Background: Veledimex (VDX)-regulatable interleukin-12 (IL-12) gene therapy in recurrent glioblastoma (rGBM) was reported to show tumor infiltration of CD8+ T cells, encouraging survival, but also up-regulation of immune checkpoint signaling, providing the rationale for a combination trial with immune checkpoint inhibition.
Methods: An open-label, multi-institutional, dose-escalation phase I trial in rGBM subjects (NCT03636477) accrued 21 subjects in 3 dose-escalating cohorts: (1) neoadjuvant then ongoing nivolumab (1mg/kg) and VDX (10 mg) (n = 3); (2) neoadjuvant then ongoing nivolumab (3 mg/kg) and VDX (10 mg) (n = 3); and (3) neoadjuvant then ongoing nivolumab (3 mg/kg) and VDX (20 mg) (n = 15). Nivolumab was administered 7 (±3) days before resection of the rGBM followed by peritumoral injection of IL-12 gene therapy.
Human interleukin-12 (hIL-12) is a cytokine with anticancer activity, but its systemic application is limited by toxic inflammatory responses. We assessed the safety and biological effects of an hIL-12 gene, transcriptionally regulated by an oral activator. A multicenter phase 1 dose-escalation trial (NCT02026271) treated 31 patients undergoing resection of recurrent high-grade glioma.
View Article and Find Full Text PDFDisabil Rehabil
August 2011
Purpose: We sought to examine the employment experiences in a population of adolescents and young adults with cystic fibrosis (CF).
Methods: We administered a cross-sectional survey to 68 individuals with CF, ages 16-25 years, assessing current employment experiences and perceptions of the impact of CF on these experiences.
Results: Forty-six percent of respondents reported working < 20 h per week.
Objective: The aim of this study was to develop the Transition Readiness Assessment Questionnaire (TRAQ), a measure of readiness for transition from pediatric to adult healthcare for youth with special health care needs (YSHCN).
Methods: We administered TRAQ to 192 YSHCN aged 16-26 years in three primary diagnostic categories, conducted factor analysis, and assessed differences in TRAQ scores by age, gender, race, and primary diagnosis type.
Results: Factor analysis identified two TRAQ domains with high internal consistency: Skills for Self-Management and Skills for Self-Advocacy.