A Phase III, Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and Granulocyte Colony Stimulating Factor (G-CSF) as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous Transplantation in Subjects With Multiple Myeloma (MM)

NCT03246529 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 1 other identifier: BL-8040.SCM.301
• Study Type: interventional
• Target: 180
• Locations: 5 countries
• Sites: 18

Brief Summary

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.

Conditions

Multiple Myeloma

Outcome Measures

Primary Outcomes (1)

• 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.

  From first day of study treatment (G-CSF) until day of second apheresis which was planned to occur on Day 6

Secondary Outcomes (8)

• Percentage of Subjects Mobilizing ≥2 × 10^6 CD34+ Cells/kg in 1 Apheresis Session

  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

  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

  End of engraftment period, which was defined as 29 days post transplantation

• Time to Platelet Engraftment, After Auto-HCT

  End of engraftment period, which was defined as 29 days post transplantation

• Subjects With Graft Durability at 100 Days Post Transplant/ Early Termination

  Day 100 Post-Transplantation (± 7 days)

Other Outcomes (3)

• Overall Survival Until September 2028

  End of Study

• Relapse Free Survival Until September 2028

  End Of Study

• Annualized Relapse Rate Until September 2028

  End of Study

Study Arms (2)

BL-8040 1.25 mg/kg + G-CSF

EXPERIMENTAL

Double-blind placebo-controlled setting designed to assess the safety, tolerability and efficacy of G-CSF + BL-8040 as compared to G-CSF + Placebo, for stem cell mobilization in MM.

Drug: BL-8040 1.25 mg/kg + G-CSF

Placebo + G-CSF

ACTIVE COMPARATOR

Double-blind placebo-controlled setting designed to assess the safety, tolerability and efficacy of G-CSF + BL-8040 as compared to G-CSF + Placebo, for stem cell mobilization in MM.

Drug: Placebo +G-CSF

Interventions

BL-8040 1.25 mg/kg + G-CSF DRUG

Up to 2 subcutaneous (SC) injections of BL-8040 are anticipated during the study. Injections of G-CSF per standard of care

BL-8040 1.25 mg/kg + G-CSF

Placebo +G-CSF DRUG

Up to 2 SC injections of Placebo are anticipated during the study. Injections of G-CSF per standard of care

Placebo + G-CSF

Eligibility Criteria

• Age: 18 Years - 78 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically confirmed Multiple Myeloma prior to enrolment and randomization.
• At least 1 week (7 days) from last induction cycle of combination/multi-agent cyto-reductive chemotherapy (e.g., KRD \[carfilzomib, lenalidomide, dexamethasone\] or VRD (e.g., bortezomib, lenalidomide, dexamethasone) or last single agent chemotherapy (e.g., lenalidomide, pomalidomide, bortezomib, dexamethasone, etc.) prior to the first dose of G-CSF for mobilization.
• Eligible for autologous hematopoietic stem cell transplantation according to the Investigator's discretion.
• The subjects should be in first or second CR (including CR and SCR) or PR (including PR and VGPR).
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
• Adequate organ function at screening as defined as below:
• Hematology:
• White blood cell counts more than 2.5 x 10\^9/L
• Absolute neutrophil count more than 1.5 x 10\^9/L
• Platelet count more than 100 x10\^9/L Renal Function:
• Glomerular Filtration Rate (GFR) value of ≥15 mL/min/1.732 calculated by Modification of Diet in Renal Disease (MDRD) equation
• Hepatic function:
• Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤ 2.5 x ULN
• Total Bilirubin ≤ 2.0 x Upper Limit Normal (ULN) unless the subject has Gilbert disease
• Coagulation test:

You may not qualify if:

• Previous history of autologous or allogeneic-Hematopoietic Cell Transplantation (HCT).
• Failed previous Hematopoietic Stem Cell (HSC) collections or collection attempts.
• Taken any of the listed below concomitant medications, growth factors or stimulating agents within the designated washout period:
• Dexamethasone: 7 days;
• Thalidomide: 7 days;
• Lenalidomide: 7 days;
• Pomalidomide: 7 days;
• Bortezomib: 7 days;
• Carfilzomib: 7 days;
• G-CSF: 14 days;
• Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) or Neulasta®: 21 days;
• Erythropoietin or erythrocyte stimulating agents: 30 days;
• Eltrombopag, romiplostim or platelet stimulating agents: 30 days;
• Carmustine (BCNU): 42 days/6 weeks;
• Daratumumab: 28 days;

Sponsors & Collaborators

• BioLineRx, Ltd.: lead

Study Sites (18)

UCLA Medical Center

Los Angeles, California, 167817, United States

Location

University of Florida

Gainesville, Florida, 100278, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Loyola University Medical Center

Chicago, Illinois, 60611, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Mayo Clinic

Rochester, Minnesota, 55902, United States

Location

The Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45221, United States

Location

MD Anderson Cancer Center

Houston Texas, Texas, 77030, United States

Location

Huntsman Cancer Institute in University of Utah

Salt Lake City, Utah, 84112, United States

Location

University of Koln

Cologne, Koln, 50923, Germany

Location

Central Hospital of Southern Pest National Institute of Hematology and Infectious Diseases

Budapest, 1097, Hungary

Location

University of Debrecen

Debrecen, 4032, Hungary

Location

Div. Clinicizzata di Ematologia - Policlinico Vittorio Emanuele

Catania, 95124, Italy

Location

Presidio Ospedaliero Morelli Viale Europa

Reggio Calabria, 89133, Italy

Location

Hospital de La Santa Creu I Sant Pau

Barcelona, 08041, Spain

Location

Hospital University Ramon y Cajal

Madrid, 135250, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Related Publications (2)

• Crees ZD, Rettig MP, Jayasinghe RG, Stockerl-Goldstein K, Larson SM, Arpad I, Milone GA, Martino M, Stiff P, Sborov D, Pereira D, Micallef I, Moreno-Jimenez G, Mikala G, Coronel MLP, Holtick U, Hiemenz J, Qazilbash MH, Hardy N, Latif T, Garcia-Cadenas I, Vainstein-Haras A, Sorani E, Gliko-Kabir I, Goldstein I, Ickowicz D, Shemesh-Darvish L, Kadosh S, Gao F, Schroeder MA, Vij R, DiPersio JF. Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial. Nat Med. 2023 Apr;29(4):869-879. doi: 10.1038/s41591-023-02273-z. Epub 2023 Apr 17.

  PMID: 37069359 DERIVED

• Crees ZD, Stockerl-Goldstein K, Vainstein A, Chen H, DiPersio JF. GENESIS: Phase III trial evaluating BL-8040 + G-CSF to mobilize hematopoietic cells for autologous transplant in myeloma. Future Oncol. 2019 Nov;15(31):3555-3563. doi: 10.2217/fon-2019-0380. Epub 2019 Sep 9.

  PMID: 31495201 DERIVED

MeSH Terms

Conditions

Multiple Myeloma

Interventions

4-fluorobenzoyl-TN-14003; Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: VP Clinical & Medical Affairs
• Organization: BioLineRx Ltd

clinicaltrials@biolinerx.com

+972-8-6429100

Study Officials

• John DiPersio, MD

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

• Crees Zachary, MD

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Subjects were randomized using a 2:1 ratio to receive G-CSF + BL-8040 or G-CSF + Placebo, respectively. Randomization will use permuted blocks stratifying subjects by US geographical region (NorthEast, SouthEast, MidWest, SouthWest and NorthWest), remission status (CR vs. PR), and baseline platelet count (\< 200 × 10\^9/L or ≥ 200 × 10\^9/L).

Study Record Dates

• First Submitted: August 2, 2017
• First Posted: August 11, 2017
• Study Start: March 23, 2018
• Primary Completion: December 22, 2020
• Study Completion (Estimated): September 30, 2029
• Last Updated: January 15, 2026
• Results First Posted: November 7, 2023

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); DE (1); US (10); HU (2); IT (2)