Acute Myeloid Leukemia (AML)Phase 2NCT02238925
An Open Label Phase II Pharmacokinetic and Pharmacodynamic Assessment of the Potential for QTc Prolongation Following First Induction Treatment With CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in Acute Leukemias and MDS Patients
A Phase 2 study testing CPX-351 in people with Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL), run by Jazz Pharmaceuticals at 5 sites in United States.
Trial summary
The purpose of this study is to assess the effects of CPX-351 on cardiac repolarization, assess plasma drug levels, asses serum copper levels, and assess drug levels in urine. Efficacy and Safety will be assessed in all patients enrolled to the study. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS)
- 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
- 26 participants
- Where
- 5 sitesUnited States
- Study dates
- Jul 2014to Jan 2016
- 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 to 80 years
- 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 to ≤ 80 years at the time of signing the informed consent form
- Life expectancy of at least 3 months
- Pathological confirmation by bone marrow documenting the following:
- Newly Diagnosed De novo AML according to WHO criteria except for Acute Promyelocytic Leukemia or patients with known favorable cytogenetics (see exclusion)
- Newly Diagnosed Secondary AML age <60 years and ≥76 to 80 years, defined as having a history of an antecedent hematologic disorder (myelodysplastic syndromes [MDS], myeloproliferative disease [MPD]or history of cytotoxic treatment for non-hematologic malignancy)
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- Patients with relapsed/refractory AML regardless of cytogenetic risk
- Patients with relapsed/refractory ALL
- Patients with MDS (IPSS score ≥ 1.5)
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2
- Able to adhere to the study visit schedule and other protocol requirements
- Laboratory values fulfilling the following:
- Serum Creatinine ≤ 2.0mg/dL
- Hepatic function with a score of < 7 points according to the Child-Pugh System
- Serum alanine aminotransferase or aspartate aminotransferase < 3 times the ULN. Note: If elevated liver enzymes are related to disease; contact medical monitor to discuss.
- Cardiac ejection fraction ≥50% by ECHO or MUGA
- Screening and Baseline QTcF (Fridericia's) less than 470 msec
- Patients with second malignancies in remission may be eligible if there is clinical evidence of disease stability for a period of greater than 6 months 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.
- All men and women must agree to practice effective contraception during the study period if not otherwise documented to be infertile.
Who cannot join
- Patients eligible for participation in Study CLTR0310-301 (Phase III study of CPX-351 NCT01696084) or who have already participated in that study are not eligible for this study.
- Patients taking medications known to prolong the QTc interval directly or that interact pharmacodynamically with medicines to prolong the QTc interval.
- Rhythm abnormalities (other than sinus bradycardia with HR < 50 bpm)
- AV block (other than 1o AV Block with PR > 200 msec)
- Bundle branch block or QRS ≥ 120 msec
- Abnormal T wave morphology (other than slight flattening)
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- Pathological U waves
- Other QRS or T/U morphology preventing accurate determination of QT interval
- Patients with unexplained syncope, history of or known risk factors for torsade des pointes, including congenital long QT syndrome, or family history of LQTS.
- Patients with history of and/or current evidence of myocardial impairment (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)
- Newly diagnosed patients with Acute promyelocytic leukemia [t(15;17)] or favorable cytogenetics, including t(8;21) or inv16
- Clinical evidence of active CNS leukemic involvement
- Chemotherapy or other investigational anticancer therapeutic drugs within 1 week prior to study entry unless AEs have resolved and there is no interference with the assessment of efficacy or safety; in the event of rapidly proliferative disease, however, the use of hydroxyurea is permitted up to 12 hours before study entry. Patients with prior bone marrow or stem cell transplant, considered for inclusion, should be discussed with the medical monitor first.
- Any serious medical condition or psychiatric illness that would prevent the patient from providing informed consent
- Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent)
- Active or uncontrolled infection. Patients with any 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 fevers believed to be due to leukemia or MDS are eligible provided a thorough infection work-up is negative and the patient is clinically and hemodynamically stable.
- Pregnant or lactating women
- Hypersensitivity to cytarabine, daunorubicin or liposomal products
- History of Wilson's disease or other copper-related metabolic disorder
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Franciscan Saint Francis Health
Indianapolis, Indiana, 46237
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Oregon Health & Science University
Portland, Oregon, 97239
University of Kansas Cancer Center
Westwood, Kansas, 66205
About this study
The purpose of this study is to assess the effects of CPX-351 on cardiac repolarization, assess plasma drug levels, asses serum copper levels, and assess drug levels in urine.
Efficacy and Safety will be assessed in all patients enrolled to the study.
Read the detailed description
This study is an open-label, single-arm, Phase II, PK and pharmacodynamic (PD) trial of CPX-351 in patients with documented acute leukemia (AML or ALL) or MDS (IPSS score ≥ 1.5) and suitable for treatment with intensive chemotherapy. Each patient will be screened for hepatic impairment. Hepatic impairment will be assessed using the Child-Pugh system and only patients with a Child-Pugh score <7 points will be eligible for this study. Patients will receive up to two inductions and four consolidation courses. Patients will be monitored for safety (early deaths, adverse events, metabolic changes, etc.) and efficacy (response for AML, ALL, and MDS) while on the study.
- Conditions
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS)
- Being tested
- CPX-351
- Keywords
- Newly Diagnosed AML, Secondary AML, Relapsed/Refractory AML, Relapsed/Refractory ALL, MDS
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CPX-351Drug
| Study group | Role | What they receive |
|---|---|---|
| CPX-351 | Experimental | Single Arm Study (Patients may receive up to 2 Inductions and 4 Consolidations):
Induction 1: CPX-351 will be given intravenously at 100units/m2 on days 1, 3, and 5 over a 90 minute infusion.
Induction 2: CPX-351 will be given intravenously at 100units/m2 on days 1 and 3 over a 90 minute infusion.
Consolidations 1-4: CPX-351 will be given intravenously at 65units/m2 on days 1 and 3 over a 90 minute infusion. Receives: Drug: CPX-351 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effect of CPX-351 on Cardiac Ventricular Repolarization (QTcF)
Time-matched QTcF Changes From Baseline after the start of first infusion
Measured: 21 days
Secondary outcomes (4)
Serum Copper Levels Change From Baseline
Change from Baseline to Induction 1, Day 5
Measured: During 1st induction (up to 5 days)
Complete Response Rate
Measured: Following 1st induction, following 2nd induction if applicable
Tmax
Measured: Induction 1, Day 5
Cmax
Measured: Induction 1, Day 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2014 (estimated)
- Study startJul 2014
- Primary completionMay 2015
- Study completionJan 2016
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02238925
- Sponsor's protocol ID
- CLTR0310-206
- Status last verified
- Oct 2017
- First submitted
- 14 Aug 2014
- Last updated
- 30 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Lin TL, Newell LF, Stuart RK, Michaelis LC, Rubenstein E, Pentikis HS, Callahan T, Alvarez D, Liboiron BD, Mayer LD, Wang Q, Banerjee K, Louie AC. A phase 2 study to assess the pharmacokinetics and pharmacodynamics of CPX-351 and its effects on cardiac repolarization in patients with acute leukemias. Cancer Chemother Pharmacol. 2019 Jul;84(1):163-173. doi: 10.1007/s00280-019-03856-9. Epub 2019 May 16.
PubMed 31098682
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Phase II Pharmacokinetic and Pharmacodynamic Assessment of the Potential for QTc Prolongation Following First Induction Treatment With CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in Acute Leukemias and MDS Patients
- ClinicalTrials.gov ID
- NCT02238925
- Acronym / sponsor ID
- CLTR0310-206
- Conditions
- Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Myelodysplastic Syndrome (MDS)
- Keywords
- Newly Diagnosed AML, Secondary AML, Relapsed/Refractory AML, Relapsed/Refractory ALL, MDS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jul 2014
- Primary completion
- May 2015 (actual)
- Study completion
- Jan 2016 (actual)
- First posted
- 12 Sep 2014 (estimated)
- Results
- Posted 30 Nov 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the effects of CPX-351 on cardiac repolarization, assess plasma drug levels, asses serum copper levels, and assess drug levels in urine.
Efficacy and Safety will be assessed in all patients enrolled to the study.
Detailed description
This study is an open-label, single-arm, Phase II, PK and pharmacodynamic (PD) trial of CPX-351 in patients with documented acute leukemia (AML or ALL) or MDS (IPSS score ≥ 1.5) and suitable for treatment with intensive chemotherapy. Each patient will be screened for hepatic impairment. Hepatic impairment will be assessed using the Child-Pugh system and only patients with a Child-Pugh score <7 points will be eligible for this study. Patients will receive up to two inductions and four consolidation courses. Patients will be monitored for safety (early deaths, adverse events, metabolic changes, etc.) and efficacy (response for AML, ALL, and MDS) while on the study.
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 | Experimental | Drug: CPX-351Single Arm Study (Patients may receive up to 2 Inductions and 4 Consolidations): Induction 1: CPX-351 will be given intravenously at 100units/m2 on days 1, 3, and 5 over a 90 minute infusion. Induction 2: CPX-351 will be given intravenously at 100units/m2 on days 1 and 3 over a 90 minute infusion. Consolidations 1-4: CPX-351 will be given intravenously at 65units/m2 on days 1 and 3 over a 90 minute infusion. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Ability to understand and voluntarily sign an informed consent form
- Age ≥ 18 to ≤ 80 years at the time of signing the informed consent form
- Life expectancy of at least 3 months
- Pathological confirmation by bone marrow documenting the following:
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2
Key exclusion criteria (19 listed)
- Patients eligible for participation in Study CLTR0310-301 (Phase III study of CPX-351 NCT01696084) or who have already participated in that study are not eligible for this study.
- Patients taking medications known to prolong the QTc interval directly or that interact pharmacodynamically with medicines to prolong the QTc interval.
- Rhythm abnormalities (other than sinus bradycardia with HR < 50 bpm)
- AV block (other than 1o AV Block with PR > 200 msec)
- Bundle branch block or QRS ≥ 120 msec
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 | Drug | CPX-351 |
Primary outcome
Effect of CPX-351 on Cardiac Ventricular Repolarization (QTcF)
Time frame: 21 days
Time-matched QTcF Changes From Baseline after the start of first infusion
4 secondary outcomes
Serum Copper Levels Change From Baseline
Time frame: During 1st induction (up to 5 days)
Complete Response Rate
Time frame: Following 1st induction, following 2nd induction if applicable
Tmax
Time frame: Induction 1, Day 5
Cmax
Time frame: Induction 1, Day 5
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31098682Lin TL, Newell LF, Stuart RK, Michaelis LC, Rubenstein E, Pentikis HS, Callahan T, Alvarez D, Liboiron BD, Mayer LD, Wang Q, Banerjee K, Louie AC. A phase 2 study to assess the pharmacokinetics and pharmacodynamics of CPX-351 and its effects on cardiac repolarization in patients with acute leukemias. Cancer Chemother Pharmacol. 2019 Jul;84(1):163-173. doi: 10.1007/s00280-019-03856-9. Epub 2019 May 16.
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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.
The record lists no contact. Use the official record to reach the sponsor.
5 sites 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 Acute Myeloid Leukemia (AML) and Acute Lymphoblastic Leukemia (ALL), run by Jazz Pharmaceuticals at 5 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 19 inclusion and 19 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 30 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 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 30 Nov 2017). 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?
Effect of CPX-351 on Cardiac Ventricular Repolarization (QTcF) (time frame: 21 days). 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 NCT02238925, 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 30 Nov 2017.