Clinical Trials and Research Projects:
The Center is actively investigating 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. IVIG Treatment in Skin Disease. The PI is Dr. Hummers. Sites: Johns Hopkins Scleroderma Center and Georgetown University. This study is investigating whether IVIG will soften the skin of scleroderma patients. We are actively recruiting individuals with diffuse scleroderma who have a disease duration of less than 5 years with active skin involvement. Participants must also have experienced no improvement or worsening skin in the past 4 months in spite of treatment6 with methotrexate, Cellcept, Imuran, anti-TNF agent or D-penicillamine. There is a 1 in 4 chance of receiving placebo in this trial.
2. Cancer in Systemic Sclerosis: Investigating the Connection. The 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. SPIN Study. The PI is Dr. Wigley. Johns Hopkins is one of many international collaborators cooperating with Principal Investigator, Brett D. Thombs from the Lady Davis Institute for Medical Research of the Jewish General Hospital in Montreal, Quebec, Canada. The overall study goal is to enroll 1,000 and 2,000 participants with scleroderma over the next 5 years. Participants will be asked to complete online questionnaires every 3 months. The long-term goal of the study is to develop a way to offer low cost, easily accessible interventions that will improve quality of life.
4. Botox Study. The PI is Dr. Scott Lifchez. We are actively recruiting for this study. This study is being done to determine whether Local injection with Botulinum Toxin A into the space around the digital vessels will improve Raynaud’s phenomenon symptoms in scleroderma patients. This study is complete.
5. Skin Biopsy Specimens as Biomarkers of Scleroderma and the Response to Mycophenolate Mofetil (MMF or Cellcept). The PI is Dr. Zsuzsanna McMahan at Johns Hopkins University. We are one of several sites conducting this study. The purpose of this study is to assess whether skin biopsy specimens from patients with limited cutaneous scleroderma and diffuse scleroderma can be used as measures of activity of disease to predict disease course and response to myocophenolate mofetil (MMF), a commonly prescribed drug to treat SSc skin and lung disease. There is currently no way to predict disease course or whether a patient will benefit from MMF. We are actively enrolling individuals with diffuse scleroderma or who meet at least three of the five criteria for CREST (Calcinosis, Raynaud Phenomenon, Esophageal dysmotility, Sclerodactyly, Telangiectasias) and who will be beginning MMF treatment for their disease.
6. The Natural History of Pulmonary Hypertension (PAH) in Scleroderma (PHAROS). The PI is Dr Hummers (Actelion) at Johns Hopkins. 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. This study is complete but data analysis is ongoing.
7. Rituxan in Pulmonary Arterial Hypertension. The PI Dr. Paul Hassoun at Johns Hopkins. 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.
8. Prospective Registry of Early Systemic Sclerosis (PRESS). The goal of this registry is to better our understanding of the epidemiology and biology of early diffuse systemic sclerosis. This study is actively recruiting.
9. Gastrointestinal involvement registry. The PI is Dr. McMahan at Johns Hopkins. We are following individuals who are experiencing GERD, Small Bowel Bacterial Overgrowth, Constipation, Fecal Incontinence and Gastroparesis to see if there is improvement in symptoms after a change in treatment is implemented. We are currently enrolling new participants into this multicenter study.
10. IVIG for active scleromyxedema study. The 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.
11. JBT-10 in Diffuse Cutaneous Systemic Sclerosis. The Johns Hopkins PI is Dr. Hummers (Corbus). This is a multicenter study of an investigational drug for diffuse scleroderma. Patients having an onset of early diffuse scleroderma less than six years ago are included. Enrollment is currently closed but participants are being followed.
12. Cytori Star Study in scleroderma. The Johns Hopkins PI is Dr. Hummers (Cytori Therapeutics). This study uses celution processed adipose derived regenerative cells to treat finger contractures in scleroderma patients. Enrollment is closed. Participants are being followed.
13. A double blind, randomized, placebo-controlled trial evaluating efficacy and safety of oral nintedanib treatment for at least 52 weeks in patients with systemic sclerosis associated interstitial lung disease (SSc-ILD). The Johns Hopkins PI is Dr. Hummers (Boehringer-Ingelheim). We are actively enrolling participants who have a disease duration of less than 5 years.