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Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies
Phase 2 trial testing BL-8040 in Acute Myelogenous Leukemia in 50 participants. Completed in 7 April 2023.
| Lead sponsor | Washington University School of Medicine |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | non randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 50 |
| Start date | 31 March 2016 |
| Primary completion | 12 April 2018 |
| Estimated completion | 7 April 2023 |
| Sites | 3 locations across United States |
Washington University School of Medicine
Adults 18 to 75, any sex, with Acute Myelogenous Leukemia or Acute Lymphoblastic Leukemia. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 22 |
-Adverse events will be graded according to the NCI CTCAE version 4.03
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 17 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 1 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 5 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 6 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 2 |
-Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/μL following conditioning regimen induced nadir.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 13 | 11 – 26 |
-Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 20,000/μL without platelet transfusion support for 7 days.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 20 | 15 – 41 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0 |
* Acute GVHD rate and worst severity is noted * 4 organ categories (skin, liver, lower GI, and upper GI) * Skin: Grade I: 1-2 , Grade II: 3, Grade III: N/A, Grade IV: 4 * Liver: Grade I: 0, Grade II: 1, Grade III: 2-4, Grade IV: N/A * Lower GI: Grade I: 0, Grade II: 1: Grade II: 2-3: Grade IV: 4 * Upper GI: Grade I: 0, Grade II: 1, Grade III: N/A, Grade IV: N/A * The cumulative incidence of grade 2-4 acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0.3636 | 0.1690 – 0.5623 |
* Chronic GVHD rate and severity for the first 365 days after PBSC infusion will be assessed based on the NIH criteria * The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0.5000 | 0.2676 – 0.6946 |
| Group | Value | 95% CI |
|---|---|---|
| Arm 1: Donors | 9 |
-CMV reactivation will be defined as a positive test for CMV viremia as determined by an antigenemia assay or quantitative PCR that results in the administration of antiviral treatment directed against CMV
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 8 |
-Death that results from a transplant procedure related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0.2273 | 0.0792 – 0.4208 |
-Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made
| Group | Value | 95% CI |
|---|---|---|
| Arm 2: Recipients | 0.227 | 0.079 – 0.420 |
Time frame: -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | — | — |
| Acute kidney injury | Renal and urinary disorders | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Sepsis | Infections and infestations | — | — |
| Mucositis oral | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Fever | General disorders | — | — |
| Generalized weakness | General disorders | — | — |
| Upper respiratory infection | Infections and infestations | — | — |
| Fall | Injury, poisoning and procedural complications | — | — |
| Headache | Nervous system disorders | — | — |
| Delirium | Psychiatric disorders | — | — |
| Genital edema | Reproductive system and breast disorders | — | — |
| Testicular pain | Reproductive system and breast disorders | — | — |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | — | — |
| Reaction | System | Arm 1: Donors | Arm 2: Recipients |
|---|---|---|---|
| Lymphocyte count increased | Investigations | — | — |
| Platelet count decreased | Investigations | — | — |
| Injection site reaction | General disorders | — | — |
| Allergic reaction | Immune system disorders | — | — |
| Anemia | Blood and lymphatic system disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| Hypocalcemia | Metabolism and nutrition disorders | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Mucositis oral | Gastrointestinal disorders | — | — |
| Lymphocyte count decreased | Investigations | — | — |
| Oral dysesthesia | Gastrointestinal disorders | — | — |
| Hypophosphatemia | Metabolism and nutrition disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Hyperglycemia | Metabolism and nutrition disorders | — | — |
| Hypokalemia | Metabolism and nutrition disorders | — | — |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — |
| Fatigue | General disorders | — | — |
| Headache | Nervous system disorders | — | — |
| Paresthesia | Nervous system disorders | — | — |
| Sinus tachycardia | Cardiac disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Clostridium difficile | Infections and infestations | — | — |
| Lung infection | Infections and infestations | — | — |
| Urinary tract infection | Infections and infestations | — | — |
| Alanine aminotransferase increased | Investigations | — | — |
| White blood cell decreased | Investigations | — | — |
| Hyperkalemia | Metabolism and nutrition disorders | — | — |
| Hyperuricemia | Metabolism and nutrition disorders | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Bone pain | Musculoskeletal and connective tissue disorders | — | — |
| Hypotension | Vascular disorders | — | — |
| Acute systolic heart failure | Cardiac disorders | — | — |
| Chest pain-cardiac | Cardiac disorders | — | — |
| Sinus brachycardia | Cardiac disorders | — | — |
| Eye pain | Eye disorders | — | — |
| Watering eyes | Eye disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Colitis | Gastrointestinal disorders | — | — |
| Diarrhea | Gastrointestinal disorders | — | — |
| Esophagitis | Gastrointestinal disorders | — | — |
Most-reported serious reactions: Diarrhea, Acute kidney injury, Febrile neutropenia, Sepsis, Mucositis oral, Nausea, Vomiting, Fever.
Data from ClinicalTrials.gov NCT02639559 adverse events section.
Current protocols use G-CSF to mobilize hematopoietic progenitor cells from matched sibling and volunteer unrelated donors. Unfortunately, this process requires four to six days of G-CSF injection and can be associated with side effects, most notably bone pain and rarely splenic rupture. BL-8040 is given as a single SC injection, and collection of cells occurs on the same day as BL-8040 administration. This study will evaluate the safety and efficacy of this novel agent for hematopoietic progenitor cell mobilization and allogeneic transplantation based on the following hypotheses: * Healthy HLA-matched donors receiving one injection of BL-8040 will mobilize sufficient CD34+ cells (at least 2.0 x 10\^6 CD34+ cells/kg recipient weight) following no more than two leukapheresis collections to support a hematopoietic cell transplant. * The hematopoietic cells mobilized by SC BL-8040 will be functional and will result in prompt and durable hematopoietic engraftment following transplantation into HLA-identical siblings with advanced hematological malignancies using various non-myeloablative and myeloablative conditioning regimens and regimens for routine GVHD prophylaxis. * If these hypotheses 1 and 2 are confirmed after an interim safety analysis of the data, then the study will continue and include recruitment of haploidentical donors.
8 peer-reviewed publications reference this trial (live from Europe PMC):
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