WHAT IS THE CONQUER REGISTRY AND WHY IS IT NEEDED?
Scleroderma can manifest in many different ways. Some patients have fibrotic skin disease while others do not. Some patients have lung disease, heart disease, or gastrointestinal disease while other patients have many affected organs. Why is this and why is it so difficult for physicians to predict an individual patient’s disease course? Similarly, scleroderma patients need and are treated with many different therapies that span from simply monitoring the disease coupled with symptomatic therapy (e.g. H2 blockers and PPIs for heartburn/reflux) all the way to aggressive therapy aimed at modifying their underlying disease, such as chemotherapeutic immune system resetting with autologous stem cell rescue. What is the right therapy for any one patient and what is the range of outcomes that patient might expect? Unfortunately, the answers to critical questions such as these are not fully understood and comprehensive data does not yet exist to allow researchers to evaluate them.
The path to answering these questions requires tracking and collecting data on the health status, disease complications, treatments and outcomes of many patients over many years. The Scleroderma Research Foundation began developing the CONQUER Registry in 2013 with the goal of defining the epidemiology of scleroderma in the current era by enrolling a large group of patients and building a powerful database to track them. Data will be collected over the course of the normal clinic visits – ultimately for thousands of patients over many years. The data generated through the CONQUER Registry will allow researchers to refine disease subsets (going beyond “diffuse” and “limited”) and track patient outcomes for each subset with the aim of enabling more precise and tailored care for individual patients.
BACKGROUND AND HISTORY
Due to the rarity of scleroderma, a broad consortium of scleroderma centers is required to enroll a sufficiently large patient population and assemble a sufficiently comprehensive data set to enable deeper insights into the disease. International scleroderma groups have launched large observational studies in Europe, Canada, and Australia; however, these do not necessarily reflect the U.S. scleroderma patient population and, in any case, have some methodologic flaws that compromise their utility.
In 2011, the Prospective Registry of Early Systemic Sclerosis (PRESS) registry was developed in the U.S. in response to the FDA’s and the NIH’s calls for attention to Therapeutics for Rare and Neglected Diseases and developing tissue banks linked to clinical outcomes. The PRESS investigators were approached by the Scleroderma Research Foundation (“SRF”) to build upon their efforts. The overarching goal of the partnership of the SRF and the participating academic Scleroderma Centers is to amass the broadest possible cohort of early-stage scleroderma patients. This highly collaborative effort, initially among 12 of the largest scleroderma centers in the U.S., has been named CONQUER (an acronym for COllaborative, National QUality and Efficacy Registry for Tracking Disease Progression in Systemic Sclerosis (scleroderma) Patients).
The CONQUER Registry will enable researchers to:
- Determine whether certain disease features are associated with or predict clinical and patient-reported outcomes (short-term and long-term).
- Identify which patients require early and aggressive intervention and which patients are better suited for “watchful waiting
- Evaluate the response in the real world (as opposed to the controlled and constrained setting of a clinical trial) to therapeutic agents and to combinations of therapeutic agents.
- Drive more personalized and effective therapy for patients
- Develop insights into drug toxicities that are unique to scleroderma patients (e.g. underlying heart disease due to pulmonary vascular disease)
- Understanding patient satisfaction/issues with the current Standard of Care
- Collect biosamples for future analyses (e.g. genetic factors contributing to disease)
- Establish and support a collaborative network for U.S. scleroderma investigators
- Support the critical infrastructure for future scleroderma studies, including trials for novel therapeutics
Participation in the CONQUER Registry enables patients to:
- Make a critical contribution to better therapy for all patients with minimal personal impact. (This is an observational trial and blood is the only biosample that must be collected)
- Be positioned on the cutting edge of disease diagnostics
- Supported in the tracking of their disease with precision
WHO IS ELIGIBLE TO PARTICIPATE IN THE CONQUER REGISTRY PROJECT?
Eligible participants will be identified by on-site staff at participating institutions. Inclusion criteria are:
- Patients 18 years of age and older, inclusive
- Patients who meet the 2013 ACR/EULAR classification criteria for systemic sclerosis;
- Patients who are less than 5 years from onset of first non- Raynaud’s Phenomenon symptom attributed to systemic sclerosis
Clinical data collected for CONQUER will be entered into a National Institute of Health (NIH) sponsored RedCap database, which is a secure web-based application designed exclusively to support data capture for research studies.
The Data Coordinating Center (DCC) at the University of Utah School of Medicine will provides data coordination and management services for the CONQUER Registry as it does for a variety of national research networks.
The Biorepository Center at the University of Texas Health Science Center at Houston will provide processing, storage and management services for the biosamples of the CONQUER Registry as it does for a variety of national research networks.
HOW THE CONQUER REGISTRY WORKS
CAN PATIENTS CHOOSE TO WITHDRAW FROM THE CONQUER STUDY IN THE FUTURE AFTER BEING SELECTED TO PARTICIPATE?
Study participants will be allowed to withdraw from the registry at any time. This will not influence their routine care. Data already collected will remain in the database and biosamples already collected will remain in the biorepository.
HOW CAN I LEARN MORE ABOUT THE CONQUER REGISTRY PROJECT?
WHICH INSTITUTIONS ARE PARTICIPATING IN THE CONQUER REGISTRY PROJECT?
The 13 institutions listed below are the sites participating in the CONQUER REGISTRY Project.
We do anticipate that other expert sites will be added over time and are in the process of evaluating such additions now.
|Institution||Principal Investigator||Contact Information|
|Columbia University||Elana Bernstein, MD, MScfirstname.lastname@example.org|
|George Washington University||Victoria Shanmugam, MD||202-741-2488|
|Georgetown University||Virginia Steen, MD||Smb228@georgetown.edu|
|Hospital for Special Surgery||Jessica Gordon, MDemail@example.com|
|Johns Hopkins University||Ami Shah, MD
Laura Hummers, MD
|Mass General Hospital||Flavia Castelino, MDfirstname.lastname@example.org|
|Medical University of South Carolina||Faye Hant, DO, MSCR||843-792-1991|
|Northwestern University||Chase Correia, MDemail@example.com|
|Stanford University||Lori Chung, MD, MS||Mstark2@stanford.edu|
|University of Michigan||Dinesh Khanna, MDfirstname.lastname@example.org|
|University of Pennsylvania||Nora Sandorfi, MD||215-614-4430|
|The University of Texas Health Science Center at Houston||Shervin Assassi, MD||713-486-3100|
|University of Utah||Tracy Frech, MD||801-585-6468|
WHAT IS THE SCLERODERMA RESEARCH FOUNDATION?
The Scleroderma Research Foundation (“SRF”) is a non-profit, 501(c) (3) organization based in San Francisco. The Scleroderma Research Foundation (SRF) is the nation’s leading nonprofit investor in medical research and its mission is to fund and facilitate the most promising, highest quality research aimed at improved therapies, and ultimately, a cure for scleroderma. In addition to its basic and translational research program, the SRF funds and supports various Scleroderma Centers of Excellence. The SRF has achieved the highest possible rating from Charity Navigator, America’s premier charity evaluator, for sound fiscal management and commitment to accountability and transparency.
WHO ARE THE SPONSORS/FUNDERS OF THE CONQUER REGISTRY PROJECT?
The Scleroderma Research Foundation (“SRF”) is a non-profit, 501(c) (3) is the primary sponsor of the CONQUER Registry and is responsible for the coordination and distribution of support for the participating institutions.
PRIMARY AND COORDINATING SPONSOR
The SRF has entered into strategic partnerships with several other sponsors that are providing economic and other elements of support to ensure the success of the CONQUER Registry. We gratefully acknowledge the following external sponsors and partners of the CONQUER Registry: