Leukemia, Myeloid, AcutePhase 2NCT03335267
Investigator Initiated Trial of CPX-351 for Untreated Acute Myeloid Leukemia
A Phase 2 study testing CPX-351 in people with Leukemia, Myeloid, Acute, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
This is an open label study to assess the suitability of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML. Patients may have received prior AML treatment with non-intensive regimens, e.g. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Leukemia, Myeloid, Acute
- 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
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Oct 2017to 29 May 2020
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Ability to understand and voluntarily give informed consent
- Age≥60 years at the time of study treatment
- Pathological diagnosis of AML according to WHO criteria (with >20% blasts in the peripheral blood or bone marrow) including:
- De novo AML with normal karyotype or adverse karyotypes (including patients with karyotypic abnormalities characteristic of MDS)
- Secondary AML: transformed from prior MDS or MPN, confirmed by bone marrow documentation of prior antecedent hematologic disorder
- Therapy-related AML: t-AML, requires documented history of prior cytotoxic therapy or ionizing radiotherapy for an unrelated disease
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- Performance status >50% KPS, ECOG 0-2
- Laboratory values fulfilling the following:
- Serum creatinine < 2.5 mg/dL
- Serum total bilirubin < 2.5 mg/dL,
- Serum alanine aminotransferase or aspartate aminotransferase < 3 times the ULN
- Patients with elevated liver enzymes and serum creatinine values secondary to AML are eligible after discussion with PI
- Cardiac ejection fraction ≥ 50% by echocardiography or MUGA
- Patients with history of second malignancies in remission may be eligible if there is clinical evidence of disease stability off cytotoxic chemotherapy, documented by imaging, tumor marker studies, etc., at screening. Patients maintained on long-term non-chemotherapy treatment, e.g., hormonal therapy, are eligible.
Who cannot join
- Acute promyelocytic leukemia [t(15;17)]
- Clinical evidence of active CNS leukemia
- Prior intensive chemotherapy for AML with anthracycline/cytarabine-based regimens and/ or prior HSCT. Patients may have been treated with commercially available or investigational hypomethylating agents (e.g. decitabine, azacitidine, SGI-110), lenalidomide, or low-dose cytarabine (not to exceed 20 mg/m2 daily for 14 days for ≤ 6 cycles)
- Prior treatment including HMA, systemic chemotherapy, surgery, or radiation therapy must have been completed at least 7 days before start of study treatment or after discussion with PI. Treatment with investigational agents must have been completed at least 14 days prior to study drug treatment. Hydroxyurea is permitted for control of blood counts before the start of study treatment. Toxicities associated with prior therapies must have recovered to grade 1 or less prior to start of study treatment.
- Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent).
- Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent obtaining informed consent
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- Patients with myocardial impairment of any cause (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, and congestive heart failure) resulting in heart failure by New York Heart Association Criteria (Class III or IV staging)
- Active or uncontrolled infection. Patients with an infection receiving treatment (antibiotic, antifungal or antiviral treatment) may be entered into the study but must be afebrile and hemodynamically stable for ≥72 hrs.
- Patients with current or recent evidence of invasive fungal infection (blood or tissue culture); patients with recent fungal infection must have a subsequent negative cultures to be eligible
- Known HIV (new testing not required) or evidence of active hepatitis B or C infection (with rising transaminase values)
- Hypersensitivity to cytarabine, daunorubicin or liposomal products
- History of Wilson's disease or other copper-metabolism disorder
- History of prior bone marrow or solid organ transplantation
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medical College
New York, New York, 10021
About this study
Official title: Phase II Trial of CPX (Cytarabine:Daunorubicin) Liposome Injection in Patients >/=60 Years of Age With AML Previously Untreated By Intensive Chemotherapy
This is an open label study to assess the suitability of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML. Patients may have received prior AML treatment with non-intensive regimens, e.g. hypomethylating agents, low dose Ara C or lenolidomide, but may not have received intensive AML treatment with anthracyclines and/or cytarabine prior to enrollment on this trial. The outcome of elderly patients following intensive treatment with CPX-351 will be measured by clinical endpoints for efficacy and safety and by biological/functional response.
- Conditions
- Leukemia, Myeloid, Acute
- Being tested
- CPX-351
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
Cytarabine:Daunorubicin Liposome Injection
| Study group | Role | What they receive |
|---|---|---|
| CPX-351 (Cytarabine:Daunorubicin) Injection | Experimental | Dosing for first induction: CPX-351
• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5
Dosing for second induction:
• CPX-351 at 100 u/m2 will be administered on days 1 and 3
Dosing for consolidation:
• CPX-351 at 65 u/m2 will be administered on days 1 and 3 Receives: Drug: CPX-351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Efficacy
Overall survival is measured from the date of registration to death from any cause. Patients not known to have died will be censored on the date they were last known to be alive. Patients were followed for 2.5 years.
Measured: 2.5 Years
30-Day Mortality Rate
Mortality rate at Day 30
Measured: 30 Days
Secondary outcomes (4)
Complete Response Rate (CR, CRp, CRi, and CR+CRp+CRi)
The number of patients who achieve response will be divided by the number of patients in the efficacy population to determine response rate.
Measured: 30 days post-treatment, up to 3 months post-baseline
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the MOCA
The Montreal Cognitive Assessment (MOCA) is a 30-point test which assesses several cognitive domains. Possible total scores range from 0 to 30. The results can be interpreted as follows: normal cognition: 26-30 points, mild cognitive impairment: 18-25 points, moderate cognitive impairment: 10-17 points, and severe cognitive impairment: under 10 points.
Measured: Screening through 30 days post-treatment, up to 3 months post-baseline
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the Blessed Orientation-Memory-Concentration Test
The Blessed Orientation-Memory-Concentration Test is designed to evaluate older patients for early dementia. Possible total scores range from 0 (all items answered correctly) to 28 (all items answered incorrectly).
Measured: Screening through 30 days post-treatment, up to 3 months post-baseline
Incidence of Adverse Events
Adverse events included neutropenic fever, transient episodes of pericarditis, febrile neutropenia, streptococcus bacteremia, hypotensive episode, severe diffuse cerebral dysfunction, UTI (klebsiella pneumonia), anorexia with malnutrition, hypophosphatemia, hypokalemia, purple macules, elevated ALT, hypoalbuminemia, hyperbilirubinemia, syncope, joint pain, transaminitis, bacteremia, typhlitis, VRE bacteremia, Osteomyelitis, Decreased LVEF, GI adenovirus, Acute kidney injury, pulmonary edema, neutropenia, bronchospasm, bone pain, urinary incontinence, c. difficile infection, mucositis, and vaginal pain.
Measured: Through treatment completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2017
- Study start19 Oct 2017
- Primary completion19 Dec 2018
- Study completion29 May 2020
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 Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe 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
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Ellen K Ritchie, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03335267
- Sponsor's protocol ID
- 1510016710
- Status last verified
- Feb 2024
- First submitted
- 7 Sep 2017
- Last updated
- 22 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of CPX (Cytarabine:Daunorubicin) Liposome Injection in Patients >/=60 Years of Age With AML Previously Untreated By Intensive Chemotherapy
- ClinicalTrials.gov ID
- NCT03335267
- Acronym / sponsor ID
- 1510016710
- Conditions
- Leukemia, Myeloid, Acute
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 19 Oct 2017 (actual)
- Primary completion
- 19 Dec 2018 (actual)
- Study completion
- 29 May 2020 (actual)
- First posted
- 7 Nov 2017 (actual)
- Results
- Posted 22 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open label study to assess the suitability of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML. Patients may have received prior AML treatment with non-intensive regimens, e.g. hypomethylating agents, low dose Ara C or lenolidomide, but may not have received intensive AML treatment with anthracyclines and/or cytarabine prior to enrollment on this trial. The outcome of elderly patients following intensive treatment with CPX-351 will be measured by clinical endpoints for efficacy and safety and by biological/functional response.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CPX-351 (Cytarabine:Daunorubicin) Injection | Experimental | Drug: CPX-351Dosing for first induction: CPX-351 • CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5 Dosing for second induction: • CPX-351 at 100 u/m2 will be administered on days 1 and 3 Dosing for consolidation: • CPX-351 at 65 u/m2 will be administered on days 1 and 3 |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Ability to understand and voluntarily give informed consent
- Age≥60 years at the time of study treatment
- Pathological diagnosis of AML according to WHO criteria (with >20% blasts in the peripheral blood or bone marrow) including:
- De novo AML with normal karyotype or adverse karyotypes (including patients with karyotypic abnormalities characteristic of MDS)
- Secondary AML: transformed from prior MDS or MPN, confirmed by bone marrow documentation of prior antecedent hematologic disorder
Key exclusion criteria (13 listed)
- Acute promyelocytic leukemia [t(15;17)]
- Clinical evidence of active CNS leukemia
- Prior intensive chemotherapy for AML with anthracycline/cytarabine-based regimens and/ or prior HSCT. Patients may have been treated with commercially available or investigational hypomethylating agents (e.g. decitabine, azacitidine, SGI-110), lenalidomide, or low-dose cytarabine (not to exceed 20 mg/m2 daily for 14 days for ≤ 6 cycles)
- Prior treatment including HMA, systemic chemotherapy, surgery, or radiation therapy must have been completed at least 7 days before start of study treatment or after discussion with PI. Treatment with investigational agents must have been completed at least 14 days prior to study drug treatment. Hydroxyurea is permitted for control of blood counts before the start of study treatment. Toxicities associated with prior therapies must have recovered to grade 1 or less prior to start of study treatment.
- Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent).
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-351Cytarabine:Daunorubicin Liposome Injection | Drug | CPX-351 (Cytarabine:Daunorubicin) Injection |
Primary outcome
Primary Efficacy
Time frame: 2.5 Years
Overall survival is measured from the date of registration to death from any cause. Patients not known to have died will be censored on the date they were last known to be alive. Patients were followed for 2.5 years.
30-Day Mortality Rate
Time frame: 30 Days
Mortality rate at Day 30
4 secondary outcomes
Complete Response Rate (CR, CRp, CRi, and CR+CRp+CRi)
Time frame: 30 days post-treatment, up to 3 months post-baseline
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the MOCA
Time frame: Screening through 30 days post-treatment, up to 3 months post-baseline
Change in Relationship of Cognitive Function to Treatment Response and OS, Event-free Survival and Morphologic Leukemia Free State as Measured by the Blessed Orientation-Memory-Concentration Test
Time frame: Screening through 30 days post-treatment, up to 3 months post-baseline
Incidence of Adverse Events
Time frame: Through treatment completion, an average of 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 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.
Ellen K Ritchie, MD principal investigator
Weill Medical College of Cornell University
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 Leukemia, Myeloid, Acute, run by Weill Medical College of Cornell University 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 14 inclusion and 13 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 22 Feb 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 22 Feb 2024). 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?
Primary Efficacy (time frame: 2.5 Years). The study lists 2 primary outcomes in all. 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 22 Feb 2024.