This phase II trial studies how well nivolumab works in treating patients with acute myeloid
leukemia that has decreased or disappeared but may still be in the body (remission), and is
at high risk for returning (relapse). Immunotherapy with monoclonal antibodies, such as
nivolumab, may help the body's immune system attack the cancer, and may interfere with the
ability of tumor cells to grow and spread.
- Brief Title: Nivolumab in Acute Myeloid Leukemia (AML) in Remission at High Risk for Relapse
- Official Title: PD-1 Inhibition With Nivolumab for the Treatment of Patients With Acute Myeloid Leukemia in Remission at High Risk for Relapse
Clinical Trial IDs
- ORG STUDY ID:
- SECONDARY ID:
- NCT ID:
- Acute Myeloid Leukemia
The goal of this clinical research study is to learn if opdivo (nivolumab) can help to
control AML. The safety of nivolumab will also be studied.
This is an investigational study. Nivolumab is FDA approved and commercially available for
the treatment of melanoma. It is considered investigational to use nivolumab to treat AML.
The study doctor can explain how the study drug is designed to work.
Up to 30 participants will be enrolled in this study. All will take part at MD Anderson.
Study Drug Administration:
Each study cycle is 28 days.
If you are found to be eligible to take part in this study, you will receive nivolumab by
vein over about 1 hour on Days 1 and 15 of each cycle. After Cycle 6, you may receive
nivolumab 1 time each month. After you have been on study for about 1 year, you may receive
the study drug 1 time every 3 months. If during this time, the disease appears to get worse,
you may go back to receiving the study drug on Days 1 and 15 of each cycle. The study doctor
will discuss this with you.
On Day 1 of Cycle 1, you will stay in the clinic for up to 2 hours after the end of your
infusion so the study staff can check on your health. After that, you will stay in the clinic
for about 1 hour after the end of your infusion.
On Days 1 and 15 of Cycle 1, one time during Cycles 2 and 3, and then every 8-12 weeks after
that until Cycle 6, you will have a physical exam. If you continue therapy after Cycle 6, you
will have a physical exam every 2-3 months.
One (1) time each week during Cycle 1, every 2 weeks during Cycles 2 and 3, and then 1 time
during each cycle after that, blood (about 2-3 teaspoons) will be drawn for routine tests.
These tests may be done more often if your doctor thinks it is needed.
On Day 28 of Cycles 1, 3, and 5 and then one time every 3 months after that, you will have a
bone marrow aspiration to check the status of the disease and for cytogenetic testing.
Length of Study:
You may continue receiving the study drug for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drug if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.
Your participation on this study will be over after you have completed the End-of-Treatment
Within 30 days after your last dose of study drug:
- You will have a physical exam.
- Blood (about 2-3 teaspoons) will be drawn for routine tests.
- If it has not been done within the last 6 weeks and the doctor thinks it is possible,
you will have a bone marrow aspirate to check the status of the disease and for
If you cannot come to the clinic for the End-of-Treatment visit, you will be called by the
study staff and asked about your health. This call should last about 5-10 minutes.
|Nivolumab||Experimental||Participants receive Nivolumab 3 mg/kg by vein every 2 weeks for 6 cycles. One cycle defined as 4 weeks. Continuation beyond 6 cycles allowed for participants deriving benefit without unacceptable toxicity. The dose after 6 cycles may be modified to an every 4 weeks schedule until 12 months from start of therapy, then every 3 months until disease progression.|
1. Patients with AML in remission (defined as CR, CR with incomplete platelet recovery
-CRp-, CR with incomplete hematologic recovery -CRi-, or partial remission defined as
a bone marrow with <10% blasts after therapy with or without hematologic recovery).
2. High risk for relapse defined as: 1st CR with high risk features for relapse
(including history of prior malignancy treated with chemotherapy or radiotherapy, or
history of myelodysplastic syndrome, myeloproliferative disorder, chronic
myelomonocytic leukemia, MDS/MPN or other hematologic malignancy thought to have
evolved to AML [i.e., secondary AML, sAML]; high risk cytogenetics at diagnosis; FLT3
mutated at diagnosis; or presence or minimal residual disease assessed by PCR,
cytogenetics, and/or flow cytometry at time of enrollment) 2nd CR regardless of
disease characteristics at the time of diagnosis.
3. Have received induction chemotherapy and at least one cycle of consolidation
chemotherapy. Patients should have achieved a CR within 12 months of enrollment onto
4. No further chemotherapy or SCT planned at the time of enrollment.
5. Age 18 years or older.
6. Adequate organ function: creatinine </= 1.5 x ULN; serum bilirubin </= 1.5 x ULN; AST
and ALT </= 2.5 x ULN.
7. ECOG performance status </= 2
8. Females of childbearing potential must have a negative serum or urine beta human
chorionic gonadotrophin (b-hCG) pregnancy test result within 24 hours prior to the
first dose of treatment and must agree to use an effective contraception method during
the study and for 23 weeks after the last dose of the study drug. Females of non-
childbearing potential are those who are postmenopausal greater than 1 year or who
have had a bilateral tubal ligation or hysterectomy.
9. Males who have partners of childbearing potential must agree to use an effective
contraceptive method during the study and for 31 weeks following the last dose of
10. Patients or their legally authorized representative must provide written informed
1. History of another primary invasive malignancy that has not been definitively treated
or in remission for at least 2 years. Patients with non-melanoma skin cancers or with
carcinomas in situ are eligible regardless of the time from diagnosis (including
2. Any major surgery, radiotherapy, chemotherapy, biologic therapy, immunotherapy,
experimental therapy within 2 weeks prior to the first dose of the study drugs.
3. Patients with any other known concurrent severe and/or uncontrolled medical condition
(e.g. uncontrolled diabetes; cardiovascular disease including congestive heart failure
NYHA Class III or IV, myocardial infarction within 6 months, and poorly controlled
hypertension; chronic renal failure; or active uncontrolled infection) which, in the
opinion of the investigator could compromise participation in the study.
4. Patients unwilling or unable to comply with the protocol.
5. Patients who are on steroids (> 10 mg/day or equivalent) or immune suppression
6. Patients with autoimmune diseases (e.g., rheumatoid arthritis, systemic progressive
sclerosis [scleroderma], Systemic Lupus Erythematosus, autoimmune vasculitis [e.g.,
7. Patients with a history of Inflammatory Bowel Disease such as Crohn's disease and
8. Patients known to be positive for hepatitis B surface antigen expression or with
active hepatitis C infection (positive by polymerase chain reaction or on antiviral
therapy for hepatitis C within the last 6 months), or with known HIV infection.
9. Current therapy with other systemic anti-neoplastic or anti-neoplastic investigational
10. Females who are pregnant or lactating
11. Patients with history of previous immunomodulatory therapy.(not including lenalidomide
|Maximum Eligible Age:||N/A|
|Minimum Eligible Age:||18 Years|
Primary Outcome Measures
|Measure:||Recurrence-Free Survival (RFS)|
|Time Frame:||6 months|
|Description:||Status disease determined by bone marrow aspiration. Kaplan-Meier methodology used to compute the RFS rate.|
|Lead Sponsor:||M.D. Anderson Cancer Center|
- Acute myeloid leukemia
- High risk for relapse