Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)

NCT ID: NCT02410551

Last Updated: 2018-10-30

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-15

Study Completion Date

2017-01-20

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Myeloproliferative Diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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.

Group Type EXPERIMENTAL

Pacritinib

Intervention Type DRUG

200 mg by mouth twice a day for 60 days.

Busulfan

Intervention Type DRUG

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

Intervention Type BEHAVIORAL

Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.

Phone Calls

Intervention Type BEHAVIORAL

Phone calls made by study staff to participant on second and third week of each month.

Allogeneic Stem Cell Transplantation

Intervention Type PROCEDURE

Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.

Fludarabine

Intervention Type DRUG

Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).

Interventions

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Pacritinib

200 mg by mouth twice a day for 60 days.

Intervention Type 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.

Intervention Type DRUG

Questionnaires

Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.

Intervention Type BEHAVIORAL

Phone Calls

Phone calls made by study staff to participant on second and third week of each month.

Intervention Type BEHAVIORAL

Allogeneic Stem Cell Transplantation

Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.

Intervention Type PROCEDURE

Fludarabine

Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).

Intervention Type DRUG

Other Intervention Names

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Busulfex Myleran Surveys Stem cell transplant Fludarabine phosphate Fludara

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CTI BioPharma

INDUSTRY

Sponsor Role collaborator

M.D. Anderson Cancer Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Uday Popat, MD

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

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University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status

Countries

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United States

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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http://www.mdanderson.org

University of Texas MD Anderson Cancer Center Website

Other Identifiers

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NCI-2015-01123

Identifier Type: REGISTRY

Identifier Source: secondary_id

2014-0786

Identifier Type: -

Identifier Source: org_study_id

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