Acute Myeloid LeukemiaPhase 1NCT05558124
CPX-351 in Combination With Gemtuzumab Ozogamicin in Newly Diagnosed AML
A Phase 1 study testing Vyxeos and Gemtuzumab Ozogamicin in people with Acute Myeloid Leukemia, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
The purpose of the study is to determine the safety of combining the drugs gemtuzumab ozogamicin (GO) with CPX-351 in order to treat the disease, as well as to find the maximum tolerated dose level and recommended Phase 2 dose level of GO with a fixed dose of CPX-351. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Vyxeos and Gemtuzumab Ozogamicin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Feb 2023to Apr 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged ≥18 and ≤70 years with newly diagnosed any risk AML as defined by ELN 2017 criteria
- For females of child-bearing potential: use of highly effective contraception upon enrollment and during study participation and for an additional 6 months after the end of CPX-351 and Gemtuzumab ozogamicin administration: A female of child-bearing potential 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
- The effects of CPX-351 and gemtuzumab ozogamicin on the developing human fetus are unknown. For this reason, women of child-bearing potential as defined above must have a negative serum or urine pregnancy test within 24 hours prior to beginning study treatment.
- For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner
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- Myeloblasts expressing CD33 as determined by flow cytometry or immunohistochemistry
- ECOG ≤ 2 and eligible to receive intensive chemotherapy as determined by the treating physician
- Prior malignancy is allowed providing it does not require concurrent therapy. Exception: Active hormonal therapy is allowed.
- Prior hypomethylating agents (HMA) therapy including azacitidine or decitabine when used for non-AML diagnoses is allowed. Most recent dose must have been ≥14 days prior to day 1 of study treatment.
- Participants must have acceptable organ function
- Adequate cardiac function defined as ejection fraction of ≥50% as determined by multigated acquisition scan (MUGA) or 2D echocardiogram.
- Hydroxyurea is allowed for cytoreduction until day 1 of study treatment
Who cannot join
- Prior treatment of AML except hydroxyurea and/or leukapheresis
- Participants with acute promyelocytic leukemia (APL).
- Known current and clinically active central nervous system (CNS) leukemia.
- Severe liver disease (cirrhosis, non-alcoholic steatohepatitis, sclerosing cholangitis) or patients with known Wilson's disease.
- Participants with known active infection with hepatitis B or hepatitis C virus
- Known allergic reactions to components of the CPX-351 (cytarabine or daunorubicin) or Gemtuzumab ozogamicin.
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- Patients with any prior anthracycline exposure plus any planned on-study anthracycline exposure cannot not exceed 550 mg/m2 of daunorubicin (or equivalent). For participants who have received radiation therapy to the mediastinum, the total cumulative dose of anthracycline should not exceed 400 mg/m2 of daunorubicin(or equivalent).
- Hemodynamically unstable (subjects requiring vasopressor support will not be eligible).
- Treatment with another investigational drug within 14 days.
- Uncontrolled cardiac disease including congestive heart failure class III or IV by the NYHA, unstable angina (angina symptoms at rest), new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months.
- Any disorder that compromises the subject's ability to give written informed consent and/or to comply with study procedures.
- Any substance abuse, severe and/or uncontrolled medical, social or psychiatric conditions that may prevent the subject from completing the study, interfere with the evaluation of safety and/or efficacy, or interfere with the interpretation of the study results.
- Female subject who is pregnant or breastfeeding.
- Any patient with a known FLT3 ITD or FLT3 TKD mutation
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
Lisa Nardelli, 813-745-4731, lisa.nardelli@moffitt.org
Onyee Chan, 1-813-745-2069, Onyee.Chan@moffitt.org
About this study
Official title: A Phase 1/1b Dose Escalation and Expansion of CPX-351 in Combination With Gemtuzumab Ozogamicin in Newly Diagnosed Acute Myeloid Leukemia
The purpose of the study is to determine the safety of combining the drugs gemtuzumab ozogamicin (GO) with CPX-351 in order to treat the disease, as well as to find the maximum tolerated dose level and recommended Phase 2 dose level of GO with a fixed dose of CPX-351.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Vyxeos, Gemtuzumab Ozogamicin
- 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.
VyxeosDrug
Also called daunorubicin-cytarabine, CPX-351
Fixed dose of Vyxeos (44 mg/m2 daunorubicin and 100 mg/m2 cytarabine) (Day 1, 3, and 5) in combination with various dose schedules of Gemtuzumab Ozogamicin (GO)
Gemtuzumab OzogamicinDrug
Also called Mylotarg
Participants will be treated at the following dose levels:
Dose Level 1 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1 Dose Level 2 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1 and 4 Dose Level 3 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1, 4, 7
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation | Experimental | Dose escalation to determine the maximum tolerated dose (MTD) of CPX-351 in combination with Gemtuzumab Ozogamicin in participants with newly diagnosed acute myeloid leukemia. Participants will be treated in cohorts of size three to six and the dosage will be escalated if the clinical toxicity is acceptable. A total of 3 dose levels will be used. Receives: Drug: Vyxeos; Drug: Gemtuzumab Ozogamicin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
Dose escalation will determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Vyxeos plus Gemtuzumab Ozogamicin. The MTD is the highest dose of the combination therapy that dose not cause unacceptable side effects.
Measured: Up to 18 Months
Secondary outcomes (4)
Rate of Complete Remission
Rate of complete remission (CR) and complete remission with incomplete blood count recovery (CRi). The definition of CR and CRi is based on the European LeukemiaNet 2017 Response Criteria for AML.
Measured: Up to 18 Months
Measurable Residual Disease
Rate of measurable residual disease via RT-PCR for core binding factor leukemia as well as NPM1 mutated AML
Measured: Up to 18 Months
Overall Survival
Overall survival is defined as the duration of time from start of treatment to the time of death from any cause or date of last contact.
Measured: Up to 5 years
Relapse Free Survival (RFS)
RFS is defined as time interval between achievement of CR to time of relapse
Measured: Up to 18 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2022
- Study start16 Feb 2023
- Primary completionApr 2027 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Onyee Chan, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05558124
- Sponsor's protocol ID
- MCC-21450
- Status last verified
- Feb 2026
- First submitted
- 22 Sep 2022
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/1b Dose Escalation and Expansion of CPX-351 in Combination With Gemtuzumab Ozogamicin in Newly Diagnosed Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT05558124
- Acronym / sponsor ID
- MCC-21450
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Jazz Pharmaceuticals
- Study start
- 16 Feb 2023 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 28 Sep 2022 (actual)
- 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 purpose of the study is to determine the safety of combining the drugs gemtuzumab ozogamicin (GO) with CPX-351 in order to treat the disease, as well as to find the maximum tolerated dose level and recommended Phase 2 dose level of GO with a fixed dose of CPX-351.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation | Experimental | Drug: Vyxeos, Drug: Gemtuzumab OzogamicinDose escalation to determine the maximum tolerated dose (MTD) of CPX-351 in combination with Gemtuzumab Ozogamicin in participants with newly diagnosed acute myeloid leukemia. Participants will be treated in cohorts of size three to six and the dosage will be escalated if the clinical toxicity is acceptable. A total of 3 dose levels will be used. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- Provision of signed and dated informed consent form
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Male or female, aged ≥18 and ≤70 years with newly diagnosed any risk AML as defined by ELN 2017 criteria
- For females of child-bearing potential: use of highly effective contraception upon enrollment and during study participation and for an additional 6 months after the end of CPX-351 and Gemtuzumab ozogamicin administration: A female of child-bearing potential 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
- The effects of CPX-351 and gemtuzumab ozogamicin on the developing human fetus are unknown. For this reason, women of child-bearing potential as defined above must have a negative serum or urine pregnancy test within 24 hours prior to beginning study treatment.
Key exclusion criteria (14 listed)
- Prior treatment of AML except hydroxyurea and/or leukapheresis
- Participants with acute promyelocytic leukemia (APL).
- Known current and clinically active central nervous system (CNS) leukemia.
- Severe liver disease (cirrhosis, non-alcoholic steatohepatitis, sclerosing cholangitis) or patients with known Wilson's disease.
- Participants with known active infection with hepatitis B or hepatitis C virus
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 |
|---|---|---|
| VyxeosAlso: daunorubicin-cytarabine, CPX-351Fixed dose of Vyxeos (44 mg/m2 daunorubicin and 100 mg/m2 cytarabine) (Day 1, 3, and 5) in combination with various dose schedules of Gemtuzumab Ozogamicin (GO) | Drug | Dose Escalation |
| Gemtuzumab OzogamicinAlso: MylotargParticipants will be treated at the following dose levels: Dose Level 1 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1 Dose Level 2 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1 and 4 Dose Level 3 - Gemtuzumab Ozogamicin will administered 3mg/m2 on Day 1, 4, 7 | Drug | Dose Escalation |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: Up to 18 Months
Dose escalation will determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Vyxeos plus Gemtuzumab Ozogamicin. The MTD is the highest dose of the combination therapy that dose not cause unacceptable side effects.
4 secondary outcomes
Rate of Complete Remission
Time frame: Up to 18 Months
Measurable Residual Disease
Time frame: Up to 18 Months
Overall Survival
Time frame: Up to 5 years
Relapse Free Survival (RFS)
Time frame: Up to 18 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
- Individual participant dataSharing planned: Undecided
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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.
Onyee Chan, MD principal investigator
Moffitt Cancer Center
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 Vyxeos and Gemtuzumab Ozogamicin in people with Acute Myeloid 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 13 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 27 Feb 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lisa Nardelli (1-813-745-4731, Lisa.Nardelli@moffitt.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) (time frame: Up to 18 Months). 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 27 Feb 2026.