Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02410551|
Recruitment Status : Terminated
First Posted : April 7, 2015
Results First Posted : October 5, 2018
Last Update Posted : October 5, 2018
|Condition or disease||Intervention/treatment||Phase|
|Myeloproliferative Diseases||Drug: Pacritinib Drug: Busulfan Behavioral: Questionnaires Behavioral: Phone Calls Procedure: Allogeneic Stem Cell Transplantation Drug: Fludarabine||Phase 2|
Hide 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.
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.
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.
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 (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN)|
|Actual Study Start Date :||June 15, 2015|
|Actual Primary Completion Date :||January 20, 2017|
|Actual Study Completion Date :||January 20, 2017|
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.
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.
Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.
Other Name: 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 Name: Stem cell transplant
Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).
- 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 are invaluable since no one was able to complete 60 days of Pacritinib.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02410551
|United States, Texas|
|University of Texas MD Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Uday Popat, MD||M.D. Anderson Cancer Center|