A total of 122 subjects were randomized into the study and investigated in the double-blind placebo-controlled setting to assess the efficacy and safety of G-CSF + BL-8040 as compared to G-CSF + placebo.
* Part 1: This lead-in period, designed to ascertain the dose of BL-8040, enrolled a total of 12 subjects to an open labeled treatment to assess the efficacy, safety, pharmacokinetic (PK) and pharmacodynamic (PD) parameters of treatment with G-CSF 10 µg/kg/day and BL-8040 1.25 mg/kg, per study protocol to goal collection of ≥ 6 × 10\^6 CD34+ cells/kg. * Part 2: Following the successful completion of Part 1, a total of 122 subjects were randomized into Part 2 of the study which employed a double-blind placebo-controlled setting to assess the efficacy and safety of G-CSF + BL-8040 as compared to G-CSF + placebo.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
180
Up to 2 subcutaneous (SC) injections of BL-8040 are anticipated during the study. Injections of G-CSF per standard of care
Up to 2 SC injections of Placebo are anticipated during the study. Injections of G-CSF per standard of care
UCLA Medical Center
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Miami
Miami, Florida, United States
Loyola University Medical Center
Chicago, Illinois, United States
University of Maryland
Baltimore, Maryland, United States
Mayo Clinic
Rochester, Minnesota, United States
The Washington University School of Medicine
St Louis, Missouri, United States
University of Cincinnati
Cincinnati, Ohio, United States
MD Anderson Cancer Center
Houston Texas, Texas, United States
Huntsman Cancer Institute in University of Utah
Salt Lake City, Utah, United States
...and 8 more locations
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg With up to 2 Apheresis Sessions
Percentage of subjects mobilizing ≥6 × 10\^6 CD34+ cells/kg with up to 2 apheresis sessions in preparation for autologous hematopoetic cell transplantation (auto-HCT) after treatment with G-CSF + single administration of BL-8040/placebo. Based on central laboratory data.
Time frame: From first day of study treatment (G-CSF) until day of second apheresis which was planned to occur on Day 6
Percentage of Subjects Mobilizing ≥2 × 10^6 CD34+ Cells/kg in 1 Apheresis Session
Percentage of subjects mobilizing ≥2 × 10\^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.
Time frame: From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5
Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg in 1 Apheresis Session
Percentage of subjects mobilizing ≥6 × 10\^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.
Time frame: From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5
Time to Neutrophil Engraftment, After Auto-HCT
Time to neutrophil engraftment after auto-HCT, where engraftment was defined as absolute neutrophil count (ANC) ≥0.5 × 10\^9/L for 3 days or ≥1.0 × 10\^9/L for 1 day following the conditioning regimen associated nadir.
Time frame: End of engraftment period, which was defined as 29 days post transplantation
Time to Platelet Engraftment, After Auto-HCT
Time to platelet engraftment, after auto-HCT, where engraftment was defined as the first of 3 consecutive measurements of platelet count ≥20 × 10\^9/L without platelet transfusion support for 7 days following the conditioning regimen associated nadir.
Time frame: End of engraftment period, which was defined as 29 days post transplantation
Subjects With Graft Durability at 100 Days Post Transplant/ Early Termination
Subjects achieving graft durability were defined as meeting the following 2 criteria: * Platelet count ≥50 × 10\^9/L without transfusion for at least 2 weeks. * Hemoglobin level ≥10 g/dL with no erythropoietin support or transfusions for at least 1 month. This analysis was performed in part 2 of the study only.
Time frame: Day 100 Post-Transplantation (± 7 days)
Graft Durability at 6 Months Post Transplantation
Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 6 months post transplantation
Time frame: 6 Months Post Transplantation
Graft Durability at 9 Months Post Transplantation
Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 9 months post transplantation
Time frame: 9 Months Post Transplantation
Graft Durability at 12 Months Post Transplantation
Comparability of the graft durability between the BL-8040 + G-CSF arm and the placebo + G-CSF arm at 12 months post transplantation
Time frame: 12 Month Post Transplantation
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