Trial of Eltrombopag During Consolidation Therapy in Adults With AML in Complete Remission
Study type: Interventional What is this?
Describes the nature of a clinical study. Types include:
- Observational study — observes people and measures outcomes without affecting results.
- Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
- Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
Study phase: I/II What is this?
During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.
- Rochester, Minnesota: 13-003066
NCT ID: NCT01656252
Sponsor Protocol Number: PrE0901
About this study
Patients with Acute Myeloid Leukemia (AML) in complete remission will receive eltrombopag while undergoing consolidation chemotherapy with high-dose cytarabine. Eltrombopag may help increase the number of platelets during chemotherapy and may help prevent the risk of bleeding.
Phase I will study the side effects, best dose and platelet effects of eltrombopag when given with consolidation chemotherapy. After the maximum safe and tolerated dose and schedule is found in Phase I, the study will proceed to Phase II. Phase II will confirm the dose and schedule of eltrombopag identified in Phase I that can increase platelet counts in patients receiving consolidation therapy.
Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.
See eligibility criteria
- Cytomorphologically documented diagnosis of acute myeloid leukemia (AML). Acute promyelocytic leukemia patients will be excluded (FAB M3). FAB classification, cytogenetics and molecular markers (if applicable) must be available at registration.
Phase I Enrollment:
- Must be in first or second complete remission, e.g., no evidence of active disease in blood, bone marrow (<5% blasts), or other tissues.
- For each remission, may have received no more than 2 cycles of induction treatment (any type).
- May have received no more than one course of consolidation for the current remission prior to enrollment (any type)
Phase II Enrollment:
- Must be in first complete remission, e.g., no evidence of active disease in blood, bone marrow (<5% blasts), or other tissues.
- May have received no more than 2 cycles of induction treatment (any type).
Enrollment in Either Phase:
- Remission status must be documented by a bone marrow examination up to 28 days prior to study registration.
- Have recovered from induction and first consolidation (if applicable) therapy side effects (or ≤grade 1).
- ≥18 years of age and ≤70 years of age.
- ECOG performance status 0, 1, 2.
- Have not received cytotoxic drug therapy within 21 days of registration.
- Have not received hematopoietic colony stimulating growth factors within 14 days of registration.
- Have not received packed red blood cells or platelets within 7 days of registration.
- Have not received investigational agents within 30 days of registration and will not receive any investigational agents other than eltrombopag/placebo during study.
- Signed IRB-approved informed consent.
- Willing to provide blood samples for research purposes.
- Adequate organ function obtained within 28 days prior to registration:
- Absolute neutrophil count >1 x 10⁹/L
- Platelet count >100 x 10⁹/L
- Total direct serum bilirubin ≤1.5x upper limit of normal (ULN)
- ALT and AST ≤3x ULN
- BUN and serum creatinine <2x ULN
- Albumin ≥2.5 g/dL
- PT and PTT 80-120% of institutional normal range
- Women of childbearing potential must have a negative serum pregnancy test within 14 days of registration.
- Not pregnant nor breast feeding.
- Women of childbearing potential and sexually active males must use an accepted and effective method of contraception.
- Patients of known East Asian ancestry (Chinese, Japanese, Taiwanese, and Korean) are excluded from protocol participation for safety and efficacy reasons.
- Able to swallow and retain orally administered medication.
- No clinically significant gastrointestinal abnormalities such as malabsorption syndrome or major resection of the stomach or bowels.
- No clinical evidence of hepatomegaly or splenomegaly.
- No known risk for Torsades de Pointes. (Eltrombopag use has not been shown to be associated with Torsades de Pointes.)
- No active or unresolved infection and must be off all antibiotics for at least 7 days prior to registration.
- No current evidence of invasive fungal infection.
- No known Hepatitis B, Hepatitis C active disease.
- No known Human Immunodeficiency Virus (HIV) seropositivity. The risk for potential toxicities secondary to HIV (e.g., increased risk for fatal opportunistic infection) may confound the toxicity profile of eltrombopag.
- Patients with a history of Central Nervous System (CNS) leukemia are eligible if there is documentation of no current CNS involvement on cerebrospinal fluid (CSF) examination (e.g., negative CSF by lumbar puncture) within 28 days of registration.
- No prior or concomitant malignancy in the past 5 years which is currently active and likely to interfere with the patient's treatment for AML or which is likely to increase the patient's morbidity or mortality.
- No prior chemotherapy or radiation therapy allowed (unless related to AML treatment).
- No concurrent organ damage or medical problems that would prohibit therapy.
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.
|Mayo Clinic Location
Mayo Clinic principal investigator
Mark Litzow, M.D.
Open for enrollment
Cancer Center Clinical Trials Referral Office