CTEP Protocol No: T-99-0090 CC Protocol No: 01-C-0222 Amendment: J Revised: 4/21/09 IRB Approval Date: A Phase II Randomized, Cross-Over, Double-Blinded, Placebo-Controlled Trial of the Farnesyltransferase Inhibitor R115777 in Pediatric Patients with Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas Coordinating Center: Pediatric Oncology Branch, NCI Principal Investigator: Brigitte Widemann, M.D.* (POB, NCI) NIH Associate Investigators: Frank M. Balis, M.D. (POB, NCI) Beth Fox, M.D. (POB, NCI) Kathy Warren, M.D. (NOB, NCI) Andy Gillespie, R.N. (ND, CC) Eva Dombi, MD (POB, NCI)** Nalini Jayaprakash, (POB, NCI) Diane Cole, (POB, NCI) Seth Steinberg, Ph.D. (BDMB, DCS, NCI) Nicholas Patronas, M.D. (DR, CC)** Maria Tsokos, M.D., (Laboratory of Pathology, NCI)** Pamela Wolters, Ph.D. (HAMB, NCI) Staci Martin, Ph.D. (HAMB, NCI) Jeffrey Solomon, (NIH and Sensor Systems, Inc.) Non-NIH Associate Investigators: Wanda Salzer, M.D. ** Keesler Air Force Base, Biloxi, Mississippi) 81st MDOS/SGOC 301 Fisher Street, Room 1A132 Keesler AFB, MS 39534-2519 Phone: 228-377-6524 E-mail: [email protected] Bruce R. Korf, M.D., Ph.D. ** Chair, Department of Genetics Univeristy of Alabama at Birmingham 1530 3rd Ave. S. Birmingham, AL 35294 Phone: (205)-934-9411, Fax: (205)-934-9488 E-mail: [email protected] David H. Gutmann, M.D., Ph.D. ** Department of Neurology Washington University School of Medicine St. Louis Children’s Hospital Box 8111, 660 S. Euclid Avenue St. Louis, MO 63110 Phone: (314)-632-7149, Fax: (314)-362-9462 E-mail: [email protected] 1 4/21/2009 T-99-0090, 01-C-0222 Arie Perry, M.D. ** Division of Neuropathology Washington University School of Medicine Campus Box 8111, 660 S. Euclid Avenue St. Louis, MO 63110 Phone: (314) 362-9130, Fax: (314) 362-4096 E-mail: [email protected] M. Watson, M.D., Ph.D. ** Division of Laboratory Medicine/Box 8118 Alvin J. Siteman Cancer Center/Box 8100 Washington University School of Medicine 660 S. Euclid Avenue, St. Louis, MO 63110 Phone: (314)-454-7919, Fax: (314)-454-5525 E-mail: [email protected] Margaret Wallace, Ph.D. Associate Professor, Pediatric Genetics University of Florida Box 100296, 1600 SW Archer Road Gainesville, FL 32610-0296 Phone: (352) 392-3055; Fax: (352) 392-3051 E-mail: [email protected] David Muir, Ph.D. Associate Professor Pediatric Neurology & Neuroscience Box 100296, JHMHC University of Florida, College of Medicine Gainsville, FL 32610 Phone: (352)-392-0312; Fax: (352) 392-9520 E-mail: [email protected] Participating Institution: Johns Hopkins Oncology Center (M1011) Responsible Investigator: Robert J. Arceci, M.D., Ph.D. Bunting-Blaustein Cancer Research Building 1650 Orleans Street, 2M51 Baltimore, MD 21231 Phone: 410-502-7518, Fax: 410-955-8897 E-mail: [email protected] IRB contact: Monica Carlton 550 N. Broadway, Room 1121 Baltimore, MD 21287 Phone 410-955-0350, Fax: 410-614-1328 Pharmacy contact: Pauline Newman 600 N. Wolfe Street Baltimore, MD 21205 CMSC 209 Pediatric Pharmacy Phone: 410-955-5926, Fax: 410-955-0283 E-mail: [email protected] 2 4/21/2009 T-99-0090, 01-C-0222 Participating Institution: Children’s Hospital of Philadelphia, PA (M1257) Responsible Investigator: Jean Belasco, M.D. 34th and Civic Center Blvd, Philadelphia, PA 19104 Phone: (215) 590-2848, Fax: (215) 590-4183 E-mail: [email protected] IRB contact: Carol Sazama The Children’s Hospital of Philadelphia 1st floor Abramson Building 3517 Civic Center Boulevard Philadelphia, PA 19104-4399 Phone: 215-590-2830 E-mail: [email protected] Pharmacy contact: Betsy Bickert, Pharm D. 4th floor pharmacy, 4th floor main building 34th & Civic Center Boulevard Philadelphia, PA 19104 Phone: 215-590-3833 E-mail: [email protected] Participating Institution: SUNY Upstate Medical University, NY (M1303) Responsible Investigator: Ronald Dubowy, M.D. Department of Pediatrics – 5C 750 E Adams Street, Syracuse, NY 13210 Phone: (315) 464-5294; Fax: (315) 464-7238 E-mail: [email protected] IRB contact: Karen Bilynsky, RN, CCRA SUNY Upstate Medical University, NY Department of Pediatrics 5C 750 E Adams Street, Syracuse, NY 13210 Phone: 315-464-7601, Fax: 315-464-7515 E-mail: [email protected] Pharmacy contact: Andrea Allen SUNY Upstate Medical University, NY Department of Pharmacy 5C 750 E Adams Street, Syracuse, NY 13210 Phone: 315-464-4210, Fax: 315-464-4314 E-mail: [email protected] Participating Institution: Texas Children's Hospital, Houston, TX (M1060) Responsible Investigator: Susan, M. Blaney, M.D. Division of Hematology/Oncology, 6621 Fannin Street, Texas Children's Hospital, CC 1410.00, Houston, TX 77030 Phone: 832-822-1482; Fax: 832-825-4299 E-mail: [email protected] IRB contact: Stacey Berg M.D., Kay Motil, M.D. Baylor College of Medicine One Baylor Plaza S108 3 4/21/2009 T-99-0090, 01-C-0222 Houston, TX 77030 Phone: 713-798-6970 E-mail: [email protected], [email protected] Pharmacy contact: Renee A. Robinson RPh Texas Children’s Hospital Pharmacy department MC 2-2510 6621 Fannin Street Houston, TX 77030 Phone: 713-770-3899 E-mail [email protected] Participating Institution: The Children’s Hospital, Dana-Farber Cancer Institute, Boston, MA (M1034) Responsible Investigators: Mark W. Kieran, MD, PhD Director, Pediatric Medical Neuro-Oncology Dana-Farber Cancer Institute 44 Binney Street, Boston, MA, 02115 Phone: (617) 632-4907; Fax: (617) 632-4248 E-mail: [email protected] IRB contact: Jessica Jacobs, Protocol Administrator Protocol Administration Office Dana-Farber Cancer Institute 44 Binney Street Boston, MA 02115 Phone: 617-632-5737, Fax: 617-632-2686 E-mail: [email protected] Pharmacy contact: Peter Blanding Clinical Pharmacist-Pediatric Clinic Department of Pharmacy L-2 Dana-Farber Cancer Institute 44 Binney Street Boston, MA 02115 Phone 617-632-3785, Fax: 617-632-4410 E-mail: [email protected] Participating Institution: St. Louis Children’s Hospital, St. Louis, MO (M1123) Responsible Investigator: Allison King, M.D. Washington University School of Medicine St. Louis Children’s Hospital 660 S. Euclid Avenue Campus Box 8116 St. Louis, MO 63110 Phone: (314)-286-1170, Fax: (314)-454-2780 E-mail: [email protected] IRB contact: Kirsten Cady Washington University School of Medicine 660 S. Euclid Avenue 4 4/21/2009 T-99-0090, 01-C-0222 Campus Box 8056 St. Louis, MO 63110 Phone: 314-362-7773, Fax: 314-747-0232 E-mail: [email protected] Pharmacy contact: Melissa Heigham, RPh Investigational Pharmacist St. Louis Children’s Hospital One Children’s Place 8W25 St. Louis, MO 63110 Phone: 314-454-2361, fax: 314-454-2441 E-mail: mheigham@bjc Participating Institution: Children’s Hospital Los Angeles, CA (M1118) Responsible Investigator: Jonathan L. Finley, M.D., Ch.B. Childrens Hospital Los Angeles 4650 Sunset Blvd, Mail Stop 54 Los Angeles, CA 90027 Phone 323-906-8147, Fax: (323)-660-7128 E-mail: [email protected] IRB contact: Rhonda Crutcher 4650 Sunset Blvd, Mailstop 54 Los Angeles, CA 90027 Phone: 323-660-2450 ext. 5701, Fax: 323-671-1585 E-mail: [email protected] Pharmacy contact: John Pech, MS 4650 Sunset Blvd, Mailstop 44 Los Angeles, CA 90027 Phone: 323-660-2450 ext. 5989, Fax: 323-664-0326 E-mail: [email protected] Participating Institution: Cincinnati Children’s Hospital (FWA 00002988) Responsible Investigator: John Perentesis, M.D. Division, Hematology/Oncology ML 7015 Cincinnati Children’s Hospital Medical Center 3333 Burnet Ave. Cincinnati, Ohio 45229 Phone: 513-636-8241, Fax: 513-636-3459 E-mail: [email protected] IRB Contact: Irwin Light, M.D. Cincinnati Children’s Hospital Medical Center 229 Erkenbrecher Ave. ML 5020 Cincinnati, Ohio 45229 Phone: 513-636-8039, Fax: 513-636-3959 E-mail: [email protected] Pharmacy Contact: Denise Lagory, RPh Investigational Pharmacy Cincinnati Children’s Hospital Medical Center 3333 Burnet Ave ML 1011 5 4/21/2009 T-99-0090, 01-C-0222 Cincinnati, Ohio 45229 Phone: 513-636-3016, Fax: 513-636-2740 E-mail: [email protected] Participating Institution: Children’s Memorial Hospital, Chicago, IL (M1484) Responsible Investigator: Stewart Goldman, M.D. Children’s Memorial Hospital 2300 Children’s Plaza, Box # 30 Chicago, IL 60614 Phone: (773)-880-3270, Fax: (773)-880-3053 E-mail: [email protected] IRB contact: Gary Dennison, IRB Manager Joel Frader, M.D., IRB Chair Children’s Memorial Institute for Education and Research, Office of Research Administration Institutional Review Board 2300 Children’s Plaza, Box # 205 Chicago, IL 60614 Phone: 773-880-8116 Pharmacy contact: Susan Berg, R.Ph. (Hem/Onc Pharmacy) Marianne Chan, PharmD., Director Pharmacy Services Children’s Memorial Hospital 2300 Children’s Plaza, Box 74 Chicago, IL 60614 Phone: 773-880-4471 Participating Institution: Hospital for Sick Children in Toronto (T3859) Responsible Investigator: Douglas J. Hyder, M.D. Hospital for the Sick Children 555 University Ave. Toronto, ON M5G 1X8 Phone: 416-813-7758 Fax: 416-813-8024 E-mail: [email protected] IRB Contact: Melvin Freedman, M.D. Chair, Research Ethics Board Hospital for Sick Children 555 University Ave. Toronto, ON M5G 1X8 Phone: 416-813-6152 Fax: 416-813-5327 E-mail: [email protected] Pharmacy Contact: Darcy Nicksy Research Support Pharmacist 555 University Ave. Toronto, ON M5G 1X8 Phone: 416-813-7605 6 4/21/2009 T-99-0090, 01-C-0222 Fax: 416-813-7886 E-mail: [email protected] Participating Institution: University of Alabama at Birmingham (M1149) Responsible Investigator: Alyssa Reddy, MD 1600 7th Ave. South ACC 512 Birmingham, AL 35233 Phone: (205) 939-9285, Fax: (205) 975-6377 E-mail: [email protected] IRB Contact: Sheila Moore Director, UAB Institutional Review Board Administration Building, Room 470 701 20th Street, South Birmingham, AL 35294 Phone: (205) 934-3789 E-mail: [email protected] Pharmacy Contact: Kathleen (Keen) Blair Investigational Study Pharmacist 1600 7th Ave., South Birmingham, AL 35233 Phone: (205) 939-5918, Fax: (205) 939-9934 E-mail: [email protected] Participating Institution: Klinikum Nord, Hamburg, Germany (FWA 00003228) Responsible Investigator: Victor-F.