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

Phase 1

Not yet recruiting

• Conditions: Myelodysplastic Syndromes
• Interventions: Drug: pacritinib

• Registration Number: NCT06303193
• Lead Sponsor: National Cancer Institute (NCI)

Brief Summary

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:
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Detailed Description

Background:

-Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal hematopoietic disorders characterized by peripheral cytopenia, ineffective and dysplastic hematopoiesis, and increased risk of progression to acute myeloid leukemia (AML).

-The significant genetic heterogeneity of MDS contributes to the challenges in treatment.
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Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2024-03-08
• Study First Submitted QC: 2024-03-08
• Study First Posted: 2024-03-12
• Status Verified: 2024-11-20
• Last Update Submitted: 2024-12-19
• Last Update Posted: 2024-12-20
• Study Start: 2024-12-25
• Primary Completion: 2030-01-01
• Study Completion: 2035-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 160

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
2/Phase II - Adult	pacritinib	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.
1/Phase I - Pediatric	pacritinib	Escalating doses of pacritinib (cohort 1)

Primary Outcome Measures

Name	Time	Method
Recommended phase 2 dose of pacritinib in participants 12-17 years of age	28 days	Determined by the number of participants with dose-limiting toxicities (DLTs) reported at each dose level studied
Efficacy as measured by Objective Response Rate (ORR) per each risk-based cohort	After every cycle (C4D1, C7D1, C10D1, C16D1) and yearly after	Clinical responses evaluated by CBC and bone marrow biopsy/aspirate reported separately by cohort, with a separate 95% confidence interval for each cohort

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic (PK) properties of pacritinib	28 days	AUC, half-life, and steady state concentration of pactritinib evaluated separately by dose level using descriptive statistics
Extended safety of pacritinib	Day 1 of study drug through 30 days after the last intervention	Grades and types of toxicity noted for the agent at each dose level, separately by cohort

Trial Locations

Locations (1)

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States