This research is being done to evaluate effectiveness, safety, and tolerability of a study drug called momelotinib in participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPNs), MDS/MPN-not otherwise specified (MDS/MPN-NOS), MDS/MPN with neutrophilia (MDS/MPN-N), also called as atypical chronic myeloid leukemia, or chronic neutrophilic leukemia. Momelotinib will be added to standard treatment which usually includes a hypomethylating agent like azacitidine. Treatment options for this diagnosis remain limited and investigators need better treatments to help control the disease, improve symptoms, and potentially help more patients become eligible for transplant. Participants for this study will be asked to take some screening tests which will include routine physical examination, blood tests, and imaging scans to determine eligibility for the study. Those who continue to qualify for this study will begin treatment and may be asked to remain on the study drug for up to 24 months, depending upon how they are responding to treatment. After the study drug is completed, patients will have one additional clinic visit to evaluate overall health and response to study drug. The study drug treatment on this study will include taking momelotinib by mouth in combination with azacitidine, which is given by injection for all patients for the first 5 days of each 28-day cycle. The most common side effect that may be related to participation in this study can include (i) infections which can present as fever, chills, cough, breathing problems, diarrhea, vomiting, pain or burning with urination; or (ii) low blood platelet count which can result in bruising or bleeding for longer than usual if the participant hurts themself.
This is an open-label study of MMB-HMA in MDS/MPN and CNL that will enroll up to 18 patients. Momelotinib will be administered using modified 3+3 dose escalation design followed by expansion. The first three patients will be treated at 150mg daily and if DLT criteria are not met, the remaining patients will be treated at 200mg daily to a total of 18 evaluable patients (all in combination with azacitidine). If DLTs are met within the dose escalation phase (first three patients), then the patients will be treated at 150mg daily (in combination with azacitidine) Key Eligibility Criteria: 1. Patients with a WHO diagnosis of chronic myelomonocytic leukemia (CMML), MDS/MPN-not otherwise specified (MDS/MPN-NOS), MDS/MPN with neutrophilia (MDS/MPN-N), CNL. 2. Age greater than or equal to 18 years 3. Blood counts with platelets greater than 25,000/microL, ANC greater than or equal to 0.75 x 10\^9/L (without transfusion or growth factor support) 4. Baseline splenomegaly with greater than or equal to 5 cm below costal margin or greater than or equal to 450 cm3 on imaging (ultrasound, CT or MRI)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Capsules of Momelotinib will be administered orally once a day by all participants for up to 24 months, depending on response to treatment. Dose escalation will include the first 3 patients who will receive either 150 mg or 200mg of momelotinib daily, depending on the number of dose limiting toxicities (DLTs) experienced during this period. Dose expansion will include up to 18 evaluable participants who will receive the maximum tolerated dose (either 150mg or 200 mg) daily as will be determined during the dose escalation phase.
75 mg/m2, days 1-5 in a 28-day cycle
Number of participants who achieve a complete response
Efficacy determination - complete response: number of participants who achieve a complete response as defined by international consortium response criteria for MDS/MPN in adults
Time frame: 24 weeks
Number of participants who achieve a partial response
Efficacy determination - partial response: number of participants who achieve a partial response as defined by international consortium response criteria for MDS/MPN in adults
Time frame: 24 weeks
Number of participants who achieve a clinical benefit
Efficacy determination - clinical benefit: number of participants who achieve a clinical benefit per MDS/MPN IWG criteria4
Time frame: 24 weeks
Number of participants who experience a grade 3 or higher adverse events or severe event
Safety and tolerability evaluation: number of participants who experience a grade 3 or higher adverse event events or severe adverse events as defined by CTCAE v.5.0
Time frame: 2 years
Number of Participants Who Achieve Erythroid Response
Number of patients who achieve erythroid response at week 12 per international consortium criteria
Time frame: 12 weeks
Number of Participants Who Achieve Erythroid Response
Number of participants who achieve erythroid response at week 24 per international consortium criteria
Time frame: 24 weeks
Proportion of Participants Who Achieve Spleen Size Reduction
Determine the proportion of participants who achieve a spleen volume reduction of ≥ 35% from baseline at week 12
Time frame: 12 weeks
Proportion of participants Who Achieve Spleen Size Reduction
Determine the proportion of participants who achieve a spleen volume reduction of ≥ 35% from baseline at week 24
Time frame: 24 weeks
Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Evaluate efficacy of momelotinib plus hypomethylating agents azacitidine or decitabine (MMB-HMA) on MPN-SAF TSS questionnaire. Score range 0-100, higher score worse symptoms.
Time frame: 12 weeks
Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS)
Evaluate efficacy of momelotinib plus hypomethylating agents azacitidine or decitabine (MMB-HMA) on MPN-SAF TSS questionnaire. Score range 0-100, higher score worse symptoms.
Time frame: 24 weeks
Patient Global Impression of Change (PGIC) questionnaire
Evaluate efficacy of momelotinib plus hypomethylating agents azacitidine or decitabine (MMB-HMA) on Patient Global Impression of Change (PGIC) questionnaire. A 7-point scale (1-7) rating of overall improvement. Patients rate their change as 1="very much improved," 2="much improved," 3="minimally improved," 4="no change," 5="minimally worse," 6="much worse," or 7="very much worse." Lower values represent a better outcome.
Time frame: 12 weeks
Patient Global Impression of Change (PGIC)
Evaluate efficacy of momelotinib plus hypomethylating agents azacitidine or decitabine (MMB-HMA) on Patient Global Impression of Change (PGIC) questionnaire. A 7-point scale (1-7) rating of overall improvement. Patients rate their change as 1="very much improved," 2="much improved," 3="minimally improved," 4="no change," 5="minimally worse," 6="much worse," or 7="very much worse." Lower values represent a better outcome.
Time frame: 24 weeks
Trough concentrations of MMB as single agent and in combination with HMA
Evaluate trough concentrations at week 12 in Arm A and B
Time frame: 12 weeks
Trough concentrations of MMB as single agent and in combination with HMA
Evaluate trough concentrations at week 24 in Arm A and B
Time frame: 24 weeks
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