Clinical trial NCT04269213: CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years Old
Official title: A Phase II Study of CPX-351 in Younger Patients &lt; 60 Years Old With Secondary Acute Myeloid Leukemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Acute Myeloid Leukemia With Myelodysplasia-Related Changes
Secondary Acute Myeloid Leukemia
Therapy-Related Acute Myeloid Leukemia
Lead sponsor: Roswell Park Cancer Institute
Sponsor class: OTHER
Collaborators: name: Jazz Pharmaceuticals
First posted: 2020-02-13
Enrollment: 46
Sex: ALL
Minimum age (years): 18
Maximum age (years): 59
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine (CPX-351) works in treating patients with secondary acute myeloid leukemia who are younger than 60 years old. Drugs used in chemotherapy, such as CPX-351, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Detailed description: PRIMARY OBJECTIVE:

I. To determine the complete response rate including morphologic complete remission (CR) and morphologic complete remission with incomplete blood count recovery (CRi) as defined by the International Working Group Criteria.

SECONDARY OBJECTIVE:

I. To determine CR + CRi duration, event free survival (EFS), overall survival (OS), patients successfully proceeding to allogenic hematopoietic cell transplant, and adverse events (AE).

OUTLINE:

INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity.

RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.

CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days, and then every 3 months for up to 5 years.

Eligibility criteria: Inclusion Criteria:

* Newly diagnosed:

  * Therapy-related acute myeloid leukemia (AML)
  * AML with antecedent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML)
  * AML with MDS-related changes (as per World Health Organization \[WHO\])
* Have an Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2
* Plasma creatinine =\< 1.5 x upper limit of normal (ULN)
* Total bilirubin \< 2.0 mg/dL
* Serum alanine aminotransferase and aspartate aminotransferase \< 3 x ULN
* Left ventricular ejection fraction by echocardiogram or multiple-gated acquisition \>= 50%
* Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
* Women of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to enrollment and commit to two forms of birth control
* Men must use a latex condom during any sexual contact with women of childbearing potential
* Willing to adhere to protocol specific requirements
* Participant or legal representative must understand the investigational nature of this study and sign an Independent Ethics Committee/Institutional Review Board approved written informed consent form prior to receiving any study related procedure

Exclusion Criteria:

* Prior treatment of AML
* Known clinically active central nervous system (CNS) leukemia
* Core-binding factor leukemia
* Acute promyelocytic leukemia
* Uncontrolled other malignancy
* Prior anthracycline exposure \> 368 mg/m\^2 of daunorubicin or equivalent
* Cardiovascular disease resulting in heart failure (New York Heart Association class III or IV), unstable angina (angina symptoms at rest), or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
* Hypersensitivity to cytarabine, daunorubicin, or liposomal drugs
* Known active HIV infection
* Known history of active hepatitis B or C infection
* Pre-existing liver disease (e.g. cirrhosis, chronic hepatitis B or C, nonalcoholic steatohepatitis, sclerosing cholangitis)
* Evidence of ongoing, uncontrolled systemic infection
* Pregnant or breastfeeding women
* Subject with concurrent severe and/or uncontrolled medical or psychiatric conditions that in the opinion of the investigator may impair the participation in the study or the evaluation of safety and/or efficacy
* History of Wilson disease or other copper-handling disorders
* Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug

Primary outcomes:
measure: Complete response rate (morphological complete remission [CR] and incomplete blood count recovery [CRi])
timeFrame: At day 45
Secondary outcomes:
measure: CR + CRi duration
timeFrame: Time from CR or CRi until relapse or last follow-up, assessed up to 5 years
measure: Event free survival
timeFrame: Time from treating until disease progression/relapse, death due to disease, or last follow-up, assessed up to 5 years
measure: Overall survival
timeFrame: Time from treatment until death due to any cause or last follow-up, assessed up to 5 years
measure: Allogeneic hematopoietic cell transplant rate
timeFrame: Up to 5 years
measure: Incidence of adverse events
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Buffalo
country: United States
facility: Roswell Park Cancer Institute
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical Center
state: New York
city: Pittsburgh
country: United States
facility: Allegheny Health Network Cancer Institute - West Penn Hospital
state: Pennsylvania
Study officials:
affiliation: Roswell Park Cancer Institute
name: Steven Green, MD
role: PRINCIPAL_INVESTIGATOR
References: