Abstract

This is a Phase I, multicenter safety study of Factor VIII gene therapy, using a retroviral vector and B-domain deleted factor VIII gene in individuals with severe hemophilia A. Patients must have severe disease, be over age 25, and have no evidence of inhibitors, nor use antiretroviral therapy. Safety and preliminary efficacy parameters will include factor VIII:C level anti-VIIII level, bleeding symptoms and blood chemistries. There will be a pre-treatment 36-hour factor VIII PK study to establish baseline response to infusion. The Factor VIII gene therapy, hFVIII (V) will be given intravenously three consecutive days. A total of 12-20 subjects will be enrolled with graduated dose escalation based on efficacy at the previous lower dose. This study will provide long term follow-up of safety and duration of FVIII response through a Phase I-Extension study, with monthly visits for at least one year after treatment, and thereafter through a lifetime registry with annual visits.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000056-39
Application #
6408935
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
1978-12-01
Project End
2003-11-30
Budget Start
Budget End
Support Year
39
Fiscal Year
2000
Total Cost
Indirect Cost

  Institution

Name
University of Pittsburgh
Department
Type
DUNS #
053785812
City
Pittsburgh
State
PA
Country
United States
Zip Code
15213

  Publications

Geskin, Larisa J; Akilov, Oleg E; Kwon, Soonyou et al. (2018) Therapeutic reduction of cell-mediated immunosuppression in mycosis fungoides and Sézary syndrome. Cancer Immunol Immunother 67:423-434
Ong, Jue-Sheng; Hwang, Liang-Dar; Cuellar-Partida, Gabriel et al. (2018) Assessment of moderate coffee consumption and risk of epithelial ovarian cancer: a Mendelian randomization study. Int J Epidemiol 47:450-459
Block, Matthew S; Vierkant, Robert A; Rambau, Peter F et al. (2018) MyD88 and TLR4 Expression in Epithelial Ovarian Cancer. Mayo Clin Proc 93:307-320
Cai, HuaLin; Zhou, Xiang; Dougherty, George G et al. (2018) Pregnenolone-progesterone-allopregnanolone pathway as a potential therapeutic target in first-episode antipsychotic-naïve patients with schizophrenia. Psychoneuroendocrinology 90:43-51
Coen, Paul M; Carnero, Elvis A; Goodpaster, Bret H (2018) Exercise and Bariatric Surgery: An Effective Therapeutic Strategy. Exerc Sport Sci Rev 46:262-270
Lu, Yingchang; Beeghly-Fadiel, Alicia; Wu, Lang et al. (2018) A Transcriptome-Wide Association Study Among 97,898 Women to Identify Candidate Susceptibility Genes for Epithelial Ovarian Cancer Risk. Cancer Res 78:5419-5430
Chin, Brian; Murphy, Michael L M; Cohen, Sheldon (2018) Age moderates the association between social integration and diurnal cortisol measures. Psychoneuroendocrinology 90:102-109
Hayden, Kathleen M; Baker, Laura D; Bray, George et al. (2018) Long-term impact of intensive lifestyle intervention on cognitive function assessed with the National Institutes of Health Toolbox: The Look AHEAD study. Alzheimers Dement (Amst) 10:41-48
Liu, Gang; Mukherjee, Bhramar; Lee, Seunggeun et al. (2018) Robust Tests for Additive Gene-Environment Interaction in Case-Control Studies Using Gene-Environment Independence. Am J Epidemiol 187:366-377
Bogen, Debra L; Hanusa, Barbara H; Baker, Robyn et al. (2018) Randomized Clinical Trial of Standard- Versus High-Calorie Formula for Methadone-Exposed Infants: A Feasibility Study. Hosp Pediatr 8:7-14

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