Assessing the safety and effectiveness of bexmarilimab for blood cancers

A Phase I/II Open-Label Study to Assess the Safety, Tolerability and Preliminary Efficacy of the Clever-1 Antibody Bexmarilimab in Combination With Standard of Care Therapy in Patients With Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia or Acute Myeloid Leukemia

This study is testing a new drug called bexmarilimab to see if it can safely help people with certain blood cancers, like leukemia and myelodysplastic syndromes, when used alongside standard treatments.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 181 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Faron Pharmaceuticals Ltd (industry)
• Drugs / interventions: chemotherapy, immunotherapy, prednisone, bexmarilimab
• Locations: 10 sites (Duarte, California and 9 other locations)
• Trial ID: NCT05428969 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety, tolerability, and preliminary efficacy of bexmarilimab in combination with standard care for patients with hematological malignancies, including acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), and myelodysplastic syndromes (MDS). The trial is divided into two phases: Phase 1 focuses on determining the maximum tolerated dose of bexmarilimab using a dose escalation design, while Phase 2 aims to assess the efficacy of the treatment at the recommended dose. Patients will undergo screening, treatment, and follow-up to monitor safety and disease progression.

Who should consider this trial

Good fit: Ideal candidates include adults with specific types of hematological malignancies who have not responded to previous treatments or are unfit for standard induction therapy.

Not a fit: Patients with acute promyelocytic leukemia or those who do not meet the eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with difficult-to-treat blood cancers.

How similar studies have performed: Other studies have shown promise in using immunotherapy approaches for hematological malignancies, but the specific combination of bexmarilimab with standard care is novel.

Eligibility criteria

Inclusion Criteria:

* Patient ≥ 18 years of age who presents with one of the following conditions:

  * Morphologically confirmed diagnosis of MDS with revised International Prognostic Scoring System (rIPSS) risk categories: intermediate, high and very high.
  * Morphologically confirmed diagnosis of CMML-2 with indication for azacitidine treatment.
  * CMML and MDS patient with response failure to HMA or therapy regimen including HMA.
  * Morphologically confirmed diagnosis of r/r AML following at least 1 line of prior therapies with indication for azacitidine treatment.
  * Morphologically confirmed diagnosis of AML in patients unfit for induction therapy with indication for azacitidine-venetoclax treatment.
* Leukocyte count \< 20 x10\^9/L (\< 25 x10\^9/L for newly diagnosed AML). Hydroxycarbamide use is permitted to meet this criterion in MDS and AML but not in CMML.
* Adequate renal function.
* Adequate liver function.

Exclusion Criteria:

* Patient with acute promyelocytic leukemia (APL) or myeloproliferative CMML as defined by leukocyte count \> 13 x10\^9/L.
* Eastern Cooperative Oncology Group (ECOG) performance status \>2 (except newly diagnosed AML where ECOG 3 is allowed for patients \< 75 years).
* Allogeneic transplantation less than 6 months prior screening.
* Patient with active auto-immune disorder (except type I diabetes, celiac disease, hypothyroidism requiring only hormone replacement, vitiligo, psoriasis, or alopecia).
* The patient requires systemic corticosteroid (≥10 mg/day prednisone or equivalent) or other immunosuppressive treatment.
* Less than 21 days since the last dose of intravenous anticancer chemotherapy or less than 14 days or five half-lives (whichever is shorter) from a small molecule targeted therapy or oral anticancer chemotherapy before the first study treatment.
* Any immunotherapy or investigational therapy within preceding 28 days from the first study treatment.
* Pregnant or lactating women.
* History of chronic ulcers or clinically relevant liver disease leading to Child Pugh Score C or higher.

Where this trial is running

Duarte, California and 9 other locations

• City of Hope National Medical Center — Duarte, California, United States (RECRUITING)
• Yale Cancer Center — New Haven, Connecticut, United States (RECRUITING)
• UNC Lineberger Comprehensive Cancer Center — Chapel Hill, North Carolina, United States (RECRUITING)
• University of Texas, MD Anderson Cancer Center — Houston, Texas, United States (RECRUITING)
• Helsinki University Hospital — Helsinki, Finland (RECRUITING)
• Kuopio University Hospital — Kuopio, Finland (RECRUITING)
• Oulu University Hospital — Oulu, Finland (RECRUITING)
• Tampere University Hospital — Tampere, Finland (RECRUITING)
• The Christie NHS Foundation Trust — Manchester, United Kingdom (RECRUITING)
• Royal Cornwall Hospitals NHS Trust — Truro, United Kingdom (RECRUITING)

Study contacts

• Principal investigator: Mika Kontro, MD, PhD — Helsinki University Central Hospital
• Study coordinator: Joab Williamson

Conditions: Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndromes, Relapsed/Refractory AML, Macrophages, Immunotherapy, Hematological, CLEVER-1