Actively Recruiting
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Led by National Cancer Institute (NCI) · Updated on 2026-05-14
160
Participants Needed
1
Research Sites
449 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults. MDS and MDS/MPN can also progress to acute myeloid leukemia. Treatments for these disorders are risky and not always effective. Better treatments are needed. Objective: To test a study drug (pacritinib) in adults and children with MDS or MDS/MPN. Eligibility: Children (aged 12 to 17 years) and adults (aged 18 years and older) with MDS or MDS/MPN. Design: Participants will be screened. They will have a physical exam with blood tests. They will have tests of their heart function. They may have a bone marrow biopsy: An area over the hip will be numbed; a needle will be inserted to remove a sample of soft tissue from inside the hipbone. Pacritinib is a capsule taken by mouth. All participants will take the study drug 2 times a day, every day, in 28-day cycles. They will write down the date and time they take each capsule. Doctors will assign varying dosages of the drug to different participants. Participants will have clinic visits each week during cycle 1; every 2 weeks during cycle 2; and gradually increasing to every 3 months after cycle 13. Treatment will continue for up to 8 years. Bone marrow biopsies, heart tests, and other tests will be repeated at intervals throughout the study. Participants will also fill out questionnaires about their quality of life, the symptoms of their disease, and other topics.
CONDITIONS
Official Title
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must have confirmed diagnosis of MDS or MDS/MPN, including therapy-related or germline predisposition types, according to established criteria
- Age 12 to 17 years for phase I or 18 years and older for phase II
- Adults with higher-risk MDS must have resistance, intolerance, or contraindication to hypomethylating agents
- Adults with lower-risk MDS must be refractory or ineligible for standard therapies and have severe neutropenia, symptomatic anemia, or thrombocytopenia with bleeding history
- Participants aged 12-17 with MDS must be relapsed/refractory or ineligible for immunosuppressive therapy and stem cell transplant
- Participants with MDS/MPN must be relapsed/refractory, ineligible, or without known life-prolonging therapy options
- Participants aged 12-17 must weigh at least 35 kg
- Certain prior therapies must have ended within specified timeframes before study start
- Performance status must be ECOG less than 2 for those aged 16 and older or Lansky 60% or higher for younger participants
- Adequate organ function as defined by specific lab criteria
- Individuals able to father or bear children must agree to effective contraception during and for 30 days after study drug use
- Nursing mothers must stop breastfeeding until 2 weeks after last dose
- Ability to understand and sign informed consent
You will not qualify if you...
- Platelet transfusion-refractory thrombocytopenia with inability to maintain platelets above 10,000/mcL
- Evidence of active or recent severe bleeding or hemorrhage
- Use of antiplatelet or anticoagulant medications other than low-dose aspirin within 14 days before enrollment
- Unwillingness to accept blood transfusions
- ANC below 500 cells/mcL and hospitalization for fungal infection within past year
- History of allergic reactions to similar compounds as pacritinib
- Use of certain medications that interact with pacritinib or cause QT prolongation
- Cardiac conditions including symptomatic heart failure, unstable angina, uncontrolled arrhythmias, or prolonged QTc interval
- Recent severe cardiac complications or low heart function
- Active, uncontrolled infections including HIV, hepatitis B or C
- Pregnancy
- Other causes of cytopenia or marrow disease that may interfere with results
- Uncontrolled illnesses or social situations that may limit compliance or increase risk
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
R
Rania S Hishmeh, R.N.
CONTACT
N
Najla El Jurdi, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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