Clinical Trials and Observational Studies

Johns Hopkins Scleroderma Center Research Registry   
Our Center’s research program is focused on studying human disease in patients in the context of providing outstanding clinical care — investigating disease pathogenesis, risk stratification, screening and treatment approaches, outcome measure development, clinical trials, and impact on quality of life. This registry is a large data, blood sample and tissue repository that is the foundational resource used for all investigations conducted in the Center.  Patients now have the option to link information collected through personal health tracking devices (e.g. fitbit) to data collected through the registry.

Johns Hopkins Scleroderma Center Cutaneous Tissue Repository  
This is a skin tissue bank where patients with scleroderma volunteer to donate 3 small punch skin biopsies to be used in various scleroderma research studies. This repository enables studies investigating mechanisms of disease initiation and propagation, including identification of biological pathways that could serve as new treatment targets. Additional this repository supports studies to investigate biomarkers that may predict response to different therapeutic strategies. 

Collaborative, National Quality and Efficacy Registry for Tracking Disease Progression in Systemic Sclerosis (Scleroderma) Patients (CONQUER) 
This is a multicenter patient registry for patients with early scleroderma for which we are a participating site. The aim of this study is to collect long-term information about disease course in patients with early disease. 

The Scleroderma Patient-Centered Intervention Network (SPIN) 
This is a multicenter study where participants with scleroderma answer online questionnaires every 3 months in order to determine the most important problems experienced by scleroderma patients.  This information is used to develop self-help modules available to all patients online. Learn more: www.spinsclero.com

Utility of Intensive Cancer Screening Strategies in High Risk Scleroderma Patients 
This study is testing whether breast MRI and whole body PET/CT scans improve cancer detection in patients who have had scleroderma for less than 3 years and who are positive for an autoantibody associated with increased cancer risk.    

Evaluation of Novel Tumor Markers in Scleroderma and Myositis 
The primary objective of this study is to determine the added value of tumor-associated proteins, circulating tumor cells, and free tumor DNA in detecting subclinical malignancy in patients with new onset scleroderma or myositis, and whether the presence of these tumor markers varies by autoantibody type. 

Studying the interface between cancer and autoimmunity in scleroderma

The main goal of this study is to learn how cancer, the immune system, and the symptoms of scleroderma are related. To do this, we will study carefully collected medical records and biological samples (blood and other specimens) from three groups of scleroderma patients in the United States.

We will also test a blood-based screening method called SEEK (a type of “liquid biopsy”) to see whether it can detect cancer at a very early stage—before any physical signs or symptoms appear.

Early Detection of Clinically Significant Scleroderma-Associated ILD Using At-Home Digital Monitoring Assessments 
In this study, we seek to determine whether at home spirometry assessments at frequent intervals and activity monitors that assess functional status and heart rate can aid in early detection of clinically significant interstitial lung disease that would warrant therapy.  

A Double Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of PRA023 in Subjects with Systemic Sclerosis Associated with Interstitial Lung Disease (SSc-ILD): The ATHENA-SSc-ILD Study 
This is a multicenter clinical trial evaluating the effectiveness of an investigational drug in patients with diffuse scleroderma and ILD. 

A Phase 2/3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of belimumab administered subcutaneously in adults with systemic sclerosis associated interstitial lung disease (SSc-ILD) BLISSc-ILD
This is a multicenter clinical trial evaluating the effectiveness of an investigational drug in patients with diffuse scleroderma and ILD with a disease duration less than 7 years. 

Platform Clinical Trial for Conquering Scleroderma: A Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2b Platform Clinical Trial to Evaluate the Safety and Efficacy of Investigational Products in Participants with Interstitial Lung Disease Secondary to Systemic Sclerosis- CONQUEST
This is a multicenter clinical trial where two different investigational drugs will be studied in participants with limited and diffuse scleroderma who have ILD with a disease duration less than 5 years.

Serial Stress Echocardiography in Systemic Sclerosis Patients at Risk for Pulmonary Hypertension
This study examines the changes in PAH over time.

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 
This study is a clinical trial to determine whether Ifetroban, an investigational drug, softens skin and improves pulmonary arterial hypertension. 

Sildenafil for Early Pulmonary Vascular Disease in Scleroderma (SEPVADIS) 
This study is examining whether individuals with mildly elevated pulmonary pressures have an improvement in distance walked on the 6 minute walk test after 4 months of treatment with Sildenafil. 

A Multicenter, Randomized, Parallel-group, Double-blind, Two-arm, Phase III Study to Evaluate the Safety and Efficacy of Anifrolumab Compared with Placebo in Male and Female Participants 18 to 70 Years of Age Inclusive with Systemic Sclerosis (DAISY-AZ)
This is a multicenter clinical trial studying the effectiveness of an investigational drug in individuals with diffuse scleroderma with and without ILD or individuals with limited scleroderma with ILD with a disease duration less than 6 years.

Evaluation of MTX-474 in Participants with Diffuse Cutaneous Systemic Sclerosis (dcSSc)

This multi-center clinical trial is testing a new investigational drug for people with diffuse scleroderma who have recent, worsening skin tightening. To join, patients must have had their first scleroderma symptom within the past 10 years.

Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Systemic Sclerosis

This multi-center clinical trial is enrolling people whose scleroderma is causing active skin tightening and who have had the disease for less than 5 years.

A phase 1 study evaluating the safety and preliminary efficacy of ALLO-329, a dual anti-CD19 / anti-CD70 allogeneic CAR T cell product in autoimmune disease (resolution)

This multi-center clinical trial is for people with scleroderma whose first symptom began less than 7 years ago and who still have active disease despite taking immunosuppressant medication for at least 3 months.