Myeloproliferative DiseasesPhase 2NCT02410551
Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)
A Phase 2 study testing Pacritinib, Busulfan and Questionnaires in people with Myeloproliferative Diseases, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myeloproliferative Diseases
- What is being tested?
- Pacritinib, Busulfan and Questionnaires
- Type of treatment
- Pill or oral medicine, Surgery or procedure, Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jun 2015to 20 Jan 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera or Essential Thrombocythemia.
- Patients 18 years to less than or equal to 70 years.
- Patients wanting to pursue transplant.
- Patients must have a Zubrod PS equal or less than 2.
- Calculated creatinine clearance greater than 50ml/min. using the Cockcroft-Gault equation.
- Ejection fraction equal or above 40%.
Show all 10Show fewer
- Serum direct bilirubin less than 1 mg/dl (unless due to Gilbert's syndrome or hemolysis).
- SGPT equal or less than 4 x normal values.
- Corrected DLCO equal or above 50% of expected.
- 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.
Who cannot join
- Patients with low risk myelofibrosis.
- Uncontrolled life-threatening infections.
- HIV positive.
- Patients with active viral hepatitis.
- Prior treatment with Pacritinib.
- Prior stem cell transplant.
- QTc greater than 450 ms.
- CYP3A4 strong or moderate inhibitors/inducers in the past 7 days.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: 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.
Read the 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.
- Conditions
- Myeloproliferative Diseases
- Being tested
- Pacritinib, Busulfan, Questionnaires, Phone Calls, Allogeneic Stem Cell Transplantation
- Keywords
- Myeloproliferative Diseases, Myeloproliferative Neoplasms, MPN, Myelofibrosis, Polycythemia vera, PV, Essential thrombocythemia, Allogeneic stem cell transplantation, Allo TP, Pacritinib, Busulfan, Busulfex, Myleran, Questionnaires, Surveys, Phone calls
- Medical subject headings
- Myeloproliferative Disorders, Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PacritinibDrug
200 mg by mouth twice a day for 60 days.
BusulfanDrug
Also called Busulfex, Myleran
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.
QuestionnairesBehavioural
Also called Surveys
Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.
Phone CallsBehavioural
Phone calls made by study staff to participant on second and third week of each month.
Allogeneic Stem Cell TransplantationProcedure or surgery
Also called Stem cell transplant
Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.
FludarabineDrug
Also called Fludarabine phosphate, Fludara
Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).
| Study group | Role | What they receive |
|---|---|---|
| Pacritinib + Allogeneic Stem Cell Transplantation | Experimental | 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. Receives: Drug: Pacritinib; Drug: Busulfan; Behavioral: Questionnaires; Behavioral: Phone Calls; Procedure: Allogeneic Stem Cell Transplantation; Drug: Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2015 (estimated)
- Study start15 Jun 2015
- Primary completion20 Jan 2017
- Study completion20 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myeloproliferative DiseasesStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- CTI BioPharma
- Responsible party
- Sponsor
- Investigators
- Uday Popat, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02410551
- Sponsor's protocol ID
- 2014-0786
- Other IDs
- NCI-2015-01123
- Status last verified
- Oct 2018
- First submitted
- 2 Apr 2015
- Last updated
- 30 Oct 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN)
- ClinicalTrials.gov ID
- NCT02410551
- Acronym / sponsor ID
- 2014-0786
- Conditions
- Myeloproliferative Diseases
- Keywords
- Myeloproliferative Diseases, Myeloproliferative Neoplasms, MPN, Myelofibrosis, Polycythemia vera, PV, Essential thrombocythemia, Allogeneic stem cell transplantation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 4 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- CTI BioPharma
- Study start
- 15 Jun 2015 (actual)
- Primary completion
- 20 Jan 2017 (actual)
- Study completion
- 20 Jan 2017 (actual)
- First posted
- 7 Apr 2015 (estimated)
- Results
- Posted 5 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pacritinib + Allogeneic Stem Cell Transplantation | Experimental | Drug: Pacritinib, Drug: Busulfan, Behavioral: Questionnaires, Behavioral: Phone Calls, Procedure: Allogeneic Stem Cell Transplantation, Drug: FludarabineParticipants 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera or Essential Thrombocythemia.
- Patients 18 years to less than or equal to 70 years.
- Patients wanting to pursue transplant.
- Patients must have a Zubrod PS equal or less than 2.
- Calculated creatinine clearance greater than 50ml/min. using the Cockcroft-Gault equation.
Key exclusion criteria (8 listed)
- Patients with low risk myelofibrosis.
- Uncontrolled life-threatening infections.
- HIV positive.
- Patients with active viral hepatitis.
- Prior treatment with Pacritinib.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pacritinib200 mg by mouth twice a day for 60 days. | Drug | Pacritinib + Allogeneic Stem Cell Transplantation |
| BusulfanAlso: Busulfex, MyleranBusulfan 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. | Drug | Pacritinib + Allogeneic Stem Cell Transplantation |
| QuestionnairesAlso: SurveysQuestionnaires completed at baseline, 1, 3, 6, and 12 months after transplant. | Behavioural | Pacritinib + Allogeneic Stem Cell Transplantation |
| Phone CallsPhone calls made by study staff to participant on second and third week of each month. | Behavioural | Pacritinib + Allogeneic Stem Cell Transplantation |
| Allogeneic Stem Cell TransplantationAlso: Stem cell transplantAllogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days. | Procedure or surgery | Pacritinib + Allogeneic Stem Cell Transplantation |
| FludarabineAlso: Fludarabine phosphate, FludaraFludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP). | Drug | Pacritinib + Allogeneic Stem Cell Transplantation |
Primary outcome
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Uday Popat, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pacritinib, Busulfan and Questionnaires in people with Myeloproliferative Diseases, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 30 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Oct 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2018.