Pacritinib for Myelodysplastic Syndrome

Phase 1 & 2

Waitlist Available

Led By Alain Mina, M.D.

Research Sponsored by National Cancer Institute (NCI)

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after every cycle (c4d1, c7d1, c10d1, c16d1) and yearly after

Awards & highlights

No Placebo-Only Group

Summary

"This trial aims to test a new drug, pacritinib, in adults and children with Myelodysplastic syndrome (MDS) or Myelodysplastic/myelop

Who is the study for?

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.

What is being tested?

The trial is testing pacritinib, a capsule taken orally twice daily in 28-day cycles. It's aimed at treating MDS and MDS/MPN by inhibiting certain enzymes that may affect these conditions. Dosages will vary among participants who are monitored over time through clinic visits and repeated tests.

What are the potential side effects?

While the specific side effects of pacritinib aren't listed here, kinase inhibitors like it can commonly cause fatigue, nausea, diarrhea, muscle pain, rash or changes in blood pressure. Side effects depend on individual reactions to the medication.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after every cycle (c4d1, c7d1, c10d1, c16d1) and yearly after

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after every cycle (c4d1, c7d1, c10d1, c16d1) and yearly after

This trial's timeline: 3 weeks for screening, Varies for treatment, and after every cycle (c4d1, c7d1, c10d1, c16d1) and yearly after for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Efficacy as measured by Objective Response Rate (ORR) per each risk-based cohort

Recommended phase 2 dose of pacritinib in participants 12-17 years of age

Secondary study objectives

Extended safety of pacritinib

Pharmacokinetic (PK) properties of pacritinib

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: 2/Phase II - AdultExperimental Treatment1 Intervention

Low-risk cohort (cohort 2): Initiated on pacritinib 100 mg BID. After three cycles, pacritinib dose escalated to 200 mg BID, the adult phase II recommended dose, if not in complete remission.High-risk cohort (cohort 3): Initiated on pacritnib 200 mg BID, the adult phase II recommended dose.

Group II: 1/Phase I - PediatricExperimental Treatment1 Intervention

Escalating doses of pacritinib (cohort 1)

Do I qualify?Apply below to find out