The objective of this study is to characterize the safety profile and determine the maximum tolerate dose (MTD) of BXQ-350, when given as a single agent at escalating doses, according to the investigational product (IP) related dose-limiting toxicities (DLTs) in patients with advanced solid tumors. Secondarily to assess the preliminary antitumor activity of BXQ-350 in solid tumors and recurrent high grade gliomas.
This is a first in man study of BXQ-350, a novel anti-neoplastic therapeutic agent composed of two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes. When both the components are assembled together forming stable SapC-DOPS nanovesicles (clinical formulation BXQ-350), the agent exhibits the propensity to enter the body and brain, target cells in the tumor mass, and induce cell death. The study is divided into 3 parts: 1. Dose Escalation Scheme Sequential cohorts of adult patients with advanced solid tumors and recurrent high-grade gliomas will be treated with escalating doses of BXQ-350 until the MTD is established, or in the absence of a MAD, the highest planned DL. 2. During Part 2, patients with advanced solid tumors and recurrent high-grade gliomas will be enrolled and administered BXQ-350 at the MTD determined in Part 1 or at the highest planned DL, if the MAD is not reached. 3. During Part 3, patients with either ependymoma, GI tumors , or advanced solid tumors other than HGG, will be enrolled and administered BXQ-350 at the 2.4 mg/kg dose level.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
86
BXQ-350 is a novel anti-neoplastic therapeutic agent configured from two components: Saposin C (SapC), an expressed (human) lysosomal protein, and the phospholipid dioleoylphosphatidyl-serine (DOPS), a phospholipid located on cell membranes. When both the components are assembled together stable SapC-DOPS nanovesicles are formed(clinical formulation BXQ-350).
University of Kentucky Markey Cancer Center
Lexington, Kentucky, United States
University of New Mexico Cancer Center
Albuquerque, New Mexico, United States
University of Cincinnati Barrett Center
Cincinnati, Ohio, United States
The Ohio State University Comprehensive Cancer Center
Columbus, Ohio, United States
Part 1-MTD
· To determine the maximum tolerate dose (MTD) of BXQ-350, when given as a single agent at escalating doses, according to the investigational product (IP) related dose-limiting toxicities (DLTs) in patients with advanced solid tumors
Time frame: 12 months
Part 2-RECIST
·To assess preliminary antitumor activity, defined as maximal radiological response during treatment using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1 (v1.1) criteria for solid tumors.
Time frame: 12 months
Part 2-RANO
To assess preliminary antitumor activity, defined as maximal radiological response during treatment Revised Assessment in Neuro-Oncology (RANO) criteria for recurrent high grade glioma (HGG), of BXQ-350 given as a single agent at the MTD, or highest planned dose level (DL), in the absence of a Maximum Administered Dose (MAD).
Time frame: 12 months
Part 3 - RECIST
To assess preliminary antitumor activity, defined as maximal radiological response during treatment using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1 (v1.1) criteria for solid tumors
Time frame: 12 months
Part 2- Area under Curve (AUC)
·To evaluate the AUC of BXQ-350
Time frame: 12 months
Part 2-Cmax
To evaluate the Cmax of BXQ-350
Time frame: 12 months
Part 2-half life
To evaluate the half-life (t1/2) of BXQ-350
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Time frame: 12 months
Part 2-CL
To evaluate the clearance (CL) of BXQ-350
Time frame: 12 months
Part 2-Progression-free survival (PFS-6)
To evaluate progression free survival at 6 months
Time frame: 12 months
Part 2-time to response
To evaluate time to response
Time frame: 12 months
Part 2-duration of response
To evaluate duration of response
Time frame: 12 months