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Department of Medicine

Department of Medicine

  Division of Rheumatology and Clinical Immunology

CENTERS AND INSTITUTES


Scleroderma


Scleroderma

ONGOING CLINICAL TRIALS

Clinical Drug Trials

Our Scleroderma Center is committed to participating in clinical trials.  We feel it is vital step in working together to find a treatment for Scleroderma.   Without clinical trials, our field will not advance.    If you would like additional information regarding ongoing clinical studies at our Center, please contact one of our scleroderma research coordinators, Dana Ivanco at 412-648-7040 or des2@pitt.edu or Maureen Laffoon at 412-648-7871 or  laffoonm@pitt.edu


Active Clinical Trials

Diffuse Scleroderma 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).  The purpose of this study is to examine the effect of atorvastatin (trade name Lipitor) on Raynaud symptoms and small blood vessel function in patients with early diffuse scleroderma.  Scleroderma is characterized by blood vessel injury, immune system activation and fibrosis. The blood vessel injury is thought to be important early in the disease, and many think this may be the reason most scleroderma patients experience Raynaud as the first symptom. While atorvastatin reduces cholesterol, it is recognized to have many positive effects beyond cholesterol reduction. These include improvement of blood vessel function and reduction of fibrosis. Because of these effects, it is believed that atorvastatin will improve blood vessel function and Raynaud symptoms in patients with early disease. Early disease means <3 years of  scleroderma symptoms for this study).  The trial is 16 weeks and half the patients will receive atorvastatin and half placebo. Atorvastatin (or placebo) is given as an "add-on" therapy.  This means all medications can be continued while in this trial.  There are only 3 visits over 16 weeks.

A Study of Subcutaneous Abatacept to Treat Diffuse Cutaneous Systemic Sclerosis (ASSET): This study examines the safety and effectiveness of abatacept in patients with early diffuse scleroderma (<3 years of symptoms).  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. At the end of that year all patients are allowed to enter the open-label treatment where they are guaranteed to receive drug and not placebo.

A  Phase 2, Double-blind, Randomized, Placebo-controlled Multicenter Study to Evaluate Safety, Tolerability, Efficacy, and Pharmacokinetics of JBT-101 in Diffuse Cutaneous Systemic Sclerosis: JBT-101 is an experimental (investigational) drug that is chemically similar to a chemical in cannabis, or marijuana.  However, this drug has been designed to avoid the "high" feeling of marijuana. This research study is being done to test the safety, tolerability, and efficacy of JBT-101 when it is given to subjects with diffuse cutaneous scleroderma of < 6 years  of disease. JBT-101 is entirely manufactured from chemicals, not plant or animal products. This is a 16 weeks study. Patients will be able to remain on their current scleroderma medications. 2 out of 3 will receive therapy in this study, and 1 in 3 will receive placebo.  There is an open label extension study for this trial.


Pulmonary Hypertension

A Dose-Ranging Study of the Efficacy and Safety of Bardoxolone Methyl in Patients with Pulmonary Hypertension. (LARIAT).
The LARIAT study is a phase 3 study to examine the safety, tolerability, and efficacy of bardoxolone methyl in patients with pulmonary hypertension and pulmonary fibrosis on stable medication therapy.   Bardoxolone methyl is an oral once daily antioxidant inflammation modulator that has been previously studied in pulmonary hypertension.  It  targets the inflammatory components of pulmonary hypertension, and has the potential to reduce the production of enzymes that cause fibrosis.    This study is 16 weeks, with two-thirds of patients receiving drug and one-third of patients receiving placebo.  If patients complete the trial, they are eligible for an open label phase where everyone will receive bardoxolone. 

Rituximab (Rituxan) Study: Rituxan is an immunosuppressive drug originally designed to treat lymphoma, but has been FDA- approved to treat rheumatoid arthritis. Rituximab 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, such as scleroderma. This study is directed at Scleroderma patients who have confirmed pulmonary arterial hypertension (PAH or high blood pressure in the lungs) regardless of how much skin thickening they have.  Rituximab is given by vein twice, two weeks apart. Half of the patients will receive rituximab 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.


Digital Ulcers

Pilot Study To Assess The Efficacy And Safety Of Riociguat Vs. Placebo In Scleroderma-Associated Digital Ulcers (RESCUE): This study examines the safety and effectiveness of Riociguat in scleroderma patients with digital ulcers.  Riociguat is approved by the FDA for the treatment of pulmonary arterial hypertension (PAH).   In this study half the patients will receive drug and half the patients will receive placebo.  Patient must have an active digital ulcer at the time the trial starts.  The total study time will be 36 weeks, with the potential for open label use of riociguat after the trial is completed.   


All Scleroderma Patients

The Scleroderma Patient-centered Intervention Network (SPIN) is an organization of researchers, health care providers, and people living with scleroderma from around the world.  Individuals and organizations involved in SPIN are working on a novel research project with the goal to develop, adapt and test new and existing programs to help people with scleroderma cope with their illness and manage their daily lives. The areas covered by the programs developed by SPIN include managing symptoms, daily tasks, and emotions related to illness, as well as balancing activity and rest.  This study is done using the internet.  There are no study visits once enrolled.