Pacritinib for Graft vs Host Disease

Phase-Based Estimates
1
Effectiveness
1
Safety
University of Minnesota, Minneapolis, MN
Graft vs Host Disease+2 More
Pacritinib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Graft vs Host Disease

Study Summary

A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression

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Eligible Conditions

  • Graft vs Host Disease
  • Graft Versus Host Disease (cGvHD)
  • GVHD

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Pacritinib will improve 1 primary outcome and 1 secondary outcome in patients with Graft vs Host Disease. Measurement will happen over the course of up to 21 days.

up to 100 days
Incidence of Acute GVHD
up to 21 days
STAT Activity

Trial Safety

Trial Design

2 Treatment Groups

Control
Pacritinib with Sirolimus and Tacrolimus

This trial requires 43 total participants across 2 different treatment groups

This trial involves 2 different treatments. Pacritinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Pacritinib with Sirolimus and TacrolimusPacritinib added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). Pacritinib will begin the day of the participant's transplant (Day 0) and will continue until 70 days after the transplant. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.
ControlNo treatment in the control group
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
FDA approved
Tacrolimus
FDA approved
Pacritinib
Not yet FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 100 days
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 100 days for reporting.

Closest Location

University of Minnesota - Minneapolis, MN

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Must have an available 8/8 HLA-A, -B, -C, and -DRB1 matched-related or unrelated donor allogeneic hematopoietic peripheral blood stem cell graft.
Signed informed consent.
Acute myeloid leukemia, myelodysplasia, acute lymphoblastic leukemia, chronic myeloid leukemia, chronic lymphocytic leukemia, myeloproliferative neoplasms, Hodgkin lymphoma, or non-Hodgkin lymphoma requiring a matched allogeneic hematopoietic stem cell transplantation (HSCT). Acute Leukemia (AML or ALL) must be in complete remission defined as: <5% marrow blasts with no morphologic evidence of leukemia, no peripheral blasts, marrow >20% cellular, and peripheral absolute neutrophil count >1000/uL (platelet recovery is not required). Myelodysplasia (MDS) and chronic myeloid leukemia (CML): Must have <5% marrow blasts. Myeloproliferative neoplasms (MPN): Must have <5% peripheral / marrow blasts. Note: Prior use of a JAK2 inhibitor is allowed up to 4 weeks before day 0 of alloHCT. Hodgkin and non-Hodgkin lymphoma: Must have chemosensitive disease.
Eligible donors will include healthy sibling, relative or unrelated donors that are matched with the patient at HLA-A, B, C, and DRB1 by high resolution typing as defined by the Collaborative Trials Network.
Adequate vital organ function.
Performance status: Karnofsky Performance Status Score ≥ 80%.

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Can graft vs host disease be cured?

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GVHD is potentially curable. This treatment was found to be a cure at one institution. This treatment entails allogeneic stem cell transplantation followed by myeloablative immunomodulation regimens, followed by a GVL eradication protocol, all culminating in graft vs host disease abrogation. This strategy has been tested in a number of settings and has shown promising results even if not yet proven to be a cure.

Unverified Answer

What causes graft vs host disease?

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The cause of GvHD is thought to be a combination of genetic factors and environmental factors. Both genes in the MHC and HLA loci, or genetic variants in the non-MHC genes, may be important predispositions to GvHD in unrelated transplantations.

Unverified Answer

What are common treatments for graft vs host disease?

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Graft vs host treatment consists largely of medications (e.g., corticosteroid, azathioprine, propranolol, or anti-TNF). In some cases radiation therapy may be suggested. With proper evaluation, GvHD is manageable without serious long-term damage to health.\n

Unverified Answer

What are the signs of graft vs host disease?

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The present study demonstrates a significantly impaired immune reconstitution in patients who have undergone a solid organ transplantation. Therefore, the immune function should be monitored regularly after transplantation but even more prominently in transplantations involving a simultaneous bone marrow and liver transplantation.

Unverified Answer

How many people get graft vs host disease a year in the United States?

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A total of 53,300 new cases of classic and atypical graft vs host disease were recorded in this study. The incidence of this condition is not rising, and the number of deaths from all forms of gvHD is stable. A significant reduction in incidence is suggested by current treatment: the cumulative risk of death is 0.15% for classic gvHD, and 0.08% in atypical gvHD a year, compared with 0.32% in untreated patients. These figures are not statistically significantly different. Current treatment is consistent with good results. The National Inpatient Medication Monitoring System (NIMMS) enables a continuous monitoring of the most common drugs associated with the development of gvHD.

Unverified Answer

What is graft vs host disease?

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AG, GVHD has been documented. Findings from a recent study demonstrated a significant association between increased M2 Macrophage-related genes expression and GVHD. Furthermore an altered TH1/TH2 balance in GVHD as compared with patients who didn't develop it has been demonstrated.

Unverified Answer

How serious can graft vs host disease be?

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Graft vs host disease is a serious and common problem; however, there is no conclusive evidence on the optimal management. It is most frequent in allogeneic transplants and most often occurs within 2 months after transplantation.

Unverified Answer

How does pacritinib work?

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Pacritinib effectively inhibits the growth of MM cells and leukemic cells in cell culture, and suppresses their growth and bone-marrow colonization in a xenograft model, all without toxicity to the host. Such results demonstrate a new therapeutic approach to MM.

Unverified Answer

Is pacritinib safe for people?

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The safety and effectiveness of weekly and continuous infusion pacritinib have been established in people with relapsed/refractory B-cell malignancies. The tolerability of this dose/schedule is comparable with other chemotherapy regimens.

Unverified Answer

What does pacritinib usually treat?

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Pacritinib is not effective as a second-line treatment of children with high-risk FLT3 deletion-positive, relapsed/refractory acute myeloid leukemia, for whom tyrosine kinase inhibitors and other antimetabolites have not achieved long-term responses.

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Has pacritinib proven to be more effective than a placebo?

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Pacritinib has been demonstrated to be more effective than a placebo in terms of improved survival in a randomized phase II trial for relapsed/refractory follicular lymphoma patients with B-cell chronic lymphocytic leukemia.

Unverified Answer

What is pacritinib?

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Pacritinib is a small-molecule phosphatidylinositol 3 kinase (PI3K) inhibitor with a molecular weight of 470 Da (Z = CHCHCH, R = HCOCH). Pacritinib is structurally distinct from wortmannin, the first PI3K inhibitor that was used to understand the function of PI3K signal transduction as well as to develop therapeutics for a variety of cancers. Wortmannin is synthesized from leucine, whereas pacritinib is synthesized from L-Arginine.

Unverified Answer
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