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Rare Diseases Clinical Research Network Data Management and Coordinating Center (RDCRN DMCC) Jeffrey Krischer, PhD LDN Investigator Meeting at WORLDSymposium 2017 February 17, 2017 The Rare Diseases Clinical Research Network (RDCRN) is coordinated by the Office of Rare Diseases Research (ORDR), 1 NCATS. Funding and programmatic support is provided by ORDR in collaboration with participating NIH Institutes. RDCRN Members Overview Data as of January 16, 2017 271 clinical centers (+4 since 2/21/16) 144 patient advocacy groups 3,393 consortium members (+132 since 2/21/16) 24,781 study participants currently enrolled (+3,522 since 2/21/16) 2 Active RDCRN Studies Data as of January 26, 2017 Distribution of Active RDCRN Studies 14% Number of Active RDCRN Studies by Consortium 16 86% Other Studies LDN Studies Number of Active Studies 14 12 10 8 6 4 2 0 Consortium 3 RDCRN Protocol Activation Data as of December 31, 2016 0.7 yrs N=99 0.5 yrs N=53 1.1 yrs N=38 Median time to activation from initial protocol review N = number of activated protocols 4 LDN Protocol Activation Data as of December 31, 2016 0.6 yrs N=125 0.7 yrs N=26 Median time to activation from initial protocol review N = number of activated protocols RDCRN 2nd and 3rd grant cycle excluding LDN activated protocols. Note: LDN values represent RDN2 and RDN3, since LDN joined the network. 5 LDN Accrual/Compliance by Protocol Data as of January 21, 2017 Lysosomal Disease Network Active Target Enrollment 15 Enrolled to Date 9 Protocol Compliance (%) 100 Active 58 9 100 Active 60 6 100 Active 60 22 99.35 70 39 98.01 16 8 95.68 150 10 30 141 5 6 95.54 82.14 77.78 16 4 77.46 Protocol Title Status 6711 Expanded Screening for the Fabry Trait Podocyturia, a Non-Invasive Predictor of Renal Dysfunction in Fabry Nephropathy Determinants of Renal Structural Responses to Enzyme Replacement Therapy in Fabry Disease Study and Podocytes in Fabry Renal Disease Natural History and Structural Functional Relationships in Fabry Renal Disease Carotid Structure and Function in MPS Syndromes: A Multicenter Study of Lysosomal Disease Network 6725 6727 6702 6703 6704 6729 Closed to Accrual Closed to A Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I Accrual Longitudinal Studies of Brain Structure and Function in MPS Disorders Active The Natural History of Mucolipidosis Type IV Active Synergistic Enteral Regimen for Treatment of the Gangliosidoses (Syner-G) Active 6728 An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I 6720 6714 Active 6 LDN Accrual/Compliance by Protocol Data as of February 9, 2017 Lysosomal Disease Network Protocol 6705 6722 6713 67091 67161 67262 Title Longitudinal Study of Bone and Endocrine Disease in Children with MPS I, II, and VI Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications A Natural History Study of the Gangliosidoses Determination of Cross-Reactive Immunological Material (CRIM) status and longitudinal follow-up Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis The Hunter James Kelly Research Institutes Clinical Database of Children with Krabbe Disease 1Compliance 2Compliance Status Target Enrolled Protocol Enrollment to Date Compliance (%) Active 55 55 72.28 Active 50 13 70.50 Active 52 34 43.64 Active 300 155 - Active 75 41 - Active 60 0 - information will become available pending data entry information will become available pending participant accrual 7 Collaboration with Industry Protocol LDN6703 LDN6705 LDN6707 LDN6708 LDN6709 Pharmaceutical Company Genzyme Corporation, Shire HGT Shire HGT Shire HGT Genzyme Corporation Genzyme Corporation LDN6711 Amicus Therapeutics, Shire HGT, Genzyme Corporation LDN6714 BioMarin Pharmaceutical, Inc. LDN6722 Pfizer Inc., Shire HGT Type of Support Supplemental funding Supplemental funding Supplemental funding Supplemental funding Funding for processing of whole blood sample, skin fibroblasts and mutation analysis Supplemental funding Supplemental funding (vials of Aldurazyme from commercial source) Supplemental funding 8 LDN Data Submitted to dbGaP Data submitted to the federal repository: • • • • • • • LDN6702: Natural History and Structural Functional Relationships in Fabry Renal Disease LDN6703: Longitudinal Studies of Brain Structure and Function in MPS Disorders LDN6704: The Natural History of Mucolipidosis Type IV LDN6705: Longitudinal Study of Bone and Endocrine Disease in Children with MPS I, II, and VI LDN6707: Characterizing the Neurobehavioral Phenotype(s) in MPS III LDN6709: Determination of Cross-Reactive Immunological Material (CRIM) status and longitudinal follow-up of individuals with Pompe disease LDN6716: Genotype-Phenotype Correlations of Late Infantile Neuronal Ceroid Lipofuscinosis LDN6719: Immune Response to Intrathecal Enzyme Therapy in Mucopolysaccharidosis 1 Patients 9 ClinicalTrials.gov Update Data as of January 21, 2017 All RDCRN studies should be registered • Study Chair is responsible for registering study and uploading results. LDN is currently 100% compliant with study registration. • RDCRN report tracks registration and result status for each protocol o GEN.I.14.ClinicalTrials.gov Protocol Status Report can be found on the Members’ Website by navigating to the ‘Network Reports’ folder • All NIH funded clinical trials (in whole or in part) should have results posted to CT.gov within 1 year of completion date • RDCRN CT.gov training available on Media Center https://www.federalregister.gov/documents/2016/09/21/2016-22379/nih-policy-on-the-dissemination-of-nih-fundedclinical-trial-information 10 Electronic Regulatory Binder The Electronic Regulatory Binder (E-Reg Binder) is an online interface for receiving and tracking regulatory documents • Benefits of E-Reg Binder o Centralized repository for all study related documents o Increase efficiency by streamlining document storage and auditing o Allows for the review of all regulatory documents remotely, thereby reducing time spent on-site during audit visits o Reduce costs and undue administrative burden at the local sites 11 Single IRB NIH Requirement RDN III protocols (current grant cycle): • Plan for single IRB implementation if an active study will continue into next grant cycle • Multi-center studies that are listed in current grant application, but are not yet activated, are not required to use single IRB until grant renewal in 2018 In the 2018 competitive grant renewal submission, consortium are expected to include a plan for implementation of single IRB model If preferred, single IRB model can be implemented prior to the submission of the 2018 application http://osp.od.nih.gov/sites/default/files/sIRB_Extramural_FAQs.pdf 12 OneIRB Coordinating Center The OneIRB CC interacts directly with the single IRB of Record on consortium’s behalf to: • Submit initial IRB applications, amendments, continuing reviews, and reportable events • Respond to IRB queries • Distribute IRB approval letters and stamped consent forms to sites 13 LDN Sites and OneIRB LDN Sites Utilizing OneIRB Children's Hospital of Pittsburgh Cincinnati Children's Hospital Medical Center Mayo Clinic - Rochester Oregon Health & Science University University of California, Los Angeles University of California, San Francisco University of Chicago University of Miami University of Minnesota University of Rochester University of Utah LDN Sites with Plans to Implement OneIRB Baylor College of Medicine Cedars-Sinai Medical Center Duke University Emory University 14 RDCRN Contact Registry Data as of January 16, 2017 Goals: To inform registrants about RDCRN studies available; To disseminate information about RDCRN activities Overall (All consortia): • 19,975 total registrations • 109 countries • 177 diseases represented LDN Contact Registry: • 70 total registrations • 11 countries • 24 diseases represented 15 Joining the RDCRN LDN Contact Registry “How Did You Hear About the RDCRN LDN Contact Registry?” 3% 4% 3% Internet Search 6% Medical Professional Support Group or Foundation 13% Other 56% 15% Missing Publication Word of Mouth http://www.rarediseasesnetwork.org/LDN/registry/index.htm 16 RDCRN LDN Contact Registry Enrollment Opportunities for increasing LDN Contact Registry Enrollment: • Addition of link to the RDCRN Contact Registry o LDN public website o PAG website(s) o Social media • Distribution of Contact Registry Interest Form o In clinic/at study visits o At Patient Advocacy Group (PAG) meetings • Auto-enrollment of longitudinal study participants into contact registry • Use google analytics to determine demand for public facing registry http://www.rarediseasesnetwork.org/documents/LDNContactRegistryForm.pdf 17 Recent Contact Registry Enhancements o Mobile friendly webpages o Updated enrollment form content Ability to enroll unaffected family members (for the 16 consortia who opted in to this feature: UCDC, RLD, MCC, BVMC, RKSC, INC, LDN, NEPTUNE, PIDTC, STAIR, PC, NAMDC, BBD, CEGIR, DSC, CReATe) Ability to enroll multiple affected family members at the same time (ex. one parent enrolling multiple affected children) Ability to select diseases across consortia o Option for registrants to create a login so they can update their enrollment information in the future and access a customized dashboard which includes: Area where participants can complete surveys, view completed surveys, and update information Area to echo information to participants on the number of participants enrolled, gender, age, disease, location, etc. (Participant Summary) Network and consortium announcements Consortia and PAG social media links Ability for participants to easily send a template Contact Registry invitation email to others 18 Recent Contact Registry Enhancements Utilization Update: o 908 new registrants have joined the Contact Registry since the enhancements went live 750 of the new registrants (83%) chose to set-up a username and password 43 of the new registrants have enrolled multiple patients (ex. registrant is a patient and has a child who is a patient, registrant has two children who are both patients) o 98 registrants who had joined the Contact Registry prior to the enhancements promotion have since returned to update their username and password and login to the dashboard Note: The enhancements have not been advertised to registrants. These are people that returned to the Contact Registry login page on their own and updated their information. o 52 emails have been sent by registrants to invite others to join the Contact Registry through the “More Ways to Help” tool on the dashboard 19 RDCRN Contact Registry Enhancements Enrollment Member Homepage Participant Summary (Statistics) 20 RDCRN Tools: Patient Portal • Participants and parents can complete forms at home or online in clinic. • Login information is the Participant ID and Local ID. • If interested, this could be implemented in LDN protocols in the future. 21 RDCRN Enhancements Enhancements: • • • • • • • • • Data Sharing Policy template development Remote and risk-based auditing Electronic Site Delegation Log (E-SDL) OneIRB Provision and utilization of third party applications, such as REDCap Interactive study data (accessible by participants) Cross-consortia/cross-study collaboration Outreach and awareness via Social Media Patient Advocacy Groups (PAGs) engagement 22 Thank you 23