Pomalidomide for Relapse/Refractory Waldenstrom's
This trial is active, not recruiting.
|Sponsor||M.D. Anderson Cancer Center|
|Start date||October 2010|
|End date||October 2018|
|Trial size||30 participants|
|Trial identifier||NCT01198067, 2009-0972, NCI-2012-01882|
The goal of this clinical research study is to find the highest tolerable dose of pomalidomide that can be given to patients with relapsed or refractory WM. The safety of this drug will also be studied.
This is an investigational study. Pomalidomide is FDA approved and commercially available for the treatment of certain types of MM. Its use in this study is investigational.
Up to 30 patients will take part in this study. All will be enrolled at MD Anderson.
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Maximum Tolerated Dose (MTD) of Pomalidomide in Participants with Symptomatic Waldenström Macroglobulinemia (WM)
time frame: 28 days
Efficacy of Pomalidomide in Participants with Symptomatic Waldenström Macroglobulinemia (WM)
time frame: 30 days after last dose of study drug given.
Male or female participants at least 18 years old.
Inclusion Criteria: 1. Understand and voluntarily sign an informed consent form 2. Age >/= 18 years at the time of signing the informed consent form 3. Able to adhere to the study visit schedule and other protocol requirements 4. Waldenström's Macroglobulinemia that has relapsed and/or is refractory to at least one prior line of therapy 5. All previous cancer therapy, including radiation, hormonal therapy and surgery, must have been discontinued at least 4 weeks prior to treatment in this study 6. ECOG performance status of = 2 at study entry 7. Laboratory test results within these ranges: Serum creatinine = 2.0 mg/dL; Creatinine clearance >/= 45 ml/min; Total bilirubin = 3 x Upper Limit of Normal (ULN) or Direct Bilirubin = 2 x ULN; AST (SGOT) and ALT (SGPT) = 2 x ULN; Platelet count >/= 20 K/microL; Absolute neutrophil count >/= 500 K/microL 8. Disease free of prior malignancies for >/= 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix or breast 9. Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10 to 14 days prior to and again within 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking pomalidomide 10. Continued from Inclusion #9: FCBP must also agree to ongoing pregnancy testing. Men must agree to practice complete abstinence or agree use a latex condom during sexual contact with a FCBP while participating in the study, during dose interruptions and for at least 90 days following study drug discontinuation, even if they have had a successful vasectomy. All patients must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure 11. Able to take aspirin (325mg) daily as prophylactic anticoagulation (patients intolerant to ASA may use therapeutic dose warfarin or low molecular weight heparin). 12. All study participants must be registered into the mandatory POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program. Exclusion Criteria: 1. Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form 2. Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking pomalidomide or for 28 days after stopping pomalidomide) 3. Any medical or psychiatric condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study, or confounds the ability to interpret data from the study 4. Use of any other experimental drug or therapy within 28 days of the first dose of study drug 5. Known hypersensitivity to thalidomide or lenalidomide 6. The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs 7. Any prior use of pomalidomide 8. Concurrent use of other anti-cancer agents or treatments 9. Known positive for HIV or acute hepatitis A or acute or chronic active hepatitis B or C 10. Grade >2 peripheral neuropathy 11. Neutrophil count <1000K/microL and/or platelet count <100K/microL unless infiltration by Waldenström's Macroglobulinemia equals or exceed 60% of bone marrow cellularity.
|Official title||Phase I Study of Pomalidomide in Relapsed or Refractory Waldenstrom Macroglobulinemia|
|Principal investigator||Sheeba K. Thomas, MD|
|Description||The Study Drug: Pomalidomide is designed to change the immune system, which may interfere with the development of blood vessels that help support tumor growth. This may reduce or stop the growth of cancer cells. Study Groups: If you are found to be eligible to take part in this study, you will be assigned to a dose level of pomalidomide based on when you join this study. Up to 4 dose levels of pomalidomide will be tested. Three (3) participants will be enrolled at each dose level. The first group of participants will receive the lowest dose level. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of pomalidomide is found. Dosing Schedules/Study Drug Administration: You will be enrolled in one of two dosing schedules (Schedule A or Schedule B) based on how previous participants have reacted to the study drug. - If you are in Schedule A, you will take pomalidomide by mouth 1 time each night for 28 days in a row. These 28 days make up 1 "study cycle". - If you are in Schedule B, you will take pomalidomide by mouth 1 time each night for 21 days in a row, followed by a 7-day "rest period". You should take the study drug at about the same time every night. You should take it with a glass of water on an empty stomach. Pomalidomide capsules should be swallowed whole, and should not be broken, chewed, or opened. Pomalidomide should be taken without food, at least 2 hours before or 2 hours after a meal. If a dose of pomalidomide is missed, it should be taken as soon as possible on the same day. If it is missed for the entire day, it should not be made up. Rather, it should be taken at the next scheduled time point. Patients who take more than the prescribed dose of pomalidomide should be instructed to seek emergency medical care if needed and contact study staff immediately. You should bring any unused pills/bottles with you to each study visit. You will also take aspirin every day that you take pomalidomide to help lower the chance of side effects. Study Drug Diary: You will be given a study drug diary at the beginning of each cycle. You should bring the completed diary with you to each study visit. You should write about any missed or vomited doses in your diary. If you miss a dose of pomalidomide, it should be taken as soon as possible on the same day. If you miss a dose for an entire day, do not try to make it up by taking 2 doses the next day. If you vomit a dose of study drug, do not take another dose on the same day or double the next dose. Study Visits: On Day 1 of each Cycle: - You will have a physical exam, including measurement of your vital signs and weight. - You will be asked about side effects you are having and any drugs you may be taking. - Your performance status will be recorded. - Blood (about 1 tablespoon) will be drawn for routine tests. - Blood (about 1 teaspoon) and urine will be collected to check the status of the disease. You will collect urine over a 24-hour period. You will be given a container to collect the urine in. - Blood (about 1 tablespoon) or urine will be taken for pregnancy test for women who are able to have children. On Days 8 and 22 of Cycle 1: - Blood (about 1 tablespoon) or urine will be taken for pregnancy test for women who are able become pregnant. On Day 15 of Cycle 1: - Blood (about 1 tablespoon) will be drawn for routine tests. - You will be asked about any side effects you may be having and any drugs you may be taking. - Blood (about 1 tablespoon) or urine will be taken for pregnancy test for women who are able to become pregnant. On Day 1 of every odd cycle (Cycles 3, 5, 7 and so on): - If your study doctor thinks it is needed, you may have a CT or MRI scan of the brain to check the status of the disease. - You will have an x-ray of your chest. - If your study doctor thinks it is needed, you will have x-rays of your bones to check for any bone lesions you may have. Starting with Cycle 3, you will have the following tests every 4 months for the first 2 years, then once a year as long as you are on the study, and when the study doctor thinks it is needed: - You will have CT or MRI scans of the chest, abdomen, and pelvis to check the status of the disease. If the disease completely responds to the drugs, you will have a bone marrow aspirate and biopsy to confirm complete response. Length of Study: You will be able to take pomalidomide for as long as the doctor thinks it is in your best interest. You will be taken off study if the disease gets worse or you have intolerable side effects. End-of-Treatment (discontinuation from study drug): After you are taken off study, you will have an end-of-treatment visit. The following tests and procedures will be performed: - You will have a physical exam, including measurement of your weight and vital signs. - You will asked about any side effects you may be having and any drugs you may be taking. - Your performance status will be recorded. - You will have an ECG. - Blood (about 1 tablespoon) will be drawn for routine tests. - Blood (about 1 teaspoon) and urine will be collected to check the status of the disease. You will collect urine over a 24-hour period. You will be given a container to collect the urine in. - You will have a CT and/or MRI scan of the chest, abdomen, and pelvis to check the status of the disease. - If your study doctor thinks it is needed, you may have a CT or MRI scan of the brain to check the status of the disease. - You will have an x-ray of your chest. - You will have x-rays of your bones to check for any bone lesions you may have. - Blood (about 1 tablespoon) or urine will be taken for pregnancy test for women who are able to become pregnant. Safety assessment Visit After you are taken off study, you will have a safety assessment visit 30 days after your last dose of the study drug. The following tests and procedures will be performed: You will have a physical exam, including measurement of your weight and vital signs. Your performance status will be recorded. Blood (about 1 tablespoon) will be drawn for routine tests. You will be asked about side effects you may be having. Follow-up Phone Call The study staff will call you every 6 months for 1 year to ask about any new treatments you may have started. The phone call should take about 5 minutes.|
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