Lymphoid LeukemiaPhase 2NCT03575325
Vyxeos(CPX-351) in Adults w R/R Acute Lymphoblastic Leukemia
A Phase 2 study testing CPX-351 in people with Lymphoid Leukemia and Acute Lymphoblastic Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lymphoid Leukemia, Acute Lymphoblastic Leukemia, Refractory Acute Lymphoblastic Leukemia
- What is being tested?
- CPX-351
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Oct 2018to 14 Sep 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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
Who cannot join
- 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.
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- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
H. Lee Moffitt Cancer Center and Research Institute
Tampa, Florida, 33612
About this study
Official title: 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.
- Conditions
- Lymphoid Leukemia, Acute Lymphoblastic Leukemia, Refractory Acute Lymphoblastic Leukemia, Relapsed Acute Lymphoblastic Leukemia
- Being tested
- CPX-351
- Keywords
- T-cell acute lymphoblastic leukemia, B-cell acute lymphoblastic leukemia
- Medical subject headings
- Leukemia, Lymphoid, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Burkitt Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
Also called Vyxeos, cytarabine:daunorubicin
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.
| Study group | Role | What they receive |
|---|---|---|
| CPX-351 Treatment | Experimental | 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). Receives: Drug: CPX-351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Remission Rate (CR) + CR With Incomplete Recovery (CRi)
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).
Measured: At day 28
Secondary outcomes (3)
Progression Free Survival (PFS)
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.
Measured: 12 months
Overall Survival (OS)
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.
Measured: 12 months
Minimal Residual Disease (MRD)
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).
Measured: At day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2018
- Study start11 Oct 2018
- Primary completion7 Jun 2021
- Study completion14 Sep 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Lymphoid LeukemiaStudies of the same condition, open and under way.
- More trials run by H. Lee Moffitt Cancer Center and Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open label, single arm, single center, phase 2 pilot study of Vyxeos induction \& consolidation in relapsed and refractory ALL.
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Bijal Shah, M.D., Principal investigator, H. Lee Moffitt Cancer Center and Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03575325
- Sponsor's protocol ID
- MCC-19482
- Status last verified
- Dec 2024
- First submitted
- 21 Jun 2018
- Last updated
- 12 Dec 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-Arm, Open-Label Phase 2 Pilot Study of Vyxeos (CPX-351) in Adults With Relapsed or Refractory Acute Lymphoblastic Leukemia
- ClinicalTrials.gov ID
- NCT03575325
- Acronym / sponsor ID
- MCC-19482
- Conditions
- Lymphoid Leukemia, Acute Lymphoblastic Leukemia, Refractory Acute Lymphoblastic Leukemia, Relapsed Acute Lymphoblastic Leukemia
- Keywords
- T-cell acute lymphoblastic leukemia, B-cell acute lymphoblastic leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 11 Oct 2018 (actual)
- Primary completion
- 7 Jun 2021 (actual)
- Study completion
- 14 Sep 2023 (actual)
- First posted
- 2 Jul 2018 (actual)
- Results
- Posted 14 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open label, single arm, single center, phase 2 pilot study of Vyxeos induction \& consolidation in relapsed and refractory ALL.
| Arm | Type | Receives |
|---|---|---|
| CPX-351 Treatment | Experimental | Drug: CPX-351Participants 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CPX-351Also: Vyxeos, cytarabine:daunorubicinThe 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. | Drug | CPX-351 Treatment |
Primary outcome
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).
3 secondary outcomes
Progression Free Survival (PFS)
Time frame: 12 months
Overall Survival (OS)
Time frame: 12 months
Minimal Residual Disease (MRD)
Time frame: At day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bijal Shah, M.D. principal investigator
H. Lee Moffitt Cancer Center and Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CPX-351 in people with Lymphoid Leukemia and Acute Lymphoblastic Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete Remission Rate (CR) + CR With Incomplete Recovery (CRi) (time frame: At day 28). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2024; AzTrials last refreshed its copy on 7 Nov 2026.