Ongoing Research and Clinical Trials

Clinical Drug Trials and Observational Studies

Our Scleroderma Center is committed to participating in  clinical trials.  We feel it is a vital step in working together to find treatments for scleroderma.   Without clinical trials, our field will not advance. If you are interested in participating in a trial or would like additional information regarding clinical studies at our Center, please contact Maureen Laffoon at laffoonm@pitt.edu.

Currently Enrolling Patients:

Skin Biopsy Research Repository

Enrolling:  Scleroderma Patients and Healthy Controls
The UPMC/University of Pittsburgh Scleroderma Center established a systemic sclerosis (SSc) skin biopsy research repository in which tissue specimens are stored and used for SSc research.  We are enrolling subjects who were diagnosed with SSc and healthy controls. A “control” is a person who does not have a specified disease and could be compared to a person who does have that disease.   To be eligible to participate in this research as a control, you must be 18 years of age or older.  The researchers are also maintaining a parallel SSc database of computerized medical information.

The cause of SSc is not fully understood although hereditary factors, environmental exposures and immune system dysfunction may each play a role.  The SSc skin biopsy research repository and database will allow continued research into the complications, causes and treatment of SSc.  All biopsies and will be processed and stored in Dr. Lafyatis’ research laboratory.   The skin biopsy procedure should take approximately 30 minutes to perform.

The skin cells will be analyzed for potential causes of SSc.  Studies may involve comparing test results of skin from persons with and without SSc. While the nature of future research studies using skin samples is not yet fully known, they may involve identifying inherited factors called genes that can: 1) increase the risk of developing SSc, 2) modify the severity of the disease, or 3) control components of the immune system (immunogenetic studies).   If studies would require that your skin be sent to an outside research center for testing, it would be sent without identifying information.

Evaluation  of   Brentuximab Vedotin for Diffuse Cutaneous Systemic Sclerosis: A   Phase I/II Multicenter, Rando-mized, Double-Blinded Safety Study (BRAVOS):

Brentuximab vedotin (name brand Adcetris) is a drug that was developed and has FDA approval for the treatment of lymphoma.  This research is being done to evaluate the safety and tolerability of brentuximab in the treatment of diffuse skin disease in scleroderma.   Patients must be early in their disease with worsening skin to participate.  Two of three patients will receive brentuximab vedotin, and the other individual, placebo.   The study lasts 48 weeks and involves 14 visits. Patients will be able to remain on their current scleroderma medications.

Optimizing Raynaud Phenomenon Outcome Measures in Systemic Sclerosis (ROSS):

The purpose of this research study is to improve clinical trial design when studying Raynaud phenomenon (RP) in systemic sclerosis (SSc). We are validating a new patient reported questionnaire, testing a smart phone application to assess Raynaud attack frequency/duration and comparing new imaging techniques for skin blood flow in the hands. The study is over one year, as we recognize Raynaud symptoms change across seasons, and wish to gain a better understanding of how seasons affects these new outcome measures. Ultimately, improving outcome measures could aid in having a drug show a positive change and gain approval for Raynaud treatment. The observational study will last 48 weeks, although those who start a new medication for Raynaud medication will be asked to be follow for a longer period so the effect of the new medication can be captured. There are six study visits within those 48 weeks. At each visit, all subjects will be asked to complete a one week diary the number of Raynaud attack, and on the day of the visit complete a small packet of self-administered questionnaires that collect information on your Raynaud symptoms, function, mood, and quality of life. At all visits except week 36, subjects will undergo noninvasive blood flow imaging using a laser speckle contrast imaging (LSCI) and infrared thermography (IT) machine imaging of their hand. Visits will be 90 minutes on the first day, and thereafter 30-60 minutes.

An Observational Study of the Effect of Mycophenolate Mofetil (Cellcept) in Early Diffuse Scleroderma  (MMF STUDY):

This is a NIH-supported single-center study (being performed only in Pittsburgh) to observe the effect of mycophenolate for the treatment of early diffuse scleroderma.   Mycophenolate is one of the most commonly used medications to treat diffuse scleroderma and scleroderma-related pulmonary fibrosis, however we know little about how to predict who will respond well to the medication.   Patients whose physician recommends they should be clinically treated with mycophenolate are eligible to be in this study.   When  in   this  study your doctor may change your medications at any time.   We simply observe the effect of mycophenolate,  collect data on its effect on skin, and collect blood and oral swab samples every 3 months  for the first year a patient is treated with mycophenolate.   This study can easily be combined with regular patient visits.

A Phase 2, Randomized, Placebo-controlled, Double-blind, Open-label Extension Multicenter Study to Evaluate the Efficacy and Safety of KD025 in Subjects with Diffuse Cutaneous Systemic Sclerosis (Kadmon)

This study examines the safety and effectiveness of KD025 in the treatment of diffuse skin disease in scleroderma. Our Center is one of 25 sites in the United States conducting this study. A study-wide sixty subjects will be randomized (1:1:1) to receive orally administered KD025 200 mg once a day, KD025 200 mg twice a day, or placebo. The study participation is 14 months. (4 weeks for screening, 52 weeks of dosing period, and 4 weeks of follow-up) with a total of 18 visits to our Center. The study will be double-blinded for the first 28 weeks followed by an open-label extension of 24 weeks.

A Phase I Open-Label Study to Determine the Safety and Tolerability of AVID200 in Patients with Diffuse Cutaneous Systemic Sclerosis (AVID)

AVID200 is a new drug that interferes with proteins that are thought to play a major role in the development of fibrosis in scleroderma patients. This trial seeks to assess safety and tolerability of AVID200 in those with diffuse cutaneous systemic sclerosis (dcSSc). The drug will be administered by intravenous (IV) infusion once every 2 weeks. Patients will receive a total of 3 infusions, and there will be additional follow-up safety visits for a total of 9 visits over the 23-week study. All participants will receive AVID200.

A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2 Pilot Study Evaluating Intravenous Iloprost in Subjects With Symptomatic Raynaud’s Phenomenon (RP) Secondary to Systemic Sclerosis (EICOS)

This research study is being done to find out more information about the study drug iloprost for the treatment of moderate to severe symptomatic RP in people with SSc.  Iloprost has been approved in the United States and Europe as an inhaled drug to treat people with pulmonary arterial hypertension (high blood pressure that affects the heart and lungs). The drug is also approved in Europe and New Zealand an intravenous (IV) drug for the treatment of severe disabling RP unresponsive to other therapies. The main purpose of this research study is to see if the study drug iloprost has an effect on how often symptomatic RP attacks occur compared to the frequency of symptomatic RP attacks with a placebo.  We will also study the effect of iloprost on how long symptomatic RP attacks last and the severity of the symptomatic RP attacks, compared to attacks with a placebo.  The IV are administered on 5 consecutive days and the study last 9 weeks.

Autologous Stem Cell Transplantation with CD34-Selected Peripheral Blood Stem Cells PBSC in Patients with Treatment Resistant Systemic Sclerosis (SSc)

This study will utilize intermediate doses of radiation and chemotherapy, paired with intense immunoablative serotherapy, in preparation for an autologous stem cell transplant. Here, CD34 selection will remove potentially autoreactive lymphocytes.   The hypothesis is that after T and B-cell depleted autologous graft infusion a normal immune system will reconstitute without regeneration of autoimmunity. In addition, we propose this conditioning regimen is a safer strategy from a cardiopulmonary, urinary, and bone marrow perspective compared to the published work of other centers, nevertheless, leads to improved SSc symptoms with possible delay of disease recurrence or progression.

 Validation of the preliminary Assessment of Scleroderma associated RAynaud’s Phenomenon (ASRAP) questionnaire

A team of Scleroderam specialist developed a new questionnaire for assessing the severity and impact of Raynauds phenomenon (RP) in people affected by systemic sclerosis (SSc). We hope the Assessment of Scleroderma-associated RAynaud Phenomenon (ASRAP questionnaire in short) questionnaire will help us measure the severity of Raynaud’s phenomenon more accurately. Before we start using this questionnaire in clinical trials, we must first undertake work to ensure it is reliable, feasible and measures Raynaud’s symptoms effectively. To achieve this, we are undertaking a study to test the new questionnaire across a large group of people with SSc. The study itself will involve the completion of the new ASRAP questionnaire and then complete a small pack of additional self-administered questionnaires, which shall collect information on Raynauds symptoms.  This study only requires one visit.  We anticipate approximately 20 minutes of your time to complete the study.

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