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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2015-01123 | Registry Identifier | NCI CTRP |
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| Name | Class |
|---|---|
| CTI BioPharma | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pacritinib | Drug | 200 mg by mouth twice a day for 60 days. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | Start of Pacritinib to one year post transplant |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Uday Popat, MD | M.D. Anderson Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Texas MD Anderson Cancer Center | Houston | Texas | 77030 | United States |
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| Label | URL |
|---|---|
| University of Texas MD Anderson Cancer Center Website | View source |
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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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Pacritinib Pre- Transplant | Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than 180 days |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Pacritinib Pre- Transplant | Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Four participants were not evaluable and no analysis was done. | Posted | 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. |
|
07/31/2015 to 02/25/2016
The severity of the adverse events (AEs) will be graded according to the Common Terminology Criteria v4.0 (CTCAE). We will not capture expected Grade 1 and Grade 2 AEs. Grade 3 and 4 AEs will be collected and recorded in the medical record. Only Pacritinib-related adverse events and protocol specific data will be entered into PDMS/CORe. PDMS/CORE will be used as the electronic case report form for this protocol.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pacritinib Pre- Transplant | Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rash maculo-papular | Skin and subcutaneous tissue disorders | CTCAE (4.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Popat,Uday,M.D. / Stem Cell Transplantation | UT MD Anderson Cancer Center | 713-792-8750 | upopat@mdanderson.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jan 14, 2016 | Oct 3, 2018 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D009196 | Myeloproliferative Disorders |
| D055728 | Primary Myelofibrosis |
| D011087 | Polycythemia Vera |
| D013920 | Thrombocythemia, Essential |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D019046 | Bone Marrow Neoplasms |
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| ID | Term |
|---|---|
| C561234 | 11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene |
| D002066 | Busulfan |
| D011795 | Surveys and Questionnaires |
| D033581 | Stem Cell Transplantation |
| C024352 | fludarabine |
| C042382 | fludarabine phosphate |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
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| Busulfan | 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 | Behavioral | Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant. |
|
|
| Phone Calls | Behavioral | Phone calls made by study staff to participant on second and third week of each month. |
|
| Allogeneic Stem Cell Transplantation | Procedure | Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days. |
|
|
| Fludarabine | Drug | Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP). |
|
|
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants | No |
|
| Units | Counts |
|---|---|
| Participants |
|
| Other Pre-specified | 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. | Four participants was invaluable and no analysis was done. | Posted | Start of Pacritinib to one year post transplant |
|
|
| 0 |
| 4 |
| 0 |
| 4 |
| 1 |
| 4 |
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| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D001778 | Blood Coagulation Disorders |
| D013922 | Thrombocytosis |
| D001791 | Blood Platelet Disorders |
| D006474 | Hemorrhagic Disorders |
| D008698 |
| Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D003625 | Data Collection |
| D004812 | Epidemiologic Methods |
| D008919 | Investigative Techniques |
| D017531 | Health Care Evaluation Mechanisms |
| D011787 | Quality of Health Care |
| D017530 | Health Care Quality, Access, and Evaluation |
| D011634 | Public Health |
| D004778 | Environment and Public Health |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |