Center for Genetic Disorders of Cognition and Behavior: Rett Syndrome Workshop Agenda

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Workshop: May 29-31, 2006
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CLINICAL TRIALS IN RETT SYNDROME
Workshop Agenda Note: All lectures 10-30 min. followed by a 20-30 min. period of discussion.

Day One: May 29, 2006, 1:30pm - 8:00pm
Session I:	Opening Lunch and Introductory Lectures Chair: Hugo W. Moser, M.D.
1:30pm - 2:00pm	Chair: Welcome:	Walter E. Kaufmann, M.D. Walter E. Kaufmann, M.D. Sarojini S. Budden, M.D. Daniel G. Glaze, M.D. Mary Lou Oster-Granite, Ph.D., NICHD Giovanna M. Spinella, M.D., ORD (message read by Dr. Oster-Granite) Kathy Hunter, IRSA
2:00pm - 2:30pm	Rett syndrome research: A video message. Bengt Hagberg, M.D., Ph.D. Hugo W. Moser, M.D.
2:30pm - 3:30pm	Goals of the conference: feasibility of multicenter clinical trials. Walter E. Kaufmann, M.D.
3:30pm - 4:30pm	Neuroscientific foundations of therapeutic strategies and outcome measures in Rett syndrome. Michael V. Johnston, M.D. Masaya Segawa, M.D.
4:30pm - 5:00pm	Coffee Break
Session II:	General issues about clinical trials in Rett syndrome. Chair: Alan K. Percy, M.D.
5:00pm - 6:00pm	Summary of clinical trials experience in Rett syndrome. Alan K. Percy, M.D.
6:00pm - 7:00pm	Clinical trial design and evaluation. James Korelitz, Ph.D.
7:00pm - 8:00pm	Dinner

Day Two: May 30, 2006, 8:00am - 9:00pm
Session III:	Target population: who should be treated? Chair: Alison Kerr, F.R.C.P.
8:00am - 8:45am	InterRett and the natural history of Rett syndrome. Helen Leonard, M.D. Alison Kerr, F.R.C.P.
8:45am - 9:45am	Genotype-phenotype correlations and clinical trials in Rett syndrome. John Christodoulou, F.R.A.C.P., Ph.D. N. Carolyn Schanen, M.D., Ph.D. Mark E.S. Bailey, Ph.D. Merce Pineda, M.D. Jeffrey L. Neul, M.D., Ph.D.
9:45am - 10:30am	Animal models and clinical trials in Rett syndrome. N. Carolyn Schanen, M.D., Ph.D. Jeffrey L. Neul, M.D., Ph.D.
10:30am - 11:00am	Coffee Break
11:00am - 12:00pm	Is early intervention possible? Alison Kerr, F.R.C.P. Yoshiko Nomura, M.D. Sarojini S. Budden, M.D.
12:00pm - 1:30pm	Lunch break
Session IV:	Therapeutic targets: what to use? Chair: Daniel G. Glaze, M.D.
1:30pm - 2:30pm	Pharmacological interventions I: glutamatergic cholinergic drugs. Sakkubai Naidu, M.D.
2:30pm - 3:30pm	Pharmacological interventions II: other drugs. Daniel G. Glaze, M.D.
3:30pm - 4:00pm	Tea Break
4:00pm - 4:45pm	Gene-related therapies. N. Carolyn Schanen, M.D., Ph.D. Alan K. Percy, M.D.
4:45pm - 6:00pm	Non-pharmacological interventions. Ingegerd Witt-Engerstrom, M.D., Ph.D. Alison Kerr, F.R.C.P. Sarojini S. Budden, M.D.
Session V:	Outcome targets I: what to measure? Chair: Sakkubai Naidu, M.D.
6:00pm - 7:00pm	Neurologic, behavioral, and imaging outcome measures. Sakkubai Naidu, M.D. Christa Einspieler, Ph.D. Walter E. Kaufmann, M.D.
7:00pm - 8:00pm	Physiologic outcome measures Daniel G. Glaze, M.D. Peter Julu, M.D.
8:00pm - 9:00pm	Dinner

Day Three: May 31, 2006, 7:00am - 2:00pm
7:00am - 8:00am	InterRett meeting (optional)
Session VI:	Outcome targets II: what to measure? Chair: Sarojini S. Budden, M.D.
8:00am - 8:45am	Other outcome measures. Kathleen J. Motil, M.D., Ph.D. Carmelo Cuffari, M.D. Sarojini S. Budden, M.D.
8:45am - 9:15am	Measuring Quality of Life. Daniel G. Glaze, M.D.
9:15am - 9:45am	Coffee Break
Session VII:	Roundtable discussions Chairs: Walter E. Kaufmann, M.D., Sarojini S. Budden, M.D., Daniel G. Glaze, M.D.
9:45am - 10:45am	Roundtable I: Are we ready for multicenter clinical trials? All participants
10:45am - 11:45am	Roundtable 2: Collaborative research needed for clinical trials in Rett syndrome. All participants
11:45am - 12:00pm	Concluding Remarks. Adjournment
12:00pm - 2:00pm	Lunch/Informal Discussion.