Study on Pacritinib for Treating Blood Disorders in Adults and Children Aged 12 and Older

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT06303193

Provider View

This clinical trial is testing a new drug, pacritinib, to see if it can help treat blood disorders called myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults and children aged 12 and older. The study aims to find out if the drug is safe and effective in managing these conditions.

Patient Parameters

Program Overview

The study is focused on testing a new medication called pacritinib for treating blood disorders known as myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN). These conditions can lead to serious health issues and may progress to a type of blood cancer called acute myeloid leukemia. Current treatments are not always effective, so this study aims to find better options. Participants will take pacritinib capsules twice a day in 28-day cycles, with regular check-ups and tests to monitor their health and the drug's effects. The study will last up to 8 years, with participants undergoing various tests and filling out questionnaires about their health and quality of life.

Description

This study is testing a new drug called pacritinib, which targets specific proteins involved in blood disorders like myelodysplastic syndromes (MDS) and myelodysplastic/myeloproliferative neoplasms (MDS/MPN). These disorders affect the blood and bone marrow, leading to low blood cell counts and an increased risk of developing acute myeloid leukemia. Pacritinib works by inhibiting certain proteins that contribute to these conditions, potentially offering a new treatment option.

The study is divided into two phases. Phase I focuses on determining the safe dosage for children aged 12 to 17, while Phase II evaluates the drug's effectiveness in adults aged 18 and older. Participants will take pacritinib capsules twice daily in 28-day cycles, with regular visits to the clinic for check-ups and tests. These include blood tests, heart function tests, and bone marrow biopsies. Participants will also complete questionnaires about their symptoms and quality of life.

The study aims to find out if pacritinib can help manage these blood disorders more effectively than current treatments. It will also assess the drug's safety and any potential side effects. Participants will be closely monitored throughout the study, which can last up to 8 years.

Eligibility Criteria

Inclusion Criteria

  • Must have a confirmed diagnosis of myelodysplastic syndromes (MDS) or myelodysplastic/myeloproliferative neoplasms (MDS/MPN).
  • Children aged 12-17 for Phase I and adults aged 18 and older for Phase II.
  • Adults with higher-risk MDS must have shown resistance or intolerance to certain standard treatments.
  • Adults with lower-risk MDS must be unable to receive standard treatments and have specific blood count issues.
  • Children with MDS must have relapsed or be unable to receive certain therapies or transplants.
  • Must weigh at least 35 kg (about 77 pounds).
  • Must have completed any previous treatments within specified timeframes before starting the study.
  • Must have adequate organ function and meet specific health criteria.
  • Must agree to use effective contraception during the study and for a period after the last dose.
  • Must be able to understand and sign a consent form, or have a parent/guardian do so for minors.

Exclusion Criteria

  • Cannot have a history of severe bleeding issues or require certain blood-thinning medications.
  • Cannot have had recent severe infections or allergic reactions to similar drugs.
  • Cannot be taking certain medications that interact with pacritinib.
  • Cannot have certain heart conditions or uncontrolled infections.
  • Cannot be pregnant or breastfeeding.
  • Cannot have other untreated conditions that could affect the study results or participant safety.

Get This Program In Your Inbox

Receive detailed information about this treatment opportunity to review with your healthcare provider.