A First-in-Human Phase 1 and Expanded Cohort Study of EPZ-5676 in Advanced Hematologic Malignancies, Including Acute Leukemia With Rearrangement of the MLL Gene
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 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.
- Scottsdale/Phoenix, Arizona: 13-003437
NCT ID: NCT01684150
Sponsor Protocol Number: EPZ-5676-12-001
About this study
The purpose of this study is to determine the safe dose of EPZ-5676, to evaluate the safety of EPZ-5676 in patients with advanced hematologic malignancies, and to conduct a preliminary assessment of the anti-leukemia activity of EPZ-5676 in patients with acute leukemias bearing rearrangements of the MLL gene.
Currently this study is in the MLL-r restricted/expansion phase and is only enrolling patients with rearrangements involving the MLL gene, including 11q23 or partial tandem duplications (PTD).
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
- Male and female patients aged ≥ 18 years.
- Patients with relapsed /refractory AML, ALL, or MLL with rearrangement of the MLL gene, including 11q23 or PTD, are eligible for the expanded cohort:
- At least one prior therapy;
- Refractory disease on most recent therapy, or disease recurrence following remission on most recent therapy;
- Received and failed all known effective therapies for their disease;
- Not a candidate for allogeneic stem cell transplantation
- > 10% blasts or biopsy-documented leukemia cutis or myeloid sarcoma.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Patients must have the following clinical laboratory values:
- Serum creatinine ≤2 mg/dL or creatinine clearance > 60 mL/minute;
- Total bilirubin ≤2.0 times the ULN for the institution, unless considered due to Gilbert's syndrome;
- ALT or AST ≤ twice the upper limit of normal (ULN), unless considered due to organ leukemic involvement;
- Absolute neutrophil count ≥1,000/µL (unless due to documented leukemic involvement of the bone marrow at the time of study entry)
- Platelets ≥100,000/µL (unless due to documented leukemic involvement of the bone marrow at the time of study entry).
- PT or aPTT < 1.5 times the ULN
- Able and willing to give written informed consent.
- Life expectancy of at least 3 months
- Uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements.
- Active heart disease
- Receiving any other standard treatment for their hematologic malignancy.
- Receiving strong CYP3A4 inhibitors/ inducers.
- Known history of cerebrovascular accident in the past 6 months.
- Known bleeding diathesis.
- Known, active (symptomatic) involvement of the central nervous system by leukemia.
- On immunosuppressive therapy.
- Known active infection.
- Pregnant or nursing females.
Participating Mayo Clinic locations
Study statuses change often. Please contact us for help.
|Mayo Clinic Location
Mayo Clinic principal investigator
Raoul Tibes, M.D., Ph.D.
Contact us for the latest status
Cancer Center Clinical Trials Referral Office