Clinical Coordinating Center Social & Scientific Systems, Inc. Silver Spring, Maryland Project Coordinator: Susan Sepelak, MS, MSM

Program Management and Investigator Access to Resources Responsibilities of the Clinical Coordinating Center (CCC) Gene Therapy Resource Program (GTRP) Website Investigator Registration Process Request for Service Application (RSA) Process RSA Review Process

Responsibilities of the CCC With NHLBI develop program policies and bylaws Manage the RSA receipt, review, disposition, and tracking process Maintain the GTRP website and electronic forms Provide management support to the GTRP Steering Committee and the NHLBI Data Safety Monitoring Board (DSMB) Provide Regulatory assistance to investigators via the RSA process Provide Clinical Trial Funding assistance to investigators via the RSA process

Investigator Registration

Access on-line registration form from GTRP main web page Create a user ID and password Enter profile information (name; institution; address, etc) Provide information on funding resources (e.g. grants) Upload Sections A-C of the SF424 form (biographical sketch; publications) Submit electronic form for review and approval by the NHLBI Gene Therapy Group

Investigator Registration Approval Registration approval allows an investigator to request GTRP resources Approval is predicated on the investigator’s conduct of scientific research: –In gene therapy for heart, lung, or blood diseases consistent with the NHLBI Mission – With the investigator’s primary gene therapy research site in the United States

Request for Service Application

RSA Access Approved investigator logs in through the RSA web page using ID and password to select an RSA type

RSA Content Study Information –Abstract; rationale; study design; test system Regulatory Information –Approvals by IBC, IRB (if applicable); FDA communications (if applicable) Study Funding Vector Information –Promoter; transgene; vector map, sequence, etc. –Amount of vector required

RSA Submission Investigator completes the RSA form and saves it as a “preliminary” RSA The CCC will contact each investigator to review procedural requirements The specific Core providing the service will also contact the investigator to assist in completing and finalizing the RSA Once the RSA is submitted as a “final”: –The Core submits a Feasibility Assessment –The RSA is scheduled for review

RSA Review RSA TypeReview Bodies SRBSCGTG Preclinical Vector ProductionX Clinical Vector ProductionXXX Pharmacology/Toxicology TestingXXX Clinical Trial Funding AssistanceXXX Regulatory SupportX

RSA Submission Deadlines RSAs submissions are accepted on a continuous basis However, an RSA for: – Clinical Vector Production –Pharmacology/Toxicology Testing –Clinical Trial Funding Assistance must be submitted as a “final” before February 15 and September 15 in order to be assessed by the SRB and reviewed at the bi-annual SC meeting

THE CCC IS HERE TO HELP

VISIT THE GTRP EXHIBIT BOOTH 516 Additional Information about the Program Demonstration of the GTRP Web Pages and Registration Forms Candy