Diseases
The main purpose of this study is to assess the safety and efficacy of peresolimab in adult participants with moderately-to-severely active rheumatoid arthritis.
Rheumatoid Arthritis, Arthritis, Joint Diseases, Musculoskeletal Diseases, Rheumatic Diseases, Connective Tissue Diseases, Autoimmune Diseases, Immune System Diseases
The investigators propose to study the relationship between right ventricle (RV) steatosis and RV function, exercise capacity, and outcomes in humans with pulmonary arterial hypertension (PAH) and to identify potential drivers of lipid accumulation.
Idiopathic Pulmonary Arterial Hypertension, Heritable Pulmonary Arterial Hypertension, Pulmonary Arterial Hypertension Associated With Connective Tissue Disease
Data and specimens will be collected longitudinally from patients seen in the UVA Interstitial Lung Disease (ILD) clinic in order to describe the phenotypic expression of various interstitial lung diseases. Samples will also be collected from a control group for comparison purposes. All data will be entered into a repository for future research purposes or screening for new studies that become available. This data will help identify trends and hopefully lead to a better understanding of the disease progression, treatment options, and outcomes.
Interstitial Lung Disease, Idiopathic Pulmonary Fibrosis, Sarcoidosis, Connective Tissue Diseases, Hypersensitivity Pneumonitis, Idiopathic Interstitial Pneumonias
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
Rheumatic Diseases, Adult Onset Still Disease, Ankylosing Spondylitis, Psoriatic Arthritis, Reactive Arthritis, Antiphospholipid Syndrome, Systemic Lupus Erythematosus, Behcet Disease, Dermatomyositis, Polymyositis, Giant Cell Arteritis, Lyme Disease, Mixed Connective Tissue Disease, Polymyalgia Rheumatica, Rheumatoid Arthritis, Sarcoidosis, Systemic Sclerosis, Scleroderma, Sjogren's Syndrome, Undifferentiated Connective Tissue Diseases
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have yet to demonstrate long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well-controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.(1,2,3,4) In 2019 alone, the United States Food and Drug Administration (FDA) has issued approvals for twenty-four new therapies for congenital and acquired hematologic conditions.(5) In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.(6) With this increase in potential new therapies on the horizon, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. ATHN Transcends is a cohort study to determine the safety, effectiveness, and practice of therapies used in the treatment of participants with congenital or acquired non-neoplastic blood disorders and connective tissue disorders with bleeding tendency. The study consists of 7 cohorts with additional study "arms" and "modules" branching off from the cohorts. The overarching objective of this longitudinal, observational study is to characterize the safety, effectiveness and practice of treatments for all people with congenital and acquired hematologic disorders in the US. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.(7)
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
The goal of this study is to determine whether anti-Ro/SSA antibodies are associated with acquired QT prolongation in subjects with connective tissue disease. The investigators will investigate whether gender or race influence correlation between anti-Ro antibody status and prolonged QT interval and the role of inflammatory cytokines in association with anti-Ro antibodies and QT prolongation. The investigators propose to add an additional objective to test whether QT prolongation is reversible with moderate doses of prednisone in patients with QT interval greater than 500 msec.
Long QT Syndrome, Connective Tissue Diseases, Rheumatoid Arthritis
Study ROR-PH-302, ADVANCE CAPACITY, is designed to evaluate the effects of ralinepag therapy on exercise capacity as assessed by change in peak oxygen consumption (VO2) derived from cardiopulmonary exercise testing (CPET) after 28 weeks of treatment
PAH, Pulmonary Hypertension, Hypertension, Connective Tissue Disease, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease, Pulmonary Arterial Hypertension
Patient Power is a patient research network and database (registry) to collect prospective information about demographics, self-reported diagnoses and medications, and willingness to participate in research from participants with rheumatoid arthritis (RA), spondyloarthritis (SpA), other musculoskeletal conditions, chronic neurological conditions like migraine, chronic pulmonary conditions like Chronic Obstructive Pulmonary Disease (COPD), asthma, autoimmune dermatological conditions such as psoriasis, and other chronic inflammatory or immune-mediated conditions. In addition, since patients with chronic conditions often have other co-morbidities like cardiovascular health and obesity-related metabolic disorders, these conditions will also be included. Participants will provide information from their smartphones or personal computers. The information will be used by researchers and clinicians to help patients and their providers make better, more informed decisions about treatment of chronic conditions.
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.
PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease
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 Organization (WHO) Group 1 PAH.
PAH, Pulmonary Hypertension, Pulmonary Arterial Hypertension, Hypertension, Connective Tissue Diseases, Familial Primary Pulmonary Hypertension, Vascular Diseases, Cardiovascular Diseases, Hypertension, Pulmonary, Lung Diseases, Respiratory Tract Disease
Pulmonary hypertension (PH) is a serious condition characterized by a mean pulmonary artery pressure \>=25mmHg on right heart catheterization (RHC). Despite advances in PH care, outcomes are still sub-optimal and further research is required into the pathobiology of the disease and development of biomarkers that can guide clinical care. The investigators are establishing a biobank to collect samples (blood, urine, stool) from patients with pulmonary hypertension, patients at high risk for pulmonary hypertension, healthy controls, and patients undergoing right heart catheterization. Specimens will be stored for future investigations.
Pulmonary Hypertension, Systemic Sclerosis, Mixed Connective Tissue Disease, Heart Failure With Normal Ejection Fraction, Healthy