Department of Medicine

University of Pittsburgh

Research at our Center

Many of the research studies include a patient stipend. Payment to research participants is considered compensation for time and inconvenience. There may also be reimbursement for travel to our site. The amount and schedule of all payments will be described in the informed consent form.

CLINICAL DRUG TRIALS

The Effect of Atorvastatin on Microvsacular Endothelial Function and Raynaud in Early Diffuse Scleroderma (TAMER): This is a NIH-supported single-center study (being done only in Pittsburgh) examining the effect of atorvastatin (trade name Lipitor) on Raynaud symptoms and small blood vessel circulation and function in patients with early diffuse scleroderma (less than 3 years since first scleroderma symptom). Systemic sclerosis is a disease characterized by blood vessel injury, immune system activation and fibrosis. Blood vessel injury is thought to be important early in the disease. Blood vessel complications of systemic sclerosis include Raynaud phenomenon, fingertip and toe ulcers, and pulmonary hypertension. While atorvastatin reduces cholesterol, it is recognized to have many effects beyond cholesterol reduction. These include improvement of blood vessel function and reduction of fibrosis. We hypothesize that treatment with atorvastatin over 16 weeks will improve blood vessel function and Raynaud symptoms in patients with early disease. Half the patients will receive atorvastatin and half placebo. Atorvastatin (or placebo) is given as an "add-on" therapy, so all medications can be continued while in this trial. It involves a total of 3 visits over 16 weeks.

For more information on this study, please contact one of our Research Coordinators, Dana Ivanco, at 412-648-7040, des2@pitt.edu, or Maureen Laffoon, at 412-648-7871, laffoonm@pitt.edu.


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A Phase 2a Double-Blind, Randomized, Placebo-Controlled Crossover Trial of the Acute Peripheral Vascular Effects, Safety and Tolerability of Alprostadil Topical Cream in Subjects with Raynaudís Phenomenon Secondary to Systemic Sclerosis. (RayVa). This study examines the safety and effectiveness of a medication called aprostadil on Raynaud symptoms and blood flow of the hands in patients with scleroderma. Alrpostadil is a prostaglandin, which acts to open blood vessels (a vasodilator). It has been approved in an injection form for erectile dysfunction in the US since 1995. In this study it is applied as a cream placed on the fingers of one hand. Patients will be seen twice. On one visit they will have cream with drug placed on their hand and their response to the medication in terms of blood flow measured. On another visit they will receive the cream on their hand without the drug followed again by blood flow monitoring. All scleroderma patients with moderate Raynaud symptoms are eligible for this study.

For more information on this study, please contact our Research Coordinator, Maureen Laffoon, at 412-648-7871, laffoonm@pitt.edu or Dana Ivanco, at 412-648-7040, des2@pitt.edu.


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A Study of Subcutaneous Abatacept to Treat Diffuse Cutaneous Systemic Sclerosis (ASSET): This study examines the safety and effectiveness of abatacept on patients with diffuse systemic sclerosis of less than 3 years duration. Abatacept (trade name Orencia) is a medication which has been FDA-approved since 2005 for the treatment of rheumatoid arthritis. It is administered as an injection (at home) once weekly. In this study half the patients will receive drug and half the patients will receive placebo over one year. The study is for one year, but patients may start an additional medication at six months for their scleroderma, if needed.

For more information on this study, please contact our Research Coordinator, Dana Ivanco, at 412-648-7040 or des2@pitt.edu


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Rituximab (Rituxan) Study: Rituxan is an immune suppressing drug currently used by hematologists for certain malignancies such as lymphoma. It is also approved for use in rheumatoid arthritis. Rituxan eliminates B cells from the blood stream. These cells participate in immune responses and may be responsible for some types of immune injury to tissues in patients with rheumatoid arthritis, lupus, and other related diseases, including Scleroder ma. It is given by vein twice, two weeks apart. This study is directed at Scleroderma patients who have confirmed pulmonary arterial hypertension (PAH or high blood pressure in the lungs) for less than 3 years, regardless of how much skin thickening they have. Half of the patients will receive Rituxan and half placebo. A right heart catheterization both before the study (to determine eligibility) and after 6 months on treatment (or placebo) is required. Other PAH medications can be continued throughout the study. Patients will be followed for 1 year or until the B cells in their blood have returned.

For more information on this study, please contact our Research Coordinator, Dana Ivanco, at 412-648-7040 or des2@pitt.edu.


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OBSERVATIONAL STUDIES

Banking of Biological Samples and Collection of Clinical Data for Connective Tissue Disease (CTD) Research: Connective tissue diseases are chronic diseases that involve damage of connective tissues such as the lining of the joints, blood vessel walls, muscle, skin, and certain internal organs. These diseases are also known as autoimmune diseases because the body's immune system mistakenly attacks its own tissues. Treatment has improved over the past 20 years, but there is still much that is not understood about the complications, causes, and treatments of these diseases. The purpose of this project is to set up and maintain a Rheumatology Biological Specimen Bank of blood and tissue samples and a parallel Research Databank of computerized medical information. For example, we are interested in obtaining and banking blood and skin samples (from biopsies) on patients with scleroderma. Inclusion Criteria: all ages, male or female, diagnosed by a doctor within the Department of Rheumatology with a CTD or a normal healthy individual willing to provide a blood or skin sample.

For more information on this study, please contact our Research Coordinator, Mary Lucas, at 412-383-8699 or mrl6@pitt.edu


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PHAROS: Pulmonary hypertension (PH) is increased blood pressure in the arteries supplying the lungs. In Scleroderma patients, this is caused by thickening of these arteries. It is one of the more serious complications of Scleroderma. Patients with early PH may have little warning before they develop severe shortness of breath (which is called dyspnea). The tests doctors commonly order now don't always catch the early warning signs, but they are the best tests currently available. This study will follow Scleroderma patients who are at risk to develop PH and those who have been diagnosed recently with PH. Investigators will gain a better understanding of who is likely to develop PH and their response to different therapies. This study will hopefully lead to earlier treatment or possibly prevention of PH. There are currently 15 Scleroderma research centers in the United States, including the University of Pittsburgh, enrolling adult patients with limited or diffuse Scleroderma into the PHAROS Registry. Patients who consent will provide information from Scleroderma-related medical records for the duration of this 5 year study. They will provide a single blood sample for research. They will complete questionnaires every 6 months (or more frequently if necessary), which can be sent by mail, or can be done on-line. Medical records will be collected every time patients undergo standard tests and procedures such as pulmonary function tests or echocardiograms. The information collected by this Registry will be used to develop a standard for diagnosing and treating one of the most serious complications people with Scleroderma encounter.

For more information on this study, please contact our Research Coordinator, Dana Ivanco, at 412-648-7040 or des2@pitt.edu.


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PRESS: Scleroderma is an uncommon autoimmune disease. There are two major forms of scleroderma, limited and diffuse. Limited scleroderma has skin thickening limited to the hands, arms and face and may affect the internal organs. Diffuse scleroderma usually progresses more quickly, and can have skin thickening of the entire body and involvement one or more internal organs, such as kidneys, esophagus, heart and lungs. This is an observational study of patients diagnosed by a rheumatologist with diffuse scleroderma within the last 2 years. An observational study is one in which individuals are observed and/or certain outcomes are measured but no experimental treatment procedures are done. There are currently 10 Scleroderma research centers in the United States, including the University of Pittsburgh, enrolling adult patients with early diffuse Scleroderma into the PRESS Registry. Patients who consent will provide information from Scleroderma-related medical records for the duration of this long term study. They will provide blood samples for research at each yearly clinical visit. They will complete questionnaires every 6 months (or more frequently if necessary), which can be sent by mail, or can be done at their regularly scheduled clinic visit. The Centers hope that by working together we can advance research faster.


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PEDIATRIC RESEARCH

Antinuclear Antibodies in Childhood Onset Scleroderma and the Development of a National Childhood Onset Scleroderma Registry (NRCOS): Experts agree that reports on childhood onset scleroderma, both the systemic and localized forms, are limited by small numbers of subjects, that few valid conclusions can be made based on such studies, and that research progress on this group of disorders has thus been hindered. The purpose of this study is to establish and promote a large registry of subjects with childhood onset scleroderma to stimulate future research on this group of disorders. Researchers will also examine the serological profiles in subjects with childhood onset scleroderma and determine associations with clinical and laboratory features of this group of disorders. Inclusion criteria: males and females who were less than 16 years old at the time of their first symptoms, less than 30 years old at time of enrollment, and have a diagnosis of either systemic sclerosis or localized scleroderma, or have Raynaud's phenomena or a positive ANA level. Participants can be enrolled remotely from anywhere in the country.

For more information on this study, please contact our Research Coordinator, Christina Kelsey, at 412-692-6478 or christina.kelsey@chp.edu.


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Development of Clinical Disease Outcome Measures with Biological Substudies for Localized Scleroderma (LOCUS-2): Scleroderma is an autoimmune disease of unknown etiology that is associated with skin changes including induration and hardening. The disease can occur as a systemic form (systemic sclerosis), or a localized form (localized scleroderma). Among children, localized scleroderma (LS) is much more common than systemic sclerosis. There are a few outcomes measures available for LS, however there are not standardized and used for clinical trial yet. This is an extension of a prior approved prospective multi-center study (LOCUS-1), which aimed to develop and validate clinical measures for evaluating disease activity both for clinical practice and research trials. This protocol will further standardize and refine clinical assessments. There will be two optional laboratory substudies, one to evaluate cytokine and T cell patterns during disease activity and damage, and the other to evaluate genetic markers to identify novel immunogenetic profiles specific to different types of localized scleroderma. Inclusion criteria: males and females who have a diagnosis of LS confirmed by a pediatric rheumatologist or dermatologist, who were less than 16 years old at time of disease onset and who are able to cooperate with clinical evaluation and complete the quality of life forms.

For more information on this study, please contact our Research Coordinator, Christina Kelsey, at 412-692-6478 or christina.kelsey@chp.edu.


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