Pacritinib for Myelodysplastic Syndrome
Trial Summary
What is the purpose of this trial?
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.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you must stop all current medications, but you cannot take certain medications that interact with the study drug, pacritinib. You must stop using strong inhibitors and inducers of CYP3A4 two weeks before starting the study drug. It's important to discuss your current medications with the trial team to ensure they don't interfere with the study.
How is the drug Pacritinib unique for treating myelodysplastic syndrome?
Pacritinib is unique because it is a targeted therapy that inhibits specific enzymes (kinases) involved in cell growth and survival, which may help manage myelodysplastic syndrome by addressing the underlying disease mechanisms. This approach differs from traditional treatments that may not specifically target these pathways.12345
Research Team
Alain Mina, M.D.
Principal Investigator
National Cancer Institute (NCI)
Eligibility Criteria
This trial is for children (12-17 years) and adults (18+ years) with blood disorders called Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms. Participants must be able to undergo various medical tests, including a bone marrow biopsy, and commit to an up-to-8-year study period with regular clinic visits.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive pacritinib orally in 28-day cycles, with weekly visits during cycle 1, bi-weekly visits during cycle 2, and gradually increasing to every 3 months after cycle 13. Treatment continues for up to 8 years.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including bone marrow biopsies, heart tests, and quality of life questionnaires.
Open-label extension (optional)
Participants may opt into continuation of treatment long-term
Treatment Details
Interventions
- Pacritinib
Pacritinib is already approved in United States for the following indications:
- Intermediate or high-risk primary or secondary myelofibrosis with platelet counts below 50 × 10^9/L
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor