Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Phase 1

Recruiting

• Conditions: Graft vs Host Disease
• Interventions: Drug: Pacritinib

• Registration Number: NCT05531786
• Lead Sponsor: National Cancer Institute (NCI)

Brief Summary

Background:

Chronic graft-versus-host disease (cGVHD) is an immune system disorder that can occur in people who have had a stem cell transplant. cGVHD can affect multiple organs and increase risk of disability and death. New treatments are needed to treat cGVHD after stem cell transplant.

Objective:
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Detailed Description

Background:

* Chronic GVHD (cGVHD) is a multi-organ disorder characterized by immune dysregulation, impaired organ function, and decreased survival for hematopoietic stem cell transplantation (HSCT) patients.
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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: 2022-09-03
• Study First Submitted QC: 2022-09-07
• Study First Posted: 2022-09-08
• Study Start: 2023-03-06
• Status Verified: 2024-11-20
• Last Update Submitted: 2024-11-22
• Last Update Posted: 2024-11-25
• Primary Completion: 2025-07-22
• Study Completion: 2025-07-22

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Arm 2 - Low-dose	Pacritinib	Expansion dosing to evaluate the efficacy of pacritinib 100 mg PO BID
Arm 3 - High-dose	Pacritinib	Expansion dosing to evaluate the efficacy of pacritinib 200 mg PO BID
Escalating doses of treatment	Pacritinib	Escalating doses of pacritinib to confirm safety in cGVHD

Primary Outcome Measures

Name	Time	Method
Phase I: Safety of pacritinib in refractory cGVHD.	60 days	grades and types of toxicity reported at each dose level. The overall estimate of the fraction of patients who have a DLT at the MTD will be reported.
Phase II: Overall response rate (ORR)	6 months	the fraction with clinical responses reported separately by arm, with a separate 95% confidence interval for each cohort.

Secondary Outcome Measures

Name	Time	Method
Phase 2: Safety	every 3 months through up to 12 months of treatment	Toxicities identified from day 1 of study drug, collected every 2 weeks through cycle 4, then per cycle through 30 days after the study drug administration. AEs are reported by type and grade
Phase 2: Clinical outcomes	every 3 months through up to 12 months of treatment	Rate of reduction and/or discontinuation of immunosuppressive therapy; response by organ system; time to response; duration of best response; and, failure-free survival
Phase I: Pharmacokinetics (PK)	every 3 months through up to 12 months of treatment	The secondary endpoint for PK properties includes measures such as AUC, half-life, and steady-state concentration.

Trial Locations

Locations (1)

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