Acute Myelogenous LeukemiaPhase 1NCT00875693
A Novel Sequential Treatment of Salvage and Reduced Intensity Conditioning (RIC) Chemotherapy for Allogeneic Stem-Cell Transplantation (SCT) for Primary Refractory and Relapsed Acute Myelogenous Leukemia (AML)
A Phase 1 study testing CPX-351 in people with Acute Myelogenous Leukemia and Myelodysplastic Syndrome, run by Weill Medical College of Cornell University at 1 site in United States.
Trial summary
This open-label Phase I study is designed to determine the maximum tolerated dose (MTD) for CPX-351 followed by a reduced intensity conditioning regimen and incorporates a dose-escalation schedule that sequentially enrolls 6 dosing cohorts. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myelogenous Leukemia, Myelodysplastic Syndrome
- What is being tested?
- CPX-351
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 47 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jun 2009to 27 Dec 2017
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients are included, if they fulfill at least one of the following criteria defining refractory or relapsed AML according to previously established criteria 1-3: (1) Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy. (2) First relapse. (3) Relapse refractory to salvage chemotherapy (4) Second or subsequent relapse.
- Patients with MDS, either refractory anemia with excess blasts (RAEB) I or RAEB II
- Age between 18 and 70 years old.
- Patients must have a Karnofsky Performance Status > 70.
- Each patient must be willing to participate as a research subject and must sign an informed consent form.
- If the patient has a history of a prior malignancy, they must be without any evidence of disease of that prior malignancy for at least 2 years before being eligible for transplant on this protocol. This excludes skin cancers that may have been excised within that 2 year period.
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- Patients must have adequate physical function measured by:
- Cardiac: asymptomatic or if symptomatic then left ventricular ejection fraction (LVEF) at rest must be > 50% and must improve with exercise.
- Hepatic: < 3x upper limit of normal (ULN) alanine aminotransferase (ALT) and < 1.5 total serum bilirubin, unless liver is involved with the disease or there is congenital benign hyperbilirubinemia.
- Renal: serum creatinine within normal range for age or if serum creatinine is outside the normal range, then creatinine clearance > 60-ml/min.
- Pulmonary: asymptomatic or if symptomatic, diffusing capacity of carbon monoxide (DLCO) > 45% of predicted (corrected for hemoglobin)
Who cannot join
- Impaired renal function with a measured or calculated creatinine clearance of less than 60 ml/min.
- Impaired hepatic function defined as a bilirubin greater than 1.5 x upper limit of normal or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 x normal.
- Serious active or uncontrolled infection (Infections are controlled when patients are afebrile and hemodynamically stable for 72 hours) or medical condition.
- Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
- Impaired pulmonary function with a DLCO less than 45% predicted.
- Impaired cardiac function with an ejection fraction less than 50% of predicted by echocardiogram or multigated acquisition scan (MUGA).
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- Prior daunorubicin therapy with a cumulative dose of more than 368 mg/m2 or equivalent. The anthracycline agents commonly used in treating myeloid malignancies are doxorubicin, idarubicin and mitoxantron.
- For example, a patient who receives 7 + 3 (daunorubicin 180 mg/m2) for induction and MEC regimen (mitoxantrone, etoposide, cytaragine; mitoxantrone 48 mg/m2) for salvage. The cumulative daunorubicin equivalent is 180 + (48x2) = 278 mg/m2.
- Other systemic anticancer therapy or ongoing toxicities from such therapy.
- Patients with a history of and/or current evidence of myocardial impairment (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, congestive heart failure), resulting in heart failure by New York Heart Association Class III or IV staging.
- Patients with Wilson disease or other Copper-related disorders.
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: A Novel Sequential Treatment of Salvage and Reduced Intensity Conditioning RIC) Chemotherapy for Allogeneic Stem-Cell Transplantation (SCT)for Primary Refractory and Relapsed Acute Myelogenous Leukemia
This open-label Phase I study is designed to determine the maximum tolerated dose (MTD) for CPX-351 followed by a reduced intensity conditioning regimen and incorporates a dose-escalation schedule that sequentially enrolls 6 dosing cohorts. After the determination of the MTD, the investigator reserves the option to enroll up to 10 additional subjects in an expanded safety cohort(s) at the MTD.
Refractory and relapsed AML patients who meet standard institutional criteria to undergo sequential induction/reduced intensity conditioning allogeneic transplants will be offered a transplant from a related or unrelated donor (full match or 1 antigen mismatch). Cord blood transplants will not be used in this study.
- Being tested
- CPX-351
- Keywords
- AML, leukemia, MDS
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm A dose of CPX - 351 | Experimental | Dose level 1A: 60 units/m2 days -28, -26 and -24 Dose level 2A: 80 units/m2 days -28, -26 and -24 Dose level 3A: 100 units/m2 days -28, -26 and -24 Dose level 4A: 120 units/m2 days -28, -26 and -24 Dose level 5A: 140 units/m2 days -28, -26 and -24 Dose level 6A: 160 units/m2 days -28, -26 and -24 Receives: Drug: CPX-351 |
| Arm B dose of CPX-351 | Experimental | Dose level 1B: 60 units/m2 days -21, -19 and -17 Dose level 2B: 80 units/m2 days -21, -19 and -17 Dose level 3B: 100 units/m2 days -21, -19 and -17 Dose level 4B: 120 units/m2 days -21, -19 and -17 Dose level 5B: 140 units/m2 days -21, -19 and -17 Receives: Drug: CPX-351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the toxicity and safety of the regimen
Measured: cohort dependent
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2009 (estimated)
- Study start2 Jun 2009
- Primary completion30 Jun 2014
- Study completion27 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myelogenous LeukemiaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 47 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
- Usama Gergis, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00875693
- Sponsor's protocol ID
- 0812010140
- Status last verified
- Jan 2018
- First submitted
- 2 Apr 2009
- Last updated
- 17 Jan 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Novel Sequential Treatment of Salvage and Reduced Intensity Conditioning RIC) Chemotherapy for Allogeneic Stem-Cell Transplantation (SCT)for Primary Refractory and Relapsed Acute Myelogenous Leukemia
- ClinicalTrials.gov ID
- NCT00875693
- Acronym / sponsor ID
- 0812010140
- Keywords
- AML, leukemia, MDS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 2 Jun 2009 (actual)
- Primary completion
- 30 Jun 2014 (actual)
- Study completion
- 27 Dec 2017 (actual)
- First posted
- 3 Apr 2009 (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.
This open-label Phase I study is designed to determine the maximum tolerated dose (MTD) for CPX-351 followed by a reduced intensity conditioning regimen and incorporates a dose-escalation schedule that sequentially enrolls 6 dosing cohorts. After the determination of the MTD, the investigator reserves the option to enroll up to 10 additional subjects in an expanded safety cohort(s) at the MTD.
Refractory and relapsed AML patients who meet standard institutional criteria to undergo sequential induction/reduced intensity conditioning allogeneic transplants will be offered a transplant from a related or unrelated donor (full match or 1 antigen mismatch). Cord blood transplants will not be used in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A dose of CPX - 351 | Experimental | Drug: CPX-351Dose level 1A: 60 units/m2 days -28, -26 and -24 Dose level 2A: 80 units/m2 days -28, -26 and -24 Dose level 3A: 100 units/m2 days -28, -26 and -24 Dose level 4A: 120 units/m2 days -28, -26 and -24 Dose level 5A: 140 units/m2 days -28, -26 and -24 Dose level 6A: 160 units/m2 days -28, -26 and -24 |
| Arm B dose of CPX-351 | Experimental | Drug: CPX-351Dose level 1B: 60 units/m2 days -21, -19 and -17 Dose level 2B: 80 units/m2 days -21, -19 and -17 Dose level 3B: 100 units/m2 days -21, -19 and -17 Dose level 4B: 120 units/m2 days -21, -19 and -17 Dose level 5B: 140 units/m2 days -21, -19 and -17 |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Patients are included, if they fulfill at least one of the following criteria defining refractory or relapsed AML according to previously established criteria 1-3: (1) Primary induction failure (PIF) after ≥ 2 cycles of chemotherapy. (2) First relapse. (3) Relapse refractory to salvage chemotherapy (4) Second or subsequent relapse.
- Patients with MDS, either refractory anemia with excess blasts (RAEB) I or RAEB II
- Age between 18 and 70 years old.
- Patients must have a Karnofsky Performance Status > 70.
- Each patient must be willing to participate as a research subject and must sign an informed consent form.
Key exclusion criteria (11 listed)
- Impaired renal function with a measured or calculated creatinine clearance of less than 60 ml/min.
- Impaired hepatic function defined as a bilirubin greater than 1.5 x upper limit of normal or alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 x normal.
- Serious active or uncontrolled infection (Infections are controlled when patients are afebrile and hemodynamically stable for 72 hours) or medical condition.
- Women who are pregnant or breast feeding. Women of childbearing age must use adequate contraception and have a negative pregnancy test.
- Impaired pulmonary function with a DLCO less than 45% predicted.
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-351CPX-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. | Drug | Arm A dose of CPX - 351, Arm B dose of CPX-351 |
Primary outcome
To determine the toxicity and safety of the regimen
Time frame: cohort dependent
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 Jan 2018.
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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.
Usama Gergis, 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 1 study testing CPX-351 in people with Acute Myelogenous Leukemia and Myelodysplastic Syndrome, 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 11 inclusion and 11 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 Jan 2018. 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 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 toxicity and safety of the regimen (time frame: cohort dependent). 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 17 Jan 2018.