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 Studies
1. A multi-centre, randomized, double-blind (sponsor open), placebocontrolled, repeat-dose, proof of mechanism study to evaluate the safety, tolerability,pharmacokinetics, pharmacodynamics and explore efficacy of GSK2330811 in participants with diffuse cutaneous systemic sclerosis. The Johns Hopkins Principal Investigator (PI) is Dr. Hummers. We are currently enrolling in this study.
2. A Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Ifetroban in Patients With Diffuse Cutaneous Systemic Sclerosis or Systemic Sclerosis-Associated Pulmonary Arterial Hypertension. The Johns Hopkins PI is Dr. Hummers. We are actively enrolling in this study.
3. Scleroderma Lung Study III (SLS III): Combining the anti‐fibrotic effects of pirfenidone (PFD) with mycophenolate (MMF) for treating scleroderma-related interstitial lung disease. This is a multicenter study. The Johns Hopkins PI is Dr. Hummers. We are currently enrolling in this study.
4. 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. We are currently enrolling in these studies.
5. Collaborative, National Quality and Efficacy Registry for Tracking Disease Progression in Systemic Sclerosis (Scleroderma) Patients (CONQUER). This is a multicenter registry study to gather information about the natural history of scleroderma. The Johns Hopkins PI is Dr. Shah. We are currently enrolling in this study.
6. 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. We are currently enrolling in this study.
7. JBT-101 Phase 2 Study 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 are included. Enrollment is currently closed but participants are being followed.
8. 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). This study is complete but participants are being followed an an open label extension of this study where participants are receiving nintedanib.
9. A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2 Pilot Study Evaluating Intravenous Iloprost in Subjects With Symptomatic Raynaud’s Phenomenon Secondary to Systemic Sclerosis. The Johns Hopkins PI is Dr. Shah We are currently enrolling in this study.
10. The Study of Discordant Monozygotic Twins in Scleroderma, We are colaborting with Dr. Howard Chang (Stanford University) to study the DNA of identical twins where one twin has scleroderma and the other does not. The Johns Hopkins PI is Dr. Wigley. We are currently enrolling in this study.
11. Scleroderma and Gastrointestinal Disease. We are recruiting for several studies that are investigating the value of the Whole Gut Transit Study in identifying and following GI disease progression in Scleroderma. The Johns Hopkins Invetigator is Dr. McMahan. We are currently enrolling in these studies.
12. Transcutaneous Electroacupuncture (TEA) for Gastrointestinal Motility Disorders . We are collaborating with Dr. Jiande Chen to determine if TEA is a viable treatment for GI disorders. The Scleroderma Center Collaborator is Dr. McMahan. We are currently enrolling in this study.
13. Evaluation of buccal epithelial cells as markers of disease activity, progression, and response to therapy in systemic sclerosis, PI is Dr. McMahan. We are currently enrolling in this study.
14. Evaluating the pathogenesis of hypopigmentation in systemic sclerosis, PI is Dr. McMahan. We are currently enrolling in this study.
15. 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.
16. 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.
17. Myocophenolate mofetil (MMF), a commonly prescribed Skin Biopsy Specimens as Biomarkers of Scleroderma and the Response to Mycophenolate Mofetil (MMF or Cellcept). The PI is Dr. Zsuzsanna McMahan. 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 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.
