Hematologic NeoplasmsPhase 1NCT00389428
Multicenter Study of CPX-351(Cytarabine:Daunorubicin) Liposome Injection in Patients With Advanced Hematologic Cancer.
A Phase 1 study testing CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in people with Hematologic Neoplasms, run by Jazz Pharmaceuticals at 4 sites in United States.
Trial summary
The primary objective of this study is to determine the recommended dose of CPX-351 for use in a phase 2 efficacy study in patients with leukemia. Secondarily, the study will assess the safety, serious adverse effects and how the body handles CPX-351. Preliminary evidence of antitumor activity will also be determined. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Neoplasms
- What is being tested?
- CPX-351 (Cytarabine:Daunorubicin) Liposome Injection
- Type of treatment
- Injection or infusion
- How many people
- 48 participants
- Where
- 4 sitesUnited States
- Study dates
- Sep 2006to Dec 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Ability to understand and voluntarily sign an informed consent form
- Age > 18 years at the time of signing the informed consent form
- Pathological confirmation of leukemia or myelodysplastic syndrome.
- AML according to WHO criteria; except for core-binding factor AMLs (t(8;21), inv(16) or t(16;16)) and APL
- ALL
- MDS
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- Patients with AML include the following:
- Patients in 2nd or greater relapse
- Patients in first relapse with initial CR duration lasting <6 months
- Patients in first relapse refractory to induction therapy
- Patients with primary refractory AML
- Patients with ALL include the following
- Patients with T-cell ALL refractory or in relapse following nelarabine
- Patients with other ALL that is refractory or in relapse.
- Patients with MDS include the following:
- The subset of RAEB-2 patients with >10% blasts with at least 1 prior therapy that includes a hypomethylating agent.
- Previously untreated chemotherapy induced AML
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0,1 or 2
- Able to adhere to the study visit schedule and other protocol requirements
- Life expectancy of at least 12 weeks
- Laboratory values fulfilling the following:
- Serum creatinine < 1.5 mg/dL
- Serum total bilirubin < 1.5 mg/dL
- Serum alanine aminotransferase or aspartate aminotransferase < 150 IU/liter Note: If elevated liver enzymes are related to disease; contact medical monitor to discuss.
- Cardiac ejection fraction > 50% by MUGA scan or echocardiography
- All men and women must agree to practice effective contraception during the study period if not otherwise documented to be infertile.
Who cannot join
- Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent the patient from signing the informed consent form
- Chemotherapy or other investigational anticancer therapeutic drugs in the two weeks prior to study entry; in the event of rapidly proliferative disease, however, the use of hydroxyurea is permitted up to 24 hours before study entry
- Clinical evidence of active CNS leukemic involvement
- Pregnant or lactating women
- Clinically significant cardiac disease (New York Heart Association Class III or IV)
- Severe debilitating pulmonary disease
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- Active and uncontrolled infection. Patients with an infection under active treatment with antibiotics and whose infection is controlled may be entered into the study
- Current evidence of invasive fungal infection (blood or tissue culture); HIV or hepatitis C infection
- Hypersensitivity to cytarabine, daunorubicin or liposomal products
- History of Wilson's disease or other copper-related disorder
Trial locations and contacts
4 sites in 1 country.
United States4 sites
North Shore University Hospital
Manhasset, New York, 11030
New York Presbyterian Hospital Weill Medical College of Cornell University
New York, New York, 10021
H. Lee Moffit Cancer Center & Research Institute at the University of S. Florida
Tampa, Florida, 33612-9497
Georgetown University Medical Center
Washington D.C., District of Columbia, 20007
About this study
Official title: Phase 1 Study of CPX-351(Cytarabine:Daunorubicin) Liposome Injection in Patients With Advanced Hematologic Malignancies.
The primary objective of this study is to determine the recommended dose of CPX-351 for use in a phase 2 efficacy study in patients with leukemia. Secondarily, the study will assess the safety, serious adverse effects and how the body handles CPX-351. Preliminary evidence of antitumor activity will also be determined.
Read the detailed description
CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin. The two drugs are present inside the liposome in a 5:1 molar ratio. The development of CPX-351 (cytarabine:daunorubicin) Liposome Injection was based on 1) defining a synergistic ratio of the two active moieties, cytarabine and daunorubicin, using cell-based screening assays and 2) designing a liposomal drug carrier to maintain this ratio after intravenous administration. CPX-351 was found to be more active in in vivo models of cancer than combinations of conventional cytarabine and daunorubicin. Both cytarabine and daunorubicin are active chemotherapeutic agents, each approved for clinical use in the United States for the treatment of hematological neoplasms.
CPX-351 is being developed with the hypothesis that it is superior to the currently used regimen of cytarabine and daunorubicin in the treatment of acute leukemia. This phase I study will determine the dose to carry forward into phase II trials.
- Conditions
- Hematologic Neoplasms
- Being tested
- CPX-351 (Cytarabine:Daunorubicin) Liposome Injection
- Keywords
- Hematologic malignancies, Malignancies, hematologic, Hematopoietic malignancies
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351 (Cytarabine:Daunorubicin) Liposome InjectionDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the maximum tolerated dose (MTD) for use in phase 2
Secondary outcomes (3)
To evaluate the safety and dose-limiting toxicities (DLT) of CPX-351.
To determine the pharmacokinetic parameters of CPX-351 administered in this schedule.
To assess preliminary efficacy information of CPX-351 administered in this schedule in patients with advanced leukemias.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2006 (estimated)
- Study startSep 2006
- Primary completionDec 2008
- Study completionDec 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematologic NeoplasmsStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Arthur Louie, M.D., Study director, Jazz PharmaceuticalsJonathan Kolitz, M.D., Principal investigator, New York School of Medicine at North Shore University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00389428
- Sponsor's protocol ID
- CLTR0305-101
- Status last verified
- May 2012
- First submitted
- 16 Oct 2006
- Last updated
- 17 May 2012
- Results posted
- No
Publications
Feldman EJ, Kolitz JE, Trang JM, Liboiron BD, Swenson CE, Chiarella MT, Mayer LD, Louie AC, Lancet JE. Pharmacokinetics of CPX-351; a nano-scale liposomal fixed molar ratio formulation of cytarabine:daunorubicin, in patients with advanced leukemia. Leuk Res. 2012 Oct;36(10):1283-9. doi: 10.1016/j.leukres.2012.07.006. Epub 2012 Jul 26.
PubMed 22840315
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of CPX-351(Cytarabine:Daunorubicin) Liposome Injection in Patients With Advanced Hematologic Malignancies.
- ClinicalTrials.gov ID
- NCT00389428
- Acronym / sponsor ID
- CLTR0305-101
- Conditions
- Hematologic Neoplasms
- Keywords
- Hematologic malignancies, Malignancies, hematologic, Hematopoietic malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2006
- Primary completion
- Dec 2008 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 18 Oct 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to determine the recommended dose of CPX-351 for use in a phase 2 efficacy study in patients with leukemia. Secondarily, the study will assess the safety, serious adverse effects and how the body handles CPX-351. Preliminary evidence of antitumor activity will also be determined.
Detailed description
CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin. The two drugs are present inside the liposome in a 5:1 molar ratio. The development of CPX-351 (cytarabine:daunorubicin) Liposome Injection was based on 1) defining a synergistic ratio of the two active moieties, cytarabine and daunorubicin, using cell-based screening assays and 2) designing a liposomal drug carrier to maintain this ratio after intravenous administration. CPX-351 was found to be more active in in vivo models of cancer than combinations of conventional cytarabine and daunorubicin. Both cytarabine and daunorubicin are active chemotherapeutic agents, each approved for clinical use in the United States for the treatment of hematological neoplasms.
CPX-351 is being developed with the hypothesis that it is superior to the currently used regimen of cytarabine and daunorubicin in the treatment of acute leukemia. This phase I study will determine the dose to carry forward into phase II trials.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (26 listed)
- Ability to understand and voluntarily sign an informed consent form
- Age > 18 years at the time of signing the informed consent form
- Pathological confirmation of leukemia or myelodysplastic syndrome.
- AML according to WHO criteria; except for core-binding factor AMLs (t(8;21), inv(16) or t(16;16)) and APL
- ALL
Key exclusion criteria (10 listed)
- Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent the patient from signing the informed consent form
- Chemotherapy or other investigational anticancer therapeutic drugs in the two weeks prior to study entry; in the event of rapidly proliferative disease, however, the use of hydroxyurea is permitted up to 24 hours before study entry
- Clinical evidence of active CNS leukemic involvement
- Pregnant or lactating women
- Clinically significant cardiac disease (New York Heart Association Class III or IV)
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-351 (Cytarabine:Daunorubicin) Liposome Injection | Drug | – |
Primary outcome
To determine the maximum tolerated dose (MTD) for use in phase 2
3 secondary outcomes
To evaluate the safety and dose-limiting toxicities (DLT) of CPX-351.
To determine the pharmacokinetic parameters of CPX-351 administered in this schedule.
To assess preliminary efficacy information of CPX-351 administered in this schedule in patients with advanced leukemias.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2012.
- Derived from this study: PubMed 22840315Feldman EJ, Kolitz JE, Trang JM, Liboiron BD, Swenson CE, Chiarella MT, Mayer LD, Louie AC, Lancet JE. Pharmacokinetics of CPX-351; a nano-scale liposomal fixed molar ratio formulation of cytarabine:daunorubicin, in patients with advanced leukemia. Leuk Res. 2012 Oct;36(10):1283-9. doi: 10.1016/j.leukres.2012.07.006. Epub 2012 Jul 26.
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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.
Arthur Louie, M.D. study director
Jazz Pharmaceuticals
Jonathan Kolitz, M.D. principal investigator
New York School of Medicine at North Shore University Hospital
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in people with Hematologic Neoplasms, run by Jazz Pharmaceuticals at 4 sites 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 26 inclusion and 10 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 17 May 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To determine the maximum tolerated dose (MTD) for use in phase 2. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00389428, which also lists 1 linked publication.
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 17 May 2012.