Ongoing 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:

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.

Dimethyl Fumarate, Tecfidera, In Pulmonary Arterial Hypertension Associated With Systemic Sclerosis

Tecfidera is an investigational drug that reduces the activity of chemicals in your body that are triggered by inflammation. This process is called oxidative stress. Oxidative stress is increased with pulmonary arterial hypertension associated with systemic sclerosis. The study is designed to see if Tecfidera is safe and whether it is associated with any improvement in 6-minute walk test in people with pulmonary arterial hypertension associated with systemic sclerosis. Half of the subjects will receive Tecfidera and half placebo. Other PAH medications can be continued throughout the study. This is a 36 week study with a total of 6 visits.

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 II  double-blind,  clinical trial addressing the treatment of  patients  with  active  and symptomatic  scleroderma-related interstitial lung disease (SLSIII):

This study compares the safety and effectiveness of adding on pirfenidone to current mycophenolate (Cellcept) therapy for scleroderma-related lung disease.  Pirfenidone is FDA approved for the treatment of pulmonary fibrosis.  This research is designed to test whether combining pirfenidone and mycophenolate will result in a more rapid and possibly greater improvement in lung function than occurs when mycophenolate is used alone. There are 13 office visits and 11 phone calls over 18 months.

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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