Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Leukemia

Preleukemia

Myelodysplastic Syndromes

Myeloproliferative Disorders

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Chronic Myeloproliferative Disorders

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Cytarabine

Clofarabine

Antimetabolites

Antiviral Agents

Immunosuppressive Agents

Immunologic Factors

Induction:

Clofarabine 10 mg/m2 1-2 hours by vein daily for 5 days (days 1-5) Cytarabine 20 mg subcutaneously twice daily for 7 days (days 1-7)

Consolidation:

Clofarabine 10 mg/m2 1-2 hours by vein daily for 3 days (days 1-3) Cytarabine 20 mg subcutaneously twice daily for 5 days (days 1-5)

Induction:

10 mg/m2 by vein over 1-2 hours daily for 5 days (days 1-5)

Consolidation:

10 mg/m2 by vein over 1-2 hours daily for 3 days (days 1-3)

Induction:

20 mg subcutaneously twice daily for 7 days (days 1-7)

Consolidation:

20 mg subcutaneously twice daily for 5 days (days 1-5)

Guillermo Garcia-Manero, MD

The goal of this clinical research study is to learn if clofarabine when given in combination with cytarabine can help to control myelodysplastic syndrome (MDS) after the disease could not be controlled with standard therapy. The safety of this treatment will also be studied.

Clofarabine is designed to interfere with the growth and development of cancer cells.

Cytarabine is designed to insert itself into DNA (the genetic material of cells) of cancer cells and stop the DNA from repairing itself.

Induction Cycles:

If you are found to be eligible to take part in the study, on Days 1-5 of each cycle , you will receive clofarabine by vein over 1-2 hours.

On Days 1-7 of each cycle, you will receive cytarabine by injection under the skin over several seconds 2 times a day.

You may receive up to 3 cycles at this dose and schedule (also called "induction cycles"). There are 7 treatment days in each cycle but the total length of one cycle (including rest and recovery period) is usually between 4 and 8 weeks.

Consolidation Cycles:

After you have completed the Induction Cycles, if you show a response to treatment, you can then continue with up to a total of 12 more cycles of therapy, which will be called "consolidation cycles". Not every participant may be able to receive all 12 consolidation cycles. The actual number that you will receive depends on whether or not you maintain the response and how you are able to tolerate ongoing therapy. There will be 4-8 weeks between each consolidation cycle depending on any side effects you may be having and your blood counts.

During consolidation cycles you will receive clofarabine on Days 1-3 by vein over 1-2 hours. You will receive cytarabine by injection under the skin over several seconds 2 times a day .

Induction and Consolidation Cycles:

On the days when you receive clofarabine and cytarabine (Days 1-5 during induction and Days 1-3 during consolidation), the clofarabine will be given about 3-6 hours before the cytarabine injections. You can be taught to give cytarabine injections to yourself. In this case, you can leave the clinic after receiving clofarabine. You will be required to record the injections of cytarabine in a diary unless you receive the treatments while you are in the hospital.

Study Visits:

On Day 1 of every cycle (+/- 7 days):

* You will have a physical exam, including measurements of your weight and vital signs.
* Your performance status will be recorded.
* Blood (about 1-2 teaspoons) will be drawn for routine tests.

About 4 weeks after you started your first cycle, you may have a bone marrow aspirate to check the status of the disease. After that, you may have repeat bone marrow aspirates when the doctor thinks it is needed.

It is recommended that you stay in Houston for up to the first 4 weeks of treatment. After that, you will need to return to Houston before each induction cycle. If you continue with the consolidation you can receive these treatments by your local oncologist. However, you have to return to Houston at least every 3 months for your study visits.

Length of Study:

You may continue taking the study drugs for up to 15 cycles. You will no longer be able to take the study drugs if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

This is an investigational study. Clofarabine is FDA approved and commercially available for use in pediatric patients with acute lymphoblastic leukemia. Its use in adults and in patients with MDS is investigational.

Cytarabine is FDA approved and commercially available for use in patients with acute myeloid leukemia (AML).

Up to 80 patients will take part in this study. All be enrolled at MD Anderson.

Clofarabine Plus Low-Dose Cytarabine for Patients With Higher-Risk Myelodysplastic Syndrome (MDS)

Clofarabine Plus Low-Dose Cytarabine for the Treatment of Patients With Higher-Risk Myelodysplastic Syndrome (MDS) Who Have Been Relapsing After, or Are Refractory to, Hypomethylator Therapy

Complete Response Criteria (CR must last for at least 4 weeks): Marrow: \</= 5% myeloblasts with normal maturation of all cell lines; Persistent dysplasia noted; Blood: Hemoglobin (Hb) \>/= 11 g/dL (untransfused, patient not on EPO); Neutrophils \>/= 1x109/L (not on myeloid growth factor); Platelets \>/= 100 \* 109/L (not on thrombopoietic agent); No blasts. Bone marrow aspirate and/or biopsy at the end of course 1 (day 28 +/- 7 days). The method of Thall, Simon, and Estey used to monitor response.

Overall survival defined as the time interval from study entry date to the date of death due to any cause, measured in days/months. Bayesian time-to-event model used to monitor overall survival.

M.D. Anderson Cancer Center

Induction:

Clofarabine 10 mg/m2 1-2 hours by vein daily for 5 days (days 1-5) Cytarabine 20 mg subcutaneously twice daily for 7 days (days 1-7)

Consolidation:

Clofarabine 10 mg/m2 1-2 hours by vein daily for 3 days (days 1-3) Cytarabine 20 mg subcutaneously twice daily for 5 days (days 1-5)

Clofarabine: Induction:

10 mg/m2 by vein over 1-2 hours daily for 5 days (days 1-5)

Consolidation:

10 mg/m2 by vein over 1-2 hours daily for 3 days (days 1-3)

Cytarabine: Induction:

20 mg subcutaneously twice daily for 7 days (days 1-7)

Consolidation:

20 mg subcutaneously twice daily for 5 days (days 1-5)

Clofarabine + Cytarabine

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Guillermo Garcia-Manero, MD/Professor

One of the eighty-one participants registered on the study were in evaluable for response.

Number of Participants With Complete Response (CR)

Overall Survival (OS)

Overall Study

Heart and Blood Diseases

Polycythemia Vera

Polycythemia

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Bone Marrow Neoplasms

Hematologic Neoplasms

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Essential Thrombocythemia

AZD1480

Srdan Verstovsek, MD

Ronald Hoffman, MD

Vincent Ribrag, MD

Becker Hewes, MD

This study is being conducted to test study drug AZD1480 to see how it may work to treat myeloproliferative diseases. The main purpose of this study is to determine the safety and tolerability of AZD1480. This is the first time the drug has been given to humans and is classed as a first time in man study. Its main purpose is to establish a safe dosage of the drug and provide additional information on any potential side effects this drug may cause. The study will also assess the blood levels and action of AZD1480 in the body over a period of time and will indicate whether the drug has a therapeutic effect on myeloproliferative diseases.

Study to Assess the Safety of AZD1480 in Patients With Myeloproliferative Diseases

A PhaseI/II, Open Label Multi-Centre Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of the JAK2 Inhibitor AZD1480 Administered Orally to Patients With Primary Myelofibrosis (PMF) and Post-Polycythaemia Vera/Essential Thrombocythaemia Myelofibrosis (Post-PV/ET MF

University of Texas

New York City Hoffman Center

Gustave Roussy, Cancer Campus, Grand Paris

AstraZeneca

AZD1480 may be administered orally in capsules

2.5 mg QD

5.0 mg QD

10 mg QD

70 mg QD

15 mg BID

20 mg QD

30 mg QD

50 mg QD

10 mg BID

Total

Dr Gregory Curt, MD

Pharmacokinetic.

Note - no GeoCV(%) was captured in the TFL. Hence the geometric mean was still presented as applicable but with the SD (since only available).

Pharmacokinetic Parameters Following Single Dosing: AUC0-12

Pharmacokinetic.

Note - no GeoCV(%) was captured in the TFL. Hence the geometric mean was still presented as applicable but with the SD (since only available).

From the BID schedule we can not derive the single dosing AUC0_24 and hence this is not presented/calculated.

Pharmacokinetic Parameters Following Single Dosing: AUC0-24

Pharmacokinetic.

Note - no GeoCV(%) was captured in the TFL. Hence the geometric mean was still presented as applicable but with the SD (since only available).

From the BID (twice a day dosing schedules) we can not derive the PK parameter AUC0_inf following single dosing. With that these do not contribute.

Pharmacokinetic Parameters Following Single Dosing:AUC0-inf

Pharmacokinetic Parameters Following Multiple Dosing: Cmax,ss

Pharmacokinetic Parameters Following Multiple Dosing: Cmin,ss

Pharmacokinetic Parameters Following Single Dosing: Cmax

Pharmacokinetic.

Note - no GeoCV(%) was captured in the TFL. Hence the geometric mean was still presented as applicable but with the SD (since only available).

Due to the nature of the dosing schedule this PK parameter was not reported for the BID (twice daily dosing) groups.

Pharmacokinetic Parameters Following Single Dosing: Vz/F

Pharmacokinetic Parameters Following Single Dosing: CL/F

Pharmacokinetic Parameters Following Multiple Dosing: CLss/F

Pharamcokinetic Parameters Following Single Dosing: Tmax

Pharamcokinetic Parameters Following Multiple Dosing: Tmax,ss

2 Hours post dose

4 Hours post dose

Inhibition of PSTAT3 (Count)

Fludarabine

Fludarabine phosphate

Busulfan

Alkylating Agents

Antineoplastic Agents, Alkylating

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.

Pacritinib

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.

Questionnaires

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

Phone Calls

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

Allogeneic Stem Cell Transplantation

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

Uday Popat, MD

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:

* 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.

Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)

Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN)

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.

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.

CTI BioPharma

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.

Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than

Pacritinib Pre- Transplant

Ethnicity (NIH/OMB)

Popat,Uday,M.D. / Stem Cell Transplantation

Four participants were not evaluable and no analysis was done.

Progression-free Survival (PFS)

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

Four participants was invaluable and no analysis was done.

Evaluate Safety and Efficacy of Pacritinib.

Immune System Diseases

Behaviors and Mental Disorders

Lymphoma

Multiple Myeloma

Neoplasms, Plasma Cell

Suicide

Graft vs Host Disease

Hodgkin Disease

Self-Injurious Behavior

Behavioral Symptoms

Lymphosarcoma

Homologous Wasting Disease

Hodgkin Lymphoma

Antirheumatic Agents

Dermatologic Agents

Reproductive Control Agents

Anti-Inflammatory Agents

Antiemetics

Neuroprotective Agents

Gastrointestinal Agents

Micronutrients

Hematinics

Vitamins

Tacrolimus

Methotrexate

Melphalan

Prednisolone

Lenograstim

Methylprednisolone

Methylprednisolone Acetate

Methylprednisolone Hemisuccinate

Prednisolone acetate

Prednisolone hemisuccinate

Prednisolone phosphate

Alemtuzumab

Enzyme Inhibitors

Folic Acid

Vitamin B Complex

Folic Acid Antagonists

Calcineurin Inhibitors

Glucocorticoids

Hormones

Hormone Antagonists

Protective Agents

Antineoplastic Agents, Hormonal

Antineoplastic Agents, Immunological

Folinic Acid

Folate

Vitamin B9

The first component is stem cell transplant. Goal is to administer more than 3 x 106 CD34+ cells/kg of peripheral blood progenitor cells (PBPC). The second component is the planned DLI infusion. iCasp9 (BPZ-1001)-Modified T-cells) of 3 X 10\^6/kg in 100 ml infused over approximately a one hour period between Day + 56 to Day +64. The transplant day is referred to as day zero (D0), treatment plan activities prior or after D0 are denoted as day minus (D-) or day plus (D+). Patients receive standard reduced intensity regimen using fludarabine, melphalan, and alemtuzumab to achieve engraftment with a low risk of GVHD. At approximately 60 days post transplant patients who are alive and without GVHD, receive DLI to enhance graft-vs.-malignancy and immune reconstitution.

Stem cell infusion on Day 0. Goal is to administer more than 3 x 10\^6 CD34+ cells/kg of peripheral blood progenitor cells (PBPC).

Stem Cell infusion

Starting dose of 0.015 mg/kg (ideal body weight) as a 24 hour continuous infusion daily adjusted to achieve a therapeutic level of 5-15 ng/ml (target is 10 ng/ml) between Day +1 and Day +45. Tacrolimus is changed to oral dosing when tolerated. Tacrolimus tapering should start on approximately Day +35 with the intention for the patient to be completely off the drug by approximately Day +45.

Mini Methotrexate

5 mcg/kg/day subcutaneously beginning on Day +7, and continuing until the absolute neutrophil count (ANC) is \> 500 x 10/L for 3 consecutive days.

G-CSF

iCasp9 (BPZ-1001)-Modified T-cells) of 3 X 10\^6/kg in 100 ml infused over approximately a one hour period between Day + 56 to Day +64.

Donor Lymphocyte Infusion (DLI)

0.4 mg/kg as a 2 hour infusion for patients that present with a clinical diagnosis of grade I GvHD. For patients with a clinical diagnosis of grade \> 2 GvHD, a single dose of AP1903 0.4 mg/kg as a 2 hour infusion will be administered. Patients who fail to achieve a complete response within 72 hours of an initial dose of AP1903, or within 48 hours of a second dose of AP1903, will be maintained on this dose for no less than 7 days. Patients who experience a partial response within 72 hours may receive a second dose of AP1903. Patients whose GvHD is progressing after 7 days, have no response by 14 days, or are not in a complete response at day 28 can receive secondary therapy.

AP1903

1.6 mg/kg per day by vein divided in 2 to 3 daily doses. Patients whose GvHD resolves as defined by a complete response within 72 hours, would have steroids stopped immediately. Patients who fail to achieve a complete response within 72 hours of an initial dose of AP1903, or within 48 hours of a second dose of AP1903, will be maintained on this dose for no less than 7 days. Steroids can then be tapered as tolerated to no less than 0.6 mg/kg per day at day 28.

Completion of a quality of life questionnaire that will take 10-15 minutes between Days + 28 and + 56, about twice a week until about 2 months after the T-cell infusion, and then 6 and 12 months after the stem cell transplant.

Questionnaire

Richard E. Champlin, MD, BS

The goal of this clinical research study is to learn if giving genetically changed immune cells, called T-cells, after chemotherapy will improve the response to a stem cell transplant. The safety of this treatment will also be studied.

The process of changing the DNA (the genetic material in cells) of these T-cells is called "gene transfer." Researchers want to learn if these genetically-changed T-cells are effective in attacking cancer cells in patients with leukemia, MDS, lymphoma, Hodgkin disease, or MM, after they have received an allogeneic stem cell transplant.

The chemotherapy you will be given on study is fludarabine, melphalan, and alemtuzumab. These drugs are designed to stop the growth of cancer cells, which may cause the cancer cells to die. This chemotherapy is also designed to block your body's ability to reject the donor's stem cells.

Researchers also want to learn if giving AP1903 will help the symptoms of graft-versus-host disease (GvHD) that may occur after the T-cell infusion. GvHD occurs when donor cells attack the cells of the person receiving the stem cell transplant.

Gene Transfer:

Gene transfer involves drawing blood from a transplant donor, and then separating out the T-cells using a machine. Researchers then perform a gene transfer to change the T-cells' DNA, and then inject the changed T-cells into the body of the patient receiving the transplant.

Study Drug Administration:

You will receive fludarabine, melphalan, and alemtuzumab to kill cancer cells and help prevent your body from rejecting the stem cells. The day you receive the stem cells is called Day 0. The days before you receive your stem cells are called minus days. The days after you receive the stem cells are called plus days.

On Day -7, you will be admitted to the hospital and given fluids by vein to hydrate you.

On Days -6 through -3, you will receive fludarabine by vein over 1 hour each day.

On Day -2, you will receive melphalan by vein over 30 minutes.

On Day -1, you will receive alemtuzumab by vein over 2 hours.

On Day 0, you will receive the stem cell transplant as a cell infusion by vein.

After the transplant, you will receive tacrolimus and methotrexate. At first, you will receive tacrolimus as a continuous (nonstop) infusion until you are able to take it by mouth. You will then take tacrolimus by mouth 2 times a day for about 3 weeks and then your doctor will tell you how to taper it off (gradually stop taking it). On Days +1, +3, +6, and +11, you will receive methotrexate by vein over 30 minutes.

You will receive filgrastim as an injection under the skin 1 time a day, starting 1 week after the transplant, until your blood cell levels return to normal. Filgrastim is designed to help with the growth of white blood cells.

Between Day +56 and +64, if you are in stable medical condition and have not developed GvHD, you will receive a donor lymphocyte infusion containing genetically modified T-cells by vein over 10-30 minutes. You will receive Benadryl (diphenhydramine) by vein over 15 minutes and Tylenol by mouth before the infusion to lower the risk of an allergic reaction.

If your doctor thinks it is needed due to medical problems, the T-cell infusion may be postponed for up to 6 months after the transplant.

If you develop symptoms of GvHD after the T-cell infusion, you must return to the clinic within 72 hours. Most cases of GVHD occur within 60 days of the T-cell infusion. If you have GvHD, you will receive AP1903 by vein and possibly steroids by mouth or by vein. If your doctor thinks it is needed, you may receive one more dose of AP1903 by vein 24-72 hours after the first dose.

If GvHD returns after the first treatment and your doctor thinks it is needed, you may receive AP1903 by vein and steroids by mouth or by vein.

Blood (about 2 tablespoons each time) will be drawn about 3 hours before you receive AP1903, about 2 hours after the AP1903 infusion, and then about 24 hours after the AP1903 infusion to check the level of genetically modified T-cells.

You will then come to the clinic every day for the next 3 days and for an additional 3 days after a second dose of AP1903, if given. In addition, you will come to the clinic on about Days +7, +14, +28, +42 and +56 after receiving AP1903. If your symptoms do not improve after receiving AP1903, you will be given standard drugs for GvHD.

Study Tests:

After the stem cell transplant but before the T-cell infusion, you will have the following tests and procedures to find out if you will be eligible for the T-cell infusion:

* You will have a physical exam.
* You will be checked for possible reactions to treatment, including GvHD.
* Blood (about 4 tablespoons) will be drawn for routine tests, to check your liver and kidney function, for chimerism studies (determination of donor or recipient cells), and to check for cytomegalovirus (CMV).
* If your doctor thinks it is needed, you will have a bone marrow aspiration and biopsy performed to check the status of the disease. To collect a bone marrow aspiration/biopsy, an area of the hip is numbed with anesthetic, and a small amount of bone marrow and bone is withdrawn through a large needle.

About twice a week until about 2 months after the T-cell infusion, and then 6 and 12 months after the stem cell transplant:

* You will have a physical exam, including measurement of your height, weight and vital signs.
* Your medical history will be recorded.
* You will have be checked for possible reactions to your treatment, including GvHD.
* Blood (about 4 tablespoons) will be drawn for routine tests, to check your liver and kidney function, and to check for CMV and other infections. Part of the blood may be used for chimerism studies, if your doctor thinks it is needed.
* If your doctor thinks it is needed, you may have a bone marrow aspiration and biopsy performed to check the status of the disease and for chimerism studies.

If your doctor thinks it is needed, some tests and procedures may be repeated more frequently or at different time points during the study.

Immune System and T-cell Level Tests:

If possible, blood (about 3 tablespoons) will be drawn to check the status of the disease and your immune system function:

* 4 hours after the T-cell infusion,
* once a week for 1 month after the T-cell infusion,
* and then at 6 weeks and then about 2, 3, 6, 9, and 12 months after the T-cell infusion. °Part of the blood sample will be used to check for HAMA at 3 months after the T-cell infusion.

Part of the blood drawn will be tested to check the level and function of the infused T-cells. At 3 months after the T-cell infusion, blood (about 1 teaspoon) will be drawn to check for HAMA.

Questionnaires:

You will complete a quality of life questionnaire that will take about 5-10 minutes each time:

* When you enroll on this study
* At the time of the T-cell infusion
* If you develop symptoms of GvHD after the T-cell infusion and receive AP1903, you will complete the questionnaire before you receive AP1903, and then about 7 and 14 days after you receive AP1903.
* If you do not develop symptoms of GvHD, you will complete a questionnaire about 1 month after the T-cell infusion.

Length of Treatment:

You will be off study after your 1-year follow-up visit. You will be taken off study early if:

* not enough donor cells could be collected
* you were not eligible to receive the T-cell infusion
* you have graft failure (the donor cells did not "take")
* the disease comes back and needs another treatment
* you are unable to keep appointments
* you did not return to the clinic within 72 hours of having symptoms of GvHD
* your study doctor thinks it is in your best interest

If you are taken off study, you will receive standard of care treatment.

Long-Term Follow-Up:

For safety reasons, the U.S. Food and Drug Administration (FDA) requires that patients who receive infusions of stem cells treated with a gene transfer procedure must have long-term follow-up yearly for at least 15 years after receiving the gene transfer.

You will have blood tests performed to check to make sure you do not have a type of infection called the replication-competent retrovirus (RCR). For this test, blood (up to 4 teaspoons each time) will drawn about 1, 3, and 6 months after the T-cell infusion, then once every 6 months for 5 years, and then once a year after that for 10 years.

If the RCR test results during the first year after the T-cell infusion show that you do not have the RCR infection, the rest of your leftover blood samples (left over from RCR testing in Years 2-15) will be stored at Bellicum for safety reasons. This is so researchers can study any changes in your blood (related to RCR) that may arise in Years 2-15.

You will be asked to sign a separate consent form for a long-term follow-up study, Protocol 2006-0676. If for any reason you are unable to receive the genetically modified cells, you will not be enrolled on the long-term follow-up study.

This is an investigational study. The gene transfer or infusion with genetically-changed T-cells and the drug, AP1903, are not FDA approved or commercially available for use in this type of disease. They are currently being used for research purposes only. Fludarabine, melphalan, and alemtuzumab are commercially available and FDA approved.

Up to 35 patients will take part in this study. All will be enrolled at MD Anderson.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Clofarabine Plus Low-Dose Cytarabine for Patients With Higher-Risk Myelodysplastic Syndrome (MDS)	NCT01444742	Leukemia Myeloproliferat...	Clofarabine Cytarabine	18 Years -	M.D. Anderson Cancer Center
Study to Assess the Safety of AZD1480 in Patients With Myeloproliferative Diseases	NCT00910728	Primary Myelofi... Post-Polycythae... Essential Throm...	AZD1480	25 Years - 99 Years	AstraZeneca
Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)	NCT02410551	Myeloproliferat...	Pacritinib Busulfan Questionnaires Phone Calls Allogeneic Stem... Fludarabine	18 Years - 70 Years	M.D. Anderson Cancer Center
Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene	NCT01875237	Leukemia Myeloma Myeloproliferat...	Fludarabine Melphalan Alemtuzumab Stem Cell infus... Tacrolimus Mini Methotrexa... G-CSF Donor Lymphocyt... AP1903 Methylprednisol... Questionnaire	18 Years - 65 Years	M.D. Anderson Cancer Center
Allogeneic Transplantation Using Timed Sequential Busulfan and Fludarabine Conditioning	NCT01572662	Leukemia Acute Myeloid L... Acute Lymphocyt... Chronic Myeloid... Chronic Lymphoc... Myeloproliferat... Non-Hodgkins Ly... Hodgkins Lympho... Multiple Myelom... Myelodysplastic...	Fludarabine mon... Busulfan Stem Cell Infus... Tacrolimus Methotrexate G-CSF	5 Years - 75 Years	M.D. Anderson Cancer Center
Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia	NCT00513175	Acute Myeloid L... Myelodysplasia Acute Lymphobla... Chronic Lymphoc... Follicular Lymp... Multiple Myelom... NHL Myeloproliferat... Chronic Myeloid... Renal Cell Carc... Aplastic Anemia		16 Years - 75 Years	University of California, San Francisco
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Idasanutlin Monotherapy in Participants With Hydroxyurea-Resistant/Intolerant Polycythemia Vera	NCT03287245	Polycythemia Ve...	Idasanutlin	18 Years -	Hoffmann-La Roche

Spots Global Cancer Trial Database for myeloproliferative diseases