RecruitingPhase 1 / Phase 2

Axatilimab With or Without Azacitidine for the Treatment of Patients With Advanced Phase Myeloproliferative Neoplasms, Myeloproliferative Neoplasm/Myelodysplastic Syndrome Overlap or High Risk Chronic Myelomonocytic Leukemia

Phase 1b/2 Study of Axatilimab (SNDX-6352) + Azacitidine (AZA) in Advanced Phase MPN, MPN/MDS Overlap or High-Risk CMML

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 24 months
  • Procedures mentioned: Blood draws, Biopsies

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Patients undergo bone marrow biopsy and aspiration and blood sample collection throughout the study."
  • "After completion of study treatment, patients are followed up at 30 days and then every 6 months for up to 24 months after last dose of study medication."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests the best dose of axatilimab and how well it works alone or with azacitidine. It is for adults with certain advanced or higher-risk blood cancers, including MPN, MPN/MDS overlap, and CMML. The study also checks whether these treatments are safe.

This phase Ib/II trial tests the best dose of axatilimab and effectiveness of axatilimab with or without azacitidine for the treatment of patients with advanced phase myeloproliferative neoplasms (MPN), myeloproliferative neoplasm/myelodysplastic syndrome (MPN/MDS) overlap or high risk chronic myelomonocytic leukemia (CMML). Axatilimab is an antibody that is cloned from a single white blood cell that is known to be able to recognize cancer cells and block a protein on the surface of the white blood cells that may be involved in cancer cell growth. By blocking the proteins, this may slow or halt the growth of the cancer. Azacitidine is in a class of medications called antimetabolites. It works by stopping or slowing the growth of cancer cells. Giving axatilimab with or without azacitidine may be safe and effective in treating patients with advanced phase MPN, MPN/MDS overlap or high risk CMML.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older, sign a consent form, and be able to follow study steps and talk with the study doctor.
  • Have a confirmed blood cancer listed in the study: intermediate-2 or high-risk CMML, atypical CML, certain MDS/MPN overlap cancers, or accelerated-phase MPN. These cancers affect blood-forming cells.
  • For accelerated-phase MPN, have a prior diagnosis of polycythemia vera, essential thrombocythemia, or primary myelofibrosis, meet the study's risk level, and have disease that worsened or did not respond after at least one treatment.
  • For phase Ib, have cancer that came back or did not respond after at least two cycles of treatment.
  • For phase II, have newly diagnosed cancer with no prior treatment, except hydroxyurea, ruxolitinib, or up to two cycles of certain drugs, including azacitidine or decitabine.
  • The study doctor must find that strong chemotherapy is not suitable right away. You must also be able to care for yourself and be up and about for at least half the day.
  • Meet the study's liver and kidney blood test limits.
  • If required, use the specified birth control from 14 days before joining until 90 days after the last follow-up visit. This means hormones or an IUD plus a condom, diaphragm, or cervical cap.

Who can't join

  • Have acute myeloid leukemia (AML), including acute promyelocytic leukemia or AML outside the bone marrow.
  • Be a candidate for strong chemotherapy, or not give consent for that treatment.
  • Have had an organ transplant or a blood stem cell transplant from a donor.
  • Have a past cancer that still needs treatment during the study. Some people with adequately treated cancer or those taking hormone therapy may join.
  • Have a cancer caused by earlier cancer treatment.
  • Have a known allergy or sensitivity to ingredients in axatilimab.
  • Have had pancreatitis, which is swelling of the pancreas.
  • Have had myositis, which is swelling of the muscles.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Signed informed consent must be obtained prior to participation in the study * Age ≥ 18 years at the date of signing the informed consent form (ICF) * Morphologically confirmed diagnosis of the following based on 2016 World Health Organization (WHO) classification (Arber et al 2016): Phase 1b, patients with relapsed or refractory of any of the following; phase 2, patients with newly diagnosed of any of the following: * Chronic myelomonocytic leukemia (CMML), classified as intermediate-2, OR high-risk per the CMML Specific Prognostic Scoring System (CPSS) Molecular Model * Atypical chronic myelocytic leukemia (aCML) * MDS/MPN unclassified (MDS/MPN-U) * Myeloproliferative neoplasm accelerated phase (MPN-AP) * MPN-AP requires a previous diagnosis of polycythemia vera (PV), essential thrombocythemia (ET), or primary myelofibrosis (PMF) with intermediate-2 or high risk disease according to International Prostate Symptom Score (IPSS) as well as progression on or failure to respond to at least one line of therapy. * Myelodysplastic syndrome/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T) or MDS/MPN with SF3B1 mutation and thrombocytosis (MDS/MPN-SF3B1-T). * Not suitable for immediate myeloablative/intensive chemotherapy based on investigator assessment of age, comorbidities, local guidelines, institutional practice (any or all of these) * PHASE Ib (RELAPSE \[R\]/ REFRACTORY \[R\]): Relapse/refractory patients who have received at least two cycles of disease directed therapy (prior therapies can include hypomethylating agents \[HMAs\], HMA combination therapies, and other disease directed therapies) * PHASE II (NEWLY DIAGNOSED PHASE): Newly diagnosed patients without prior treatment, including intensive induction chemotherapy. However, previous treatment with hydroxyurea, ruxolitinib, and/or up to 2 cycles of HMAs (decitabine, azacitidine, oral decitabine \[INQOVI\]) is permitted * Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN) * Total bilirubin ≤ 1.5 × ULN (except in the setting of isolated Gilbert syndrome) * Estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73m\^2 (estimation based on Modification of Diet in Renal Disease \[MDRD\] formula, by local laboratory) * Patient is able to communicate with the investigator and has the ability to comply with the requirements of the study procedures * Women of childbearing potential and men, if not surgically sterilized, should use adequate contraception from 14 days prior to study entry and until 90 days after the last follow-up visit. Adequate contraception is defined as using hormonal contraceptives or an intrauterine device combined with at least 1 of the following forms of contraception: a diaphragm or cervical cap, or a condom Exclusion Criteria: * Diagnosis of acute myeloid leukemia (AML) including acute promyelocytic leukemia and extra-medullary AML based on WHO 2016 classification (Arber et al 2016) * Patients who are candidates for myeloablative or intensive chemotherapy treatment or who do not provide consent for this treatment * History of organ transplant or allogenic hematopoietic stem cell transplant * Participants with prior malignancy, except: * Participants with history of adequately treated malignancy for which no anticancer systemic therapy (namely chemotherapy, radiotherapy or surgery) is ongoing or required during the course of the study. * Participants who are receiving adjuvant therapy such as hormone therapy are eligible. However, participants who developed therapy related neoplasms are not eligible * Previous known allergy/sensitivity to components of axatilimab * History of acute or chronic pancreatitis * History of myositis

Study sites (1)

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