RARITAN, N.J., Oct. 27, 2016 – Janssen Research & Development, LLC announced today the launch of ACCELERATE, a first-of-its-kind, patient-driven natural history registry dedicated to empowering patients with Castleman Disease (CD) to contribute their own personal medical information to advance the knowledge of the disease, including its diagnosis and treatment. CD is a rare immune system disorder, and there is poor understanding of its prognosis and treatment. Designed and led by the Castleman Disease Collaborative Network (CDCN) and The University of Pennsylvania (Penn), the patient registry is the result of an innovative collaborative research agreement among the scientific research and medical communities and patient advocacy and pharmaceutical industry thought-leaders.
ACCELERATE (Accelerating Castleman Care with Electronic Longitudinal registry, E-Repository, And Treatment Effectiveness research) is designed to collect data on longitudinal changes in CD over time, providing knowledge that can help inform and potentially advance clinical understanding, treatment and patient outcomes. It is unlike traditional registries that typically require recruiting physicians and trial sites to enroll patients and enter patient data, which can be a lengthy and complicated process. For rare diseases like CD, collecting patient information is additionally challenging. Through ACCELERATE, CD patients in the U.S. and other countries outside of Europe will be able to enroll themselves and send their medical records to Penn for patient data to be entered into the registry. In Europe, where different regulations govern the use of patient information, ACCELERATE will work with 10 clinical sites to collect patient data.
“More research around the pathogenesis of CD is urgently needed,” said David Fajgenbaum, MD, MBA, MSc, an assistant professor of Medicine at the Perelman School of Medicine at Penn and associate director of patient impact at the Penn Orphan Disease Center. Dr. Fajgenbaum is also the co-founder and Executive Director of CDCN, and will serve as the principal investigator for ACCELERATE. Diagnosed with idiopathic Multicentric Castleman Disease (iMCD) in 2010 when he was a student at Penn’s Perelman School of Medicine, Dr. Fajgenbaum almost lost his battle with the disease. “As a physician-scientist and patient with this deadly disease, I believe a more collaborative, strategic, and focused approach is necessary to accelerating research. We must work together, because patients – like myself – are waiting.”
In 2014, Janssen received U.S. Food and Drug Administration (FDA), European Commission (EC), and Health Canada approval for SYLVANT® (siltuximab), the first and only approved treatment for MCD. The registry will help fulfill a European Medicines Agency (EMA) regulatory request for Janssen to collect additional patient information on this rare disease.
The collaboration to launch ACCELERATE reflects the promise to advance knowledge around rare diseases, thanks to contributions from the healthcare industry in partnership with the scientific and medical communities. “The launch of ACCELERATE underscores our commitment to support global research collaborations between industry, the medical community and patient advocacy groups to generate real-world patient outcomes data in disease areas where there is a small population with high unmet medical need,” said Craig Tendler, M.D., Vice President of Late-Stage Development and Global Medical Affairs for Oncology, Hematology and Supportive Care at Janssen Research & Development. “We believe this registry will help transform our collective understanding and approach to treating CD. We are honored to be a part of this groundbreaking initiative.”
ACCELERATE will be managed by Penn with additional scientific expertise and patient engagement support provided by the CDCN. The Registry is targeting enrollment of up to 1,000 patients within the first five years, using their clinical data and available tissue samples to create a globally shared, virtual biorepository that will facilitate collaboration among researchers and patients across the world to help advance CD research. As the leading patient advocacy group and research organization dedicated to helping patients with CD, the CDCN will house the web-based registry and ongoing updates about ACCELERATE on its website, available at www.CDCN.org/ACCELERATE. For more information about Castleman Disease, please visit www.CastlemansConnect.com or www.CDCN.org.
About Castleman Disease (CD)
Castleman Disease (CD) is a serious immune system disorder, encompassing a group of heterogenous lymphoproliferative disorders that share common histopathological lymph node changes.[i] The number of people diagnosed with CD is unknown, but it’s estimated that approximately 6,500 to 7,700 new cases are diagnosed in the U.S. each year.[ii] As with other rare diseases, it can be difficult to diagnose Castleman Disease, especially because the symptoms are often similar to those of other diseases and conditions. The exact cause of CD is also unknown, and treatments for the disease remain scarce.
There are two types of Castleman Disease: Multicentric and Unicentric. Multicentric Castleman Disease (MCD) involves multiple groups of enlarged lymph nodes with flu-like symptoms and vital organ dysfunction.[iii] Unicentric (or localized) Castleman Disease (UCD) affects only a single group of lymph nodes.
About SYLVANT® (siltuximab)
SYLVANT® is approved globally in 45 countries. It is an anti-interleukin-6 (IL-6) chimeric monoclonal antibody that binds to human IL-6. IL-6 is a multifunctional cytokine produced by various cells such as T cells, B cells, monocytes, fibroblasts and endothelial cells. Dysregulated overproduction of IL-6 from activated B cells in affected lymph nodes has been implicated in the pathogenesis of, or mechanism causing, MCD.[iv] Information about ongoing studies with siltuximab can be found at www.clinicaltrials.gov.
Indication
SYLVANT® (siltuximab) is indicated for the treatment of patients with Multicentric Castleman Disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative.
Limitation of Use. SYLVANT® was not studied in patients with MCD who are HIV positive or HHV-8 positive because SYLVANT® did not bind to virally produced IL-6 in a nonclinical study.
Important Safety Information
CONTRAINDICATIONS – Severe hypersensitivity reaction to siltuximab or any of the excipients in SYLVANT®.
Concurrent Active Severe Infections – Do not administer to patients with severe infections until the infection resolves. SYLVANT® may mask signs and symptoms of acute inflammation including suppression of fever and of acute phase reactants such as C-reactive protein (CRP). Monitor patients closely for infections. Institute prompt anti-infective therapy and do not administer further SYLVANT® until the infection resolves.
Vaccinations – Do not administer live vaccines to patients receiving SYLVANT® because interleukin-6 (IL-6) inhibition may interfere with the normal immune response to new antigens.
Infusion Related Reactions and Hypersensitivity – Stop the infusion if the patient develops signs of anaphylaxis. Discontinue further therapy.
Stop the infusion if the patient develops mild to moderate infusion reactions. If the reaction resolves, the infusion may be restarted at a lower infusion rate. Consider medicating with antihistamines, acetaminophen, and corticosteroids. Discontinue SYLVANT® if the patient does not tolerate the infusion following these interventions.
Administer SYLVANT® in a setting that provides resuscitation equipment, medication, and personnel trained to provide resuscitation.
Gastrointestinal (GI) Perforation – Use with caution in patients who may be at increased risk for GI perforation. Promptly evaluate patients presenting with symptoms that may be associated with or suggestive of GI perforation.
Adverse Reactions – The most common adverse reactions (>10% compared to placebo) in the MCD clinical trial were pruritus, increased weight, rash, hyperuricemia, and upper respiratory tract infection.
Drug Interactions - Cytochrome P450 (CYP450) Substrates – Upon initiation or discontinuation of SYLVANT®, in patients being treated with CYP450 substrates with narrow therapeutic index, perform therapeutic monitoring of effect (e.g., warfarin) or drug concentration (e.g., cyclosporine or theophylline) as needed and adjust dose. Exercise caution when SYLVANT® is co-administered with CYP3A4 substrate drugs where a decrease in effectiveness would be undesirable (e.g., oral contraceptives, lovastatin, atorvastatin).
Please click here to read full Prescribing Information for SYLVANT®.
About the Janssen Pharmaceutical Companies
At the Janssen Pharmaceutical Companies of Johnson & Johnson, we are working to create a world without disease. Transforming lives by finding new and better ways to prevent, intercept, treat and cure disease inspires us. We bring together the best minds and pursue the most promising science. We are Janssen. We collaborate with the world for the health of everyone in it. Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenUS and www.twitter.com/JanssenGlobal.
[i] Fajgenbaum, D., & Van Rhee, F. (2015, February 27). Treatment of Castleman Disease. The Hematologist, 12(2). Retrieved October 14, 2016, from http://www.hematology.org/Thehematologist/Ask/3738.aspx
[ii] Castleman Disease Collaborative Network. About Castleman Disease. Available from: http://www.cdcn.org/about-castleman-disease
[iii] Castleman Disease Collaborative Network. About Castleman Disease. Available from: http://www.cdcn.org/about-castleman-disease
[iv] El-Osta HE, Kurzrock R. Castleman’s disease: from basic mechanisms to molecular therapeutics. Oncologist. 2011;16(4):497-511.