RecruitingPhase 1 / Phase 2

Pacritinib in CMML

Pacritinib in Combination With Azacitidine in Patients With Chronic Myelomonocytic Leukemia

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: 7 days

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
  • "Pacritinib will be initially tested at a dose of 200mg twice daily (dose level 0) in combination with azacitidine 75mg/m2, which can be administered subcutaneously or intravenously, for 7 days in a 28-day cycle."
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 pacritinib with azacitidine for adults with chronic myelomonocytic leukemia (CMML), a blood cancer. It aims to find a dose and see how well the treatment works. People may be newly diagnosed or previously treated, but must meet limits on past treatment.

This is a phase 1/2 trial of pacritinib in combination with azacitidine in patients with Chronic Myelomonocytic Leukemia (CMML). Patients will be newly diagnosed or previously treated but could not have received a prior JAK inhibitor. Patients who have previously been treated with a hypomethylating agent (HMA) must have received ≤ 1 cycle. Pacritinib will be initially tested at a dose of 200mg twice daily (dose level 0) in combination with azacitidine 75mg/m2, which can be administered subcutaneously or intravenously, for 7 days in a 28-day cycle. If there are 2 DLTs in the first 6 patients, there will be a dose escalation to pacritinib 100mg twice daily (dose level -1) and an additional 6 patients will be enrolled. Based on the phase 1, 3+3 dose de-escalation design, 6-12 patients will be enrolled in the phase 1 portion. After the completion of phase 1 and identification of the recommended phase 2 dose (RP2D), the trial will then proceed to phase 2 which will employ a Simon two stage design. This portion will include the 6 patients enrolled during the phase 1 portion at the MTD. An interim analysis for futility will occur. If 3 or fewer patients have had a clinical benefit (CB) or better, as defined by 2015 MDS/MPN IWG criteria, the PI and DSMC will meet to discuss the totality of the evidence and determine if the trial shall proceed. In the second stage, an additional 12 patients will be enrolled.

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, understand and sign the consent form, and be able to follow the study schedule.
  • Have CMML confirmed by lab testing.
  • Have a high white blood cell count (at least 13 billion per liter), or CMML rated intermediate-2 or high risk by the study’s scoring system.
  • Have never taken a JAK inhibitor, a type of cancer drug. Have had no more than one cycle of a hypomethylating agent (HMA), another type of cancer drug.
  • Be expected by the study doctor to live at least 24 weeks.
  • Meet the study’s limit for how much illness affects daily activities (ECOG score of 3 or less).
  • Meet blood test limits for liver and kidney function, blood clotting, and infection-fighting white blood cells.
  • Use effective birth control as required. Women who can become pregnant must use it during azacitidine treatment and for 6 months after; men must follow the study’s rules during treatment and for at least 90 days after.

Who can't join

  • Current or recent treatment in another study, or recent use of certain drugs that affect how the body processes medicines. Required waiting times vary.
  • Active graft-versus-host disease, when donor cells attack the body, or treatment for it that is not allowed. An organ transplant other than a bone marrow transplant also rules you out.
  • Another invasive cancer within 3 years, except certain cancers treated to cure them.
  • A serious active infection, uncontrolled HIV, active hepatitis B or C needing treatment, or cirrhosis (liver scarring). Some people with COVID-19 may qualify after a doctor’s review.
  • Recent significant bleeding or use of medicines that raise bleeding risk. Certain heart problems, heart failure, or an increased risk of abnormal heart rhythm also rule you out; some exceptions apply.
  • A serious or unstable physical or mental health condition that makes joining unsafe or affects study results. Gut problems, past gut surgery, or trouble swallowing that could affect taking pacritinib also rule you out.
  • Being pregnant or breastfeeding.
  • Being trial site or sponsor staff directly involved in this study, or having an immediate family member in that role, unless an ethics review board approves an exception.

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

Official eligibility criteria

Inclusion Criteria: * Participants must be ≥18 years of age at time of signing the Informed Consent Form (ICF). * Participants must voluntarily sign an ICF. * Participants must have a pathologically confirmed diagnosis of chronic myelomonocytic leukemia per World Health Organization (WHO) or International Consensus Classification (ICC) * Participants must be JAK inhibitor naïve. * Participants may be hypomethylating agent (HMA) naïve or can be treated with up to one prior cycle. * Participants must have either proliferative CMML (WBC ≥13 x 109/L) or have intermediate-2 or high risk CMML by the clinical/molecular CMML-specific prognostic scoring system (CPSS-Mol). * Participants must have a life expectancy of at least 24 weeks per investigator. * ECOG performance status ≤ 3. * Females of reproductive potential should use effective contraception during treatment with azacitidine and for 6 months after the last dose and males with female partners of reproductive potential should use effective contraception during treatment with azacitidine and for 3 months after the last dose. * Male participants should agree to use an adequate method of contraception starting with the first dose of study therapy through 90 days after the last dose of study therapy. * Must have adequate organ function as demonstrated by the following: * Serum total bilirubin ≤ 2.0 x upper limit of normal (ULN) unless considered due to leukemic organ involvement, Gilbert's syndrome, or hemolysis. * Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 x ULN. * Creatinine clearance (CrCl) of ≥30 mL/min. * PT or INR \<=1.5x ULN and PTT or aPTT \<=1.5x ULN. * ANC \>= 500 cells/μL. * Ability to adhere to the study visit schedule and all protocol requirements. * Ability to understand and the willingness to sign a written informed consent. Exclusion Criteria: * Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 2 weeks or within 5 half-lives of the prior investigational agent, whichever is shorter, of the first dose of treatment. * Active Graft Versus Host Disease (GVHD), or any GVHD requiring treatment with immunosuppression, with the exception of topical steroids and systemic steroids at a dose equivalent to prednisone 10mg or less and at a stable or decreasing dose. Any GVHD treatment (including calcineurin inhibitors) must be discontinued at least 28 days prior to Day 1 of study treatment. * Systemic treatment with a strong CYP3A4 inhibitor or a strong CYP450 inducer within 14 days prior to treatment Day 1 (see Appendix 13.7 and 13.8 for a list of CYP3A4 inhibitors and CYP450 inducers, respectively). Shorter washout periods may be permitted with approval of the Study Chair, provided that the washout period is at least five half-lives of the drug prior to treatment Day 1. * Other invasive malignancies within the last 3 years, except curatively treated non-melanoma skin cancer, localized prostate, cervical cancer, and any curatively treated carcinoma in situ. * Presence of active serious infection. * If a patient is identified to have COVID-19 during the screening period, participants may be considered eligible if in the opinion of the investigator there are no COVID-19 sequlae that may place the patient at a higher risk of receiving investigational treatment. * Any serious, unstable medical or psychiatric condition that would prevent, (as judged by the Investigator) the patient from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the patient at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study. * Known history of uncontrolled human immunodeficiency virus (HIV). * Significant recent bleeding history defined as NCI CTCAE grade ≥2 within 3 months prior to treatment Day 1, unless precipitated by an inciting event (e.g., surgery, trauma, or injury). * Systemic treatment with medications that increase the risk of bleeding, including anticoagulants, antiplatelet agents (except for aspirin dosages of ≤100 mg per day), anti-vascular endothelial growth factor (anti-VEGF) agents, and daily use of COX-1 inhibiting Non-steroidal anti-inflammatory drugs (NSAIDs) within 14 days prior to treatment Day 1. * Any history of CTCAE grade ≥2 cardiac conditions within 6 months prior to treatment Day 1. Patients with asymptomatic grade 2 non-dysrhythmia cardiovascular conditions may be considered for inclusion, with the approval of the Study Chair, if stable and unlikely to affect patient safety. * Heart failure other than NYHA class I (asymptomatic, without limitation). * QT corrected by the Fridericia method (QTcF) prolongation \>480 ms or other factors that increase the risk for QT interval prolongation (e.g., hypokalemia \[defined as serum potassium \<3.0 mEq/L that is persistent and refractory to correction\], or history of long QT interval syndrome). * Known active systemic hepatitis B, or C infection requiring therapy or known cirrhosis. * Is or has an immediate family member (e.g., spouse, parent/legal guardian, sibling, or child) who is investigational site or pharmaceutical sponsor staff directly involved with this trial, unless prospective IRB approval (by chair or designee) is given allowing exception to this criterion for a specific participant. * Organ transplant recipients other than bone marrow transplant. * Women who are pregnant or lactating. * Patient with known gastrointestinal (GI) disease or prior GI procedure that could interfere with the oral absorption or tolerance of pacritinib, including difficulty swallowing, are not eligible.

Study sites (1)

NY