1:40 PM An Adverse Event in a Gene Transfer Clinical Trial for X-Linked Severe Combined Immunodeficiency Disease (SCID)
Opening Remarks
Theodore Friedmann, M.D., RAC Chair
Powerpoint slides
1:40 PM Case Presentation and Followup
Christof von Kalle, M.D., Cincinnati Children's Hospital Research Foundation ( Slides not available -see webcast or video)
2:00 PM Questions and Answers
2:10 PM Review of FDA Biological Response Modifiers Advisory Committee Meeting
Stephanie L. Simek, Ph.D., U.S. Food and Drug Administration
Powerpoint Slides
2:30 PM Review of October 29, 2002, RAC Planning Session
Stephen Rose, Ph.D., and/or Theodore Friedmann, M.D.
Powerpoint Slides
2:50 PM Adverse Events in Clinical Trials Using Retroviral Vectors
Alexander Rakowsky, M.D., Office of Biotechnology Activities, NIH
Powerpont Slides
Webcast
3:10 PM BREAK
Webcast
3:25 PM Monitoring Research Participants in Clinical Trials Using Retroviral Vectors
Theodore Friedmann, M.D., and Diane Wara, M.D., Moderator
Powerpoint Slides: Framing Questions
Powerpoint Slides: Monitoring for Insertional Oncogenesis and Clonality of Hemotopoiesis after Gene Therapy
- Fabio Candotti, M.D., National Human Genome Research Institute (NHGRI), NIH
- Dale Hammerschmidt, M.D., University of Minnesota
- Harry Malech, M.D., National Institute of Allergy and Infectious Diseases (NIAID), NIH
- David Sidransky, M.D., RAC Member
- Robert Simari, M.D., RAC Member
- Christof von Kalle, M.D., Cincinnati Children's Hospital Research Foundation
- Kenneth Weinberg, M.D., University of Southern California (USC) School of Medicine/Children's Hospital of Los Angeles
4:55 PM Public Comment
5:15 PM ADJOURNMENT
Thursday, December 5, 2002
Webcast for Thursday Morning
8:30 AM Call to Order and Opening Remarks
Theodore Friedmann, M.D., RAC Chair
8:35 AM An Adverse Event in a Gene Transfer Clinical Trial for X-Linked SCID
8:35 AM Informed Consents for Clinical Trials Using Retroviral Vectors
James Childress, Ph.D., and Madison Powers, J.D., D.Phil., Moderators
Discussion Points and Strawman Powerpont Slides
Fabio Candotti, M.D., NHGRI, NIH
Dale Hammerschmidt, M.D., University of Minnesota
Requirements for Ethical Research
Powerpoint Slides
Harry Malech, M.D., NIAID, NIH
Jennifer Puck, M.D., NHGRI, NIH
Christof von Kalle, M.D., Cincinnati Children's Hospital Research Foundation
Diane Wara, M.D., RAC Member
Research Participant Monitoring (not presented - see next meeting)
Kenneth Weinberg, M.D., USC School of Medicine/Children's Hospital of Los Angeles
Continued: Monitoring
for Insertional Oncogenesis and Clonality of Hemotopoiesis after Gene Therapy
Powerpoint Slides
10:00 AM BREAK
10:15 AM Informed Consents for Clinical Trials Using Retroviral Vectors (continued)
11:00 AM Summary of RAC Recommendations and Next Steps
Monitoring
Theodore Friedmann, M.D., and Diane Wara, M.D., Moderators
Informed Consent
James Childress, Ph.D., and Madison Powers, J.D., D.Phil., Moderators
12:00 NOON LUNCH
1:00 PM Discussion of Human Gene Transfer Protocol #0210-556 entitled:
A Phase I, Open-Label, Dose-Escalation Trial Evaluating the Safety and Immunogenicity of Sequential Administration of
Recombinant DNA and Adenovirus Expressing L523S Protein in Patients With Early-Stage Non-Small Cell Lung Cancer (NSCLC)
Webcast
PI: John J. Nemunaitis, M.D., US Oncology, Dallas, Texas
Powerpoint Slides: co-presented
by Dr. Martin A. (Mac) Cheever, M.D.
RAC Reviewers:
James Childress, Ph.D.
Neal DeLuca, Ph.D
David DeMets, Ph. D
Larry Johnson, M.D.
Tab 2280 Protocol
Tab 2281
OBA Summary
OBA Letter to PI on in-depth RAC Review and Public Discussion
Outcome of Initial Review by RAC Members
Reviews From Drs. Childress, DeLuca, DeMets, and L. Johnso
PI's / Sponsor's Response
2:45 PM BREAK
3:00 PM Discussion of Human Gene Transfer Protocol #0210-557 entitled:
A Double-Blind, Placebo-Controlled, Dose-Escalation Pilot Study To Assess the Safety and Effects of AMG0001 in
Patients With Ischemic Heart Disease (IHD) Not Amenable to Coronary Artery Bypass Graft (CABG) or Percutaneous Coronary Intervention (PCI)
Webcast
PIs:
Michael Simons, M.D., Dartmouth Medical School
Brian H. Annex, M.D., Duke University School of Medicine
Powerpoint Slides
RAC Reviewers:
Bernard Lo, M.D.
David Sidransky, M.D.
Robert Simari, M.D.
Tab 2282 Protocol
Tab 2283
OBA Summary
OBA Letter to PIs on in-depth RAC Review and Public Discussion
Outcome of Initial Review by RAC Members
Reviews From Drs. Lo, Sidransky, and Simari
PIs' / Sponsor's Response
4:30 PM Continued from a.m.: Monitoring
Theodore Friedmann, M.D., and Diane Wara, M.D., Moderators
Dr. Fabio Candotti, M.D., NHGRI, NIH
Powerpoint Slides: European Society for Immunodeficiency Meeting;
Weimar, October 16 - 20, 2002
Webcast
5:30 PM ADJOURNMENT
Friday, December 6, 2002
Webcast
8:30 AM Call to Order and Opening Remarks
Theodore Friedmann, M.D., RAC Chair
8:35 AM Report From NIH RAC Informed Consent Working Group
Nancy King, J.D., or James Childress, Ph.D., or Bernard Lo, M.D.
8:45 AM Discussion of Human Gene Transfer Protocol #0208-550 entitled:
A Phase I/II Study of an Antitumor Vaccination Using (1,3) Galactosyltransferase Expressing Allogeneic Tumor Cells in
Patients With Relapsed or Refractory Breast Cancer
PI: Roscoe F. Morton, M.D., Stoddard Cancer Research Institute, Iowa Methodist Medical Center
Powerpoint Slides
Discussion of Human Gene Transfer Protocol #0210-552 entitled:
A Phase I/II Study of an Antitumor Vaccination Using (1,3) Galactosyltransferase Expressing Allogeneic Tumor Cells in Patients With
Refractory or Recurrent NSCLC
PI: John C. Morris, M.D., National Cancer Institute, NIH
RAC Reviewers:
Linda Gooding, Ph.D.
Madison Powers, J.D., D.Phil.
Diane Wara, M.D.
Ad Hoc Reviewer: John Iacomini, Ph.D., Massachusetts General Hospital
(via teleconference)
Tab 2284 Protocol
Tab 2285
OBA Summary
OBA Letter to PI on in-depth RAC Review and Public Discussion
Outcome of Initial Review by RAC Members
Reviews From Drs. Gooding, Powers, Wara, and Iacomini
PI's / Sponsor's Response
10:45 AM ADJOURNMENT
