A Phase 2 interventional study of Pacritinib and Busulfan in Myeloproliferative Diseases, sponsored by M.D. Anderson Cancer Center. Terminated at 1 site in United States. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2018-10-30.
Sponsored by M.D. Anderson Cancer Center · Phase 2, Interventional, and Treatment
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 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:
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:
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:
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.
9,365 studies on the registry are indexed under Neoplasms; 2,489 are open to participants now.
This study's enrollment of 4 is below the median of 50 across 7,253 interventional studies indexed under Neoplasms.
Browse Neoplasms studies →M.D. Anderson Cancer Center is the lead sponsor of 2,999 studies on the registry; 581 are open to participants now.
Of its 599 completed or terminated interventional studies of FDA-regulated products, 402 (67%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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 · Drug: Busulfan · Behavioral: Questionnaires · Behavioral: Phone Calls · Procedure: Allogeneic Stem Cell Transplantation · Drug: Fludarabine
200 mg by mouth twice a day for 60 days.
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.
Also known as: Busulfex, Myleran
Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.
Also known as: Surveys
Phone calls made by study staff to participant on second and third week of each month.
Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.
Also known as: Stem cell transplant
Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).
Also known as: Fludarabine phosphate, Fludara
Progression-free Survival (PFS)
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.
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.
Evaluate Safety and Efficacy of Pacritinib.
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.
Time frame: Start of Pacritinib to one year post transplant
Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Ploycythemia Vera or Essential Thrombocythemia that are 18 to 70 years old that want to pursue a transplant. They start the Pacritinib per-transplant and can proceed to transplant 60 days after starting Pacritinib but no more than 180 days.
| Milestone | Pacritinib Pre- Transplant |
|---|---|
| Started | 4 |
| Completed | 0 |
| Not completed | 4 |
| Withdrew: Adverse event | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Fda clinical hold-patients taken off | 2 |
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.
No measurements were reported for this outcome.
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.
No measurements were reported for this outcome.
Collected over 07/31/2015 to 02/25/2016. Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pacritinib Pre- Transplant | 0/4 (0%) | 0/4 (0%) | 1/4 (25%) |
| Event | Pacritinib Pre- Transplant |
|---|---|
| Rash maculo-papularSkin and subcutaneous tissue disorders | 1/4 |
| Age, Categorical(Participants) | Pacritinib Pre- Transplant |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 3 |
| >=65 years | 1 |
| Sex: Female, Male(Participants) | Pacritinib Pre- Transplant |
|---|---|
| Female | 3 |
| Male | 1 |
| Ethnicity (NIH/OMB)(Participants) | Pacritinib Pre- Transplant |
|---|---|
| Hispanic or Latino | 1 |
| Not Hispanic or Latino | 3 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Pacritinib Pre- Transplant |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 4 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(Participants) | Pacritinib Pre- Transplant |
|---|---|
| United States | 4 |
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M.D. Anderson Cancer Center