Clinical Trials

SUMMARY OF CURRENT AND PLANNED CLINICAL RESEARCH PROJECTS:

The Center is active investgating treatments that can improve our patients’ quality of life and better control disease activity.  Current, formal research studies are listed.  New studies are being planned to begin in the near future.

Current – Active Enrollment

1.  Cyclophosphamide in the treatment of scleroderma lung disease (SLSII): Hopkins.  PI is Dr. Robert Wise. An NIH sponsored multicenter trial. SLS-I was a multi-center (we were one Center) investigation that found that cyclophosphamide stabilized active scleroderma lung disease after one year of therapy. The NIH has now approved a second study (SLS II) to compare cyclophosphamide to mycophenolate mofetil. We are one of the Centers and have enrolled several patients in the trial.

2.  Cancer in Systemic Sclerosis: Investigating the Connection.  PI is Dr. Shah.  Sites: Johns Hopkins Scleroderma Center, Division of Oncology, Rheumatic Diseases Research Core Center, Royal Free Hospital Scleroderma Center; Current support through ACR REF Bridge Funding Award and Stabler Foundation with K23 application submitted to NIH): This multidisciplinary translational investigation seeks to probe the link between cancer and systemic sclerosis. Dr. Shah, et al. discovered that patients with the onset of cancer and scleroderma at nearly the same time have a high association with antibodies to RNA polymerase. The antibodies are specific for scleroderma and in our patients also target the cancer tissue. This work is now published and has launched important epidemiologic and basic work to understand how cancer and autoimmunity are linked. Multiple studies are underway.

3.  Determination of the rate of extracellular matrix synthesis and skin proliferation in patients with scleroderma. The PI is Dr. Boin (Kinemed, Inc.):  Ten patients have been enrolled and the recruitment is still open. Preliminary data have confirmed proper labeling of extracellular matrix proteins and subcutaneous fat tissue by “heavy” (deuterated) water. Analysis of collagen turnover rates in the skin of participating patients at different stages of disease is currently under development.

4.  Polarized immune response in SSc-ILD.  PI is Dr Boin:  This study has been investigating T cell polarization in the peripheral blood and bronchoalveolar lavage (BAL) fluid of SSc patients with active ILD. Flow cytometric analysis of cellular repertoires has been conducted together with measurements of relevant pro-fibrotic or pro-inflammatory cytokines and chemokines.

5.  IVIg for active scleromyxedema study. PI is Dr. Hummers:  Scleromyxedema is a very rare disease that causes a mucinous deposition in skin and internal organs. We have evaluated over 25 patients with this disease in our center and have noted a consistent improvement with IVIG treatment. Therefore, we are trying to exploit this treatment benefit to try and understand some of the underlying immunological abnormalities by studying the blood and skin before and after this treatment.

6.  Immune Response in Scleroderma (IRIS) Prospective Cohort Study. The PI is Dr. Boin:  In collaboration with our colleagues of the Department of Computer Science at the Johns Hopkins University, we are developing a computational model (algorithm) for the analysis of multidimensional data integrating measures of the ongoing immune-response with unfolding clinical phenotype of scleroderma patients.

7.  Examination of Sodium Thiosulfate to Treat Severe Calcinosis in Scleroderma. PI is Dr. Shah: In this study, we seek to describe clinical outcomes of scleroderma patients treated with sodium thiosulfate for severe calcinosis.

8.  Measuring Vascular Burden in Scleroderma Associated Raynaud’s Phenomenon. PI is Dr. Shah (MedImmune LLC): This study seeks to determine the reliability of two test measures (magnetic resonance angiography and laser Doppler imaging) in patients with scleroderma-associated Raynaud’s phenomenon. In addition, we are correlating measures of anatomy and perfusion with patient centered measurement tools, examining whether vasospasm can be differentiated from structural vascular abnormalities on MRI, and evaluating the utility of a non-contrast MR method for imaging hand arteries. This study is in the final stages of recruitment.

9.  The Natural History of Pulmonary Hypertension (PAH) in Scleroderma (PHAROS).  Hopkins’ PI is Dr Hummers (Actelion). This is a longitudinal multicenter observational study to examine outcomes in patients either at high risk for PAH or in patients recently diagnosed with pulmonary hypertension. This study continues to enroll patients and has been the source of many new investigations and publications.

10.  Rituxan in Pulmonary Arterial Hypertension.  PI at Hopkins is Dr. Paul Hassoun. This multicenter study seeks to investigate the impact of adding a novel, targeted immune suppressing medication to standard pulmonary hypertension treatments. This study is currently enrolling patients via the pulmonary hypertension clinic in collaboration with our Center.

Current – Enrollment Complete

1.   Biomarkers of Vascular Disease: a Prospective Cohort study. PI is Dr Hummers: The goal of this prospective cohort is to evaluate novel biomarkers that will predict which patients with scleroderma may develop future severe vascular disease (such as digital ulcers or pulmonary hypertension). This study enrolled 300 patients who have been followed for an average of 4.5 years now. We are now in the process of measuring biomarkers for analysis and examining the clinical features and other clinical parameters that may also help us predict these events.

2.   Scleroderma Renal Crisis. PI at Hopkins is Dr. Hummers: This is a web-based international collaborative effort initiated by Dr. Marie Hudson and colleagues in Canada to determine the harm or benefit of prophylactic treatment with ACE inhibitors in those patients who develop scleroderma renal disease. This study has completed enrollment and follow up data is currently being collected and analyzed.