Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia

March 27, 2024 updated by: H. Lee Moffitt Cancer Center and Research Institute

A Single-Arm, Open-Label Phase 2 Pilot Study of Vyxeos (CPX-351) in Adults With Relapsed or Refractory Acute Lymphoblastic Leukemia

This study involves Vyxeos (CPX-351), a formulation of a fixed combination of the two anti-tumor drugs, cytarabine and daunorubicin that will be given as an infusion over 90 minutes. This study will use what is called a "liposome" injection. This is a special fat capsule (called a liposome) that surrounds the cytarabine and daunorubicin and protects the drugs from being eliminated/destroyed by the body.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

11

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Florida
      • Tampa, Florida, United States, 33612
        • H. Lee Moffitt Cancer Center and Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Willing and able to provide written informed consent/assent for the trial.
  • Be ≥ 18 years of age on day of signing informed consent.
  • Able to adhere to the study visit schedule and other protocol requirements.
  • Pathologically confirmed B- or T-cell acute lymphoblastic or mixed phenotype acute leukemia, with >5% peripheral blood or bone marrow lymphoblasts and/or extramedullary disease >1x1cm.
  • Relapsed or refractory acute lymphoblastic leukemia after at least 1 prior cycle of therapy. Patients with Philadelphia chromosome positive disease must have failed at least two prior tyrosine kinase inhibitors.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-1.
  • Cardiac ejection fraction ≥ 50% by echocardiography or multigated acquisition scan (MUGA).
  • Must be at least 2 weeks out from any prior systemic chemotherapy, blinatumumab, radiation, and/or other investigational agents, and have recovered to grade 1 from any toxicity related to prior therapy. Glucocorticoids are permitted up to 1 day prior to the first dose.
  • Serum bilirubin and creatinine less than 1.5x upper limit of normal (ULN). AST and ALT must be less than 3x ULN, unless there is suspected liver involvement.
  • Females of childbearing potential (FCBP) must have a negative serum pregnancy test at screening. A FCBP is considered when a sexually mature female: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 12 consecutive months.
  • A FCBP must agree to use of two methods of highly effective contraception, be surgically sterile, or abstain from heterosexual activity for the course of the study through 30 days after the last dose of study treatment.
  • Male participants must agree to use an adequate method of contraception starting with the first dose of study therapy through 30 days after the last dose of study therapy. Men must agree to not donate sperm during and after the study

Exclusion Criteria:

  • Clinical evidence of active central nervous system (CNS) leukemia.
  • Any major surgery or radiation therapy within four weeks.
  • Any active infection requiring systemic therapy, including HIV, Hepatitis B, and/or Hepatitis C.
  • Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator (including but not limited to severe graft-versus-host disease, unstable angina, pulmonary hypertension, active/prior veno-occlusive disease of the liver or severe CHF (NYHA III-IV).
  • Patients with active (uncontrolled, metastatic) second malignancies.
  • Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the screening visit through 30 after the last dose of trial treatment.
  • Hypersensitivity to cytarabine, daunorubicin, or liposomal products.
  • History of Wilson's disease or other copper-metabolism disorder.
  • Patients with prior cumulative anthracycline exposure of greater than 368 mg/m^2 daunorubicin or equivalent).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CPX-351 Treatment

Participants will receive induction with CPX-351 at a dose of 100 u/m^2 administered intravenously over 90 minutes on days 1, 3 and 5 of a 28 day cycle.

This may be followed by consolidation with CPX-351 at a dose of 65 u/m^2 administered intravenously over 90 minutes on days 1 and 3 of a 28 day cycle (up to 3 cycles).
Drug: CPX-351
The infusion of CPX-351 (cytarabine:daunorubicin) Liposome Injection will be performed through a central venous catheter, using an infusion pump to ensure that the drug is infused over the specified time period.
Other Names:
  • Vyxeos
  • cytarabine:daunorubicin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete Remission Rate (CR) + CR With Incomplete Recovery (CRi)
Time Frame: At day 28
Expansion from phase II pilot to a phase II trial will depend on demonstration of CR/CRi amongst 4 of the initial 10 treated patients. Investigators will measure remission rate at day 28 to address the primary endpoint of complete remission (with or without complete hematologic recovery), as defined by Cheson Criteria (ref 27). For those with extramedullary disease, Lugano criteria will be used to assess response. This is a standard assessment of drug efficacy for phase 2 clinical trial design in acute leukemias, as response correlates closely with progression free- and overall survival (PFS and OS).
At day 28

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Progression Free Survival (PFS)
Time Frame: 12 months
Progression Free Survival as defined by Cheson and Lugano Criteria, namely progression, failure to respond, or death, as assessed from time of first treatment. Progression Free Survival will be reported as per Cheson Criteria, and analyzed using a standard Kaplan-Meier approach. Patients will undergo bone marrow biopsy evaluation after each cycle of therapy per standard of care to facilitated assessment.
12 months
Overall Survival (OS)
Time Frame: 12 months
Overall Survival as defined by Cheson and Lygano Criteria, namely death due to any cause as assessed from time of first treatment. Overall Survival will be reported as per Cheson Criteria, and analyzed using a standard Kaplan-Meier approach. Patients will undergo bone marrow biopsy evaluation after each cycle of therapy per standard of care to facilitated assessment.
12 months
Minimal Residual Disease (MRD)
Time Frame: At day 28
MRD assessment will be provided using ClonoSeq test result. ClonoSEQ® is an FDA-cleared test used to detect minimal residual disease (MRD) in bone marrow from patients with multiple myeloma or B-cell acute lymphoblastic leukemia (B-ALL) and blood or bone marrow from patients with chronic lymphocytic leukemia (CLL).
At day 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

H. Lee Moffitt Cancer Center and Research Institute

Collaborators

Jazz Pharmaceuticals

Investigators

  • Principal Investigator: Bijal Shah, M.D., H. Lee Moffitt Cancer Center and Research Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 11, 2018

Primary Completion (Actual)

June 7, 2021

Study Completion (Actual)

September 14, 2023

Study Registration Dates

First Submitted

June 21, 2018

First Submitted That Met QC Criteria

June 21, 2018

First Posted (Actual)

July 2, 2018

Study Record Updates

Last Update Posted (Actual)

March 29, 2024

Last Update Submitted That Met QC Criteria

March 27, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MCC-19482

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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