This is a single-arm, single-site run-in phase (six subjects) followed by a multicenter
continuation phase (114 subjects), Phase IV study. Eligible subjects will be receiving
CPX-351 as their usual medical care administered according to FDA approval, as a condition
This is an open-label, non-randomized study of a non-drug intervention in subjects receiving
CPX-351 as their usual medical care administered according to its FDA approval. The study
intervention is the application of a prescribed outpatient care model, including a nurse
teacher educational program and quality of life surveys for both subjects and caregivers, in
subjects who meet medical and logistical criteria. Induction therapy and medical follow-up
are performed without prophylactic admission to an inpatient facility.
1. Subjects with newly diagnosed acute myeloid leukemia who are eligible for treatment
with CPX-351 therapy according to the FDA approval and indication: therapy-related AML
(t-AML) or AML with myelodysplasia-related changes (AML-MRC).
2. Age ≥ 18 years
3. Eastern Cooperative Oncology Group (ECOG) performance score of 0 or 1 (or Karnofsky
Performance Status equivalent of 70 or above).
4. Total bilirubin ≤ 3 mg/dL.
5. Creatinine clearance > 30 mL/min by Cockcroft/Gault equation.
6. Adequate cardiac function, as assessed by ejection fraction( ≥50% for anthracycline
therapy) via multiple-gated acquisition (MUGA) or echocardiogram.
7. Total white blood cell count of ≤ 20,000/µL on peripheral blood assessment (hydrea
and/or leukapheresis allowed).
8. No evidence of active, uncontrolled infection.
9. No evidence of clinically significant disseminated intervascular coagulation (DIC)
10. No clinically significant abnormalities in core vital signs like heart rate, blood
pressure or oxygenation which require inpatient evaluation or monitoring.
11. In the opinion of the enrolling physician, the subject is not at risk for clinically
significant tumor lysis syndrome based on clinical assessment, complete blood count
(CBC), comprehensive metabolic panel (CMP), uric acid, and lactate dehydrogenase
12. In the opinion of the enrolling physician, no medical conditions that preclude the
subject or the primary caregiver from transportation to and from the outpatient
clinical care facility.
13. Both subject and the identified primary caregiver(s) signed informed consent.
14. In the opinion of the enrolling physician, there are no medical contraindications to
outpatient induction and management.
15. Male subjects, even if surgically sterilized (i.e., status post vasectomy), must agree
to one of the followings:
- Practice effective barrier contraception during the entire study period and
through six months after the last dose of CPX-351, OR
- Agree to practice true abstinence when this is in line with the preferred and
usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation,
symptothermal, postovulation methods] and withdrawal are not acceptable methods
Female subjects must meet one of the followings:
- Postmenopausal for at least one year before enrollment, OR
- Surgically sterile (i.e., undergone a hysterectomy or bilateral oophorectomy), OR
- If the subject is of childbearing potential (defined as not satisfying either of the
above two criteria), she must agree to practice two acceptable methods of
contraception (combination methods require use of two of the following: diaphragm with
spermicide, cervical cap with spermicide, contraceptive sponge, male or female condom,
hormonal contraceptive) from the time of signing of the informed consent form through
six months after the last dose of CPX-351, OR o Agree to practice true abstinence when
this is in line with the preferred and usual lifestyle of the subject. (Periodic
abstinence [e.g., calendar, ovulation, symptothermal, postovulation methods] and
withdrawal are not acceptable contraception methods.)
Logistical Inclusion Criteria
1. The subject must be able to reside within approximately 45 minutes of the hospital
where induction therapy is administered, during normal driving conditions (in the
opinion of the enrolling physician), until count recovery or 60 days post-treatment,
whichever comes first
2. The enrolling physician must verify and attest that the subject has a primary
caregiver meeting all the following criteria:
- Reside with the subject.
- Be able to care for the subject full time or arrange to share full-time care with
- Provide transportation.
- Respond to clinical issues that arise.
- Communicate with subject and physician in a timely manner.
3. The nurse teacher must verify that the subject and primary caregiver have completed
and adequately understand the study-dictated nurse teacher educational program.
4. The nurse teacher must verify the subject and primary caregiver have capacity to
comply with outpatient management program.
5. The subject must have reliable, working telephone access.
6. The subject must be willing and able to attend all protocol-dictated visits and be
seen frequently as an outpatient at the clinical care facility where induction therapy
1. Pregnant women (per pregnancy test in women of childbearing potential) or women who
2. Subjects currently receiving any investigational agents.
3. Subjects must not have current evidence of another malignancy that requires treatment.
4. Subjects diagnosed with Wilson's disease and/or copper-related metabolic disorders.