Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)

October 4, 2018 updated by: M.D. Anderson Cancer Center

Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN)

The goal of this clinical research study is to learn if giving pacritinib before standard of care drugs followed by an allogeneic stem cell transplant can help to control myeloproliferative neoplasms. The safety of this therapy will also be studied.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

Study Drug Administration:

If you are found to be eligible to take part in this study, you will take pacritinib at about the same time each day by mouth, 2 times each day. Your doctor will tell you when to start and stop taking pacritinib. You may be able to take the drug for about 2-6 months depending on how you tolerate the drug and when your transplant date is. If you do not receive your transplant, you may be able to continue taking the study drug as long as the doctor thinks it is in your best interest.

You must swallow the capsules whole with a glass (about 8 ounces) of water. Do not open, break, or chew the capsules.

If you vomit or miss a dose of pacritinib, take your next dose of pacritinib at your regular time. Do not "make up" a missed or vomited dose.

You will be given a study drug diary to write down what time you take each dose of pacritinib. You need to bring the study drug diary, any leftover study drug, and any empty study drug containers with you to each study visit.

The dose of pacritinib you receive may be lowered or stopped, if the doctor thinks it is needed.

About 21 days after your last dose of pacritinib, you will given standard of care drugs and you will have an allogeneic stem cell transplant. Your doctor will explain this treatment and the stem cell transplant to you in more detail. You will be required to sign a separate consent form.

Study Visits:

One (1) time each month:

  • You will have a physical exam.
  • Blood (about 2 teaspoons) will be drawn for routine tests and to check your kidney and liver function.
  • You will have an electrocardiogram (EKG -- a test that measures the electrical activity of the heart).

On Day 14 (+/- 2 days) of of Cycle 1, blood (about 2 teaspoons) will be drawn for routine tests and to check your kidney and liver function. You can have this blood drawn at a local lab or clinic that is closer to your home. The results will be sent to the study doctor at MD Anderson.

During Week 2 of Cycle 1, a member of the study staff will call to ask you about any symptoms you may be having. This call should last about 5-10 minutes.

Length of Study:

You will be on study for up to 1 year after the transplant. You may be taken off study early if the disease gets worse, if you have any intolerable side effects, of if you are unable to follow study directions.

Your participation on this study will be over after about 1 year of follow-up tests.

End-of-Study Visit:

Within about 7 days after your last dose of pacritinib, but before your stem cell transplant:

  • You will have a physical exam and an ultrasound, MRI, or CT scan of your abdomen to measure your liver and spleen.
  • You will have an EKG.

Before your transplant, you will have a bone marrow biopsy/aspiration to check the status of the disease.

Follow-Up Tests:

You will have follow-up visits at about 1, 3, 6, and 12 months after the transplant:

  • You will complete 3 questionnaires about your symptoms and quality of life. It should take about 20-30 minutes to complete the questionnaires.
  • At Month 3, you will have a physical exam and an ultrasound, MRI, or CT scan of your abdomen to measure your liver and spleen. This will be repeated at Month 12, if your doctor thinks it is needed.
  • At Months 3 and 12, you will have a bone marrow biopsy/aspiration to check the status of the disease.

This is an investigational study. Pacritinib is not FDA approved or commercially available. It is currently being used for research purposes only. The study doctor can explain how the study drug is designed to work.

Up to 40 participants will be enrolled in this study. All will take part at MD Anderson.

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas MD Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 68 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera or Essential Thrombocythemia.
  2. Patients 18 years to less than or equal to 70 years.
  3. Patients wanting to pursue transplant.
  4. Patients must have a Zubrod PS equal or less than 2.
  5. Calculated creatinine clearance greater than 50ml/min. using the Cockcroft-Gault equation.
  6. Ejection fraction equal or above 40%.
  7. Serum direct bilirubin less than 1 mg/dl (unless due to Gilbert's syndrome or hemolysis).
  8. SGPT equal or less than 4 x normal values.
  9. Corrected DLCO equal or above 50% of expected.
  10. Negative Beta HCG test in a woman with childbearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization) and if fertile, males and females must agree to use contraceptives.

Exclusion Criteria:

  1. Patients with low risk myelofibrosis.
  2. Uncontrolled life-threatening infections.
  3. HIV positive.
  4. Patients with active viral hepatitis.
  5. Prior treatment with Pacritinib.
  6. Prior stem cell transplant.
  7. QTc greater than 450 ms.
  8. CYP3A4 strong or moderate inhibitors/inducers in the past 7 days.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Pacritinib + Allogeneic Stem Cell Transplantation
Participants start Pacritinib 200 mg by mouth twice a day. Participants proceed to transplant after 60 days of Pacritinib but not more than 180 days. Pacritinib stopped 21 days prior to starting preparative regimen for standard of care stem cell transplantation (SOC Allo TP). SOC transplant conditioning with Fludarabine and Busulfan AUC of 4000 microMol-min per day providing that pharmacokinetic can be done, otherwise Busulfan dose given as a fixed dose of 100 mg/m2 daily for four days. Questionnaires about symptoms and quality of life completed at baseline, 1, 3, 6, and 12 months after transplant. Phone calls made by study staff to participant on second and third week of each month.
Drug: Pacritinib
200 mg by mouth twice a day for 60 days.
Drug: Busulfan
Busulfan AUC of 4000 microMol-min per day providing that pharmacokinetic can be done, otherwise Busulfan dose given as a fixed dose of 100 mg/m2 daily for four days.
Other Names:
  • Busulfex
  • Myleran
Behavioral: Questionnaires
Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.
Other Names:
  • Surveys
Behavioral: Phone Calls
Phone calls made by study staff to participant on second and third week of each month.
Procedure: Allogeneic Stem Cell Transplantation
Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.
Other Names:
  • Stem cell transplant
Drug: Fludarabine
Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).
Other Names:
  • Fludara
  • Fludarabine phosphate

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression-free Survival (PFS)
Time Frame: participants who received Pacritinib for >/= 60 days but less than 180 days who undergo transplant with a matched related or at least 7/8 matched unrelated donor. The protocol was to evaluate progression free survival at one year.
The protocol was to enroll at least 21 evaluable participants, defined as patients who received Pacritinib for >/=60 days but less than 180 days. We enrolled four participants, however all four were not evaluable since no one was able to complete 60 days of Pacritinib.
participants who received Pacritinib for >/= 60 days but less than 180 days who undergo transplant with a matched related or at least 7/8 matched unrelated donor. The protocol was to evaluate progression free survival at one year.

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate Safety and Efficacy of Pacritinib.
Time Frame: Start of Pacritinib to one year post transplant
Evaluate safety and efficacy of this therapy determined by Neutrophil and platelet engraftment, Non-relapse mortality at one year post transplant, Overall survival at one year post transplant, Liver and spleen response to Pacritinib, Immune recovery, quality of life and symptom score, Primary and secondary graft failure,Complete remission, Relapse.
Start of Pacritinib to one year post transplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

M.D. Anderson Cancer Center

Collaborators

CTI BioPharma

Investigators

  • Principal Investigator: Uday Popat, MD, M.D. Anderson Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 15, 2015

Primary Completion (Actual)

January 20, 2017

Study Completion (Actual)

January 20, 2017

Study Registration Dates

First Submitted

April 2, 2015

First Submitted That Met QC Criteria

April 2, 2015

First Posted (Estimate)

April 7, 2015

Study Record Updates

Last Update Posted (Actual)

October 30, 2018

Last Update Submitted That Met QC Criteria

October 4, 2018

Last Verified

October 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2014-0786
  • NCI-2015-01123 (Registry Identifier: NCI CTRP)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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