Gemtuzumab Ozogamicin for Acute Myeloid Leukemia

Recruiting at 1 trial location

Overseen By Mary-Elizabeth Percival

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Washington

Must not be taking: Investigational agents

Disqualifiers: Chemotherapy, Radiation, Uncontrolled illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 5 weeks

36 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of gemtuzumab ozogamicin (Mylotarg) for individuals with acute myeloid leukemia (AML), a type of blood cancer. The aim is to determine if this drug can effectively target and kill cancer cells with the CD33 marker. The trial includes participants who have already undergone chemotherapy and have a low level of cancer cells remaining, detectable by specific tests. Eligible participants should have AML with CD33 markers and no major health issues, such as uncontrolled infections or unstable heart conditions. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot have had chemotherapy or radiation therapy within 14 days before starting the study, and you cannot be on other investigational drugs.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that gemtuzumab ozogamicin is generally safe for people with acute myeloid leukemia. Studies have found that using this treatment before a stem cell transplant does not increase the risk of liver problems or early death compared to other treatments. One study used gemtuzumab ozogamicin with standard chemotherapy and found it improved patient outcomes without adding safety concerns. These findings suggest that the treatment is well-tolerated and does not cause unexpected issues for most patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Gemtuzumab ozogamicin is unique because it combines an anti-cancer drug with an antibody to specifically target and attack leukemia cells. Unlike standard chemotherapy, which can affect healthy cells and cause broader side effects, gemtuzumab ozogamicin zeroes in on CD33, a protein commonly found on the surface of leukemia cells in Acute Myeloid Leukemia (AML). This targeted approach not only aims to improve treatment effectiveness but also reduces harm to healthy cells, making it a promising option with potentially fewer side effects. Researchers are excited about this precision-targeting method as it represents a significant advancement in AML treatment.

What evidence suggests that gemtuzumab ozogamicin might be an effective treatment for acute myeloid leukemia?

Research has shown that gemtuzumab ozogamicin can help treat acute myeloid leukemia (AML). One study found that it helped 31.6% of patients with AML that had returned after treatment. For patients with a moderate risk of AML, this treatment effectively fought cancer. When combined with standard chemotherapy, gemtuzumab ozogamicin improved outcomes for patients newly diagnosed with moderate-risk AML. Additionally, for a specific type of AML called CBF-AML, using gemtuzumab at the start of treatment helped patients live longer after two years. These findings suggest that gemtuzumab ozogamicin could benefit certain groups of AML patients. Participants in this trial will receive gemtuzumab ozogamicin as part of their treatment regimen. ^{2 3 5 6 7}

Who Is on the Research Team?

Mary-Elizabeth Percival

Principal Investigator

Fred Hutch/University of Washington Cancer Consortium

Are You a Good Fit for This Trial?

This trial is for people aged 2 and older with Acute Myeloid Leukemia (AML) who have already had at least one round of standard chemo. They must show minimal remaining disease, be in good enough health to perform daily activities, and not have certain types of AML like APL. Pregnant women can't join, and those able to conceive must test negative for pregnancy.

Inclusion Criteria

My bilirubin levels are within twice the normal range, or I have Gilbert's disease.

My child's bilirubin levels are within the normal range for their age, or they have Gilbert's disease.

My kidney function is good, with a creatinine level of 2.0 mg/dL or less.

Exclusion Criteria

I have not had chemotherapy or radiation in the last 14 days.

Subjects may not be receiving other investigational agents.

I do not have any severe illnesses that would stop me from following the study's requirements.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive gemtuzumab ozogamicin intravenously on days 1, 4, 7. Treatment continues for 35 days in the absence of disease progression or unacceptable toxicity.

5 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Gemtuzumab Ozogamicin

Trial Overview

The trial tests fractionated Gemtuzumab Ozogamicin (GO), a targeted antibody-chemo combo that seeks out cancer cells by attaching to CD33 receptors on their surface. It's given to patients whose AML isn't fully gone after treatment but is low ('measurable residual disease').

How Is the Trial Designed?

Group I: Treatment (gemtuzumab ozogamicin) Experimental Treatment 2 Interventions

Patients receive gemtuzumab ozogamicin IV on days 1, 4, 7. Treatment continues for 35 days in the absence of disease progression or unacceptable toxicity. Responders and non-responders, without significant adverse events during the first course, may receive a second course of gemtuzumab ozogamicin within 60 days after course 1.

Gemtuzumab Ozogamicin is already approved in United States, European Union for the following indications:

Approved in United States as Mylotarg for:

• Acute myeloid leukemia (AML)

Approved in European Union as Mylotarg for:

• Acute myeloid leukemia (AML)

Who Is Running the Clinical Trial?

University of Washington

Lead Sponsor

Trials

1,858

Recruited

2,023,000+

Pfizer

Industry Sponsor

Trials

4,712

Recruited

50,980,000+

Known For

Vaccine Innovations

Top Products

Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Published Research Related to This Trial

Gemtuzumab ozogamicin (Mylotarg) is an effective treatment for relapsed acute myeloid leukemia (AML), targeting CD33 on leukemic cells and using a potent cytotoxic agent to induce cell death, with promising results from early phase I/II studies.

While it shows significant efficacy, there are concerns about potential adverse effects, including veno-occlusive disease and increased liver toxicity, which are important considerations as it is being tested in new patient populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Early phase I/II trials with gemtuzumab ozogamicin (Mylotarg) in acute myeloid leukemia. Nabhan, C., Tallman, MS.[2019]

Gemtuzumab ozogamicin is an effective treatment for patients with CD33 positive acute myeloid leukemia (AML) in first relapse, showing an overall response rate of 30% in Phase 2 trials involving 142 patients, with a complete response rate of 16%.

While it offers a potentially better tolerability profile compared to conventional chemotherapy, it carries risks of significant side effects, including myelosuppression, hepatotoxicity, and severe allergic reactions, necessitating careful patient monitoring and management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Approval summary: gemtuzumab ozogamicin in relapsed acute myeloid leukemia. Bross, PF., Beitz, J., Chen, G., et al.[2022]

Gemtuzumab ozogamicin is an effective treatment option for relapsed acute myeloid leukemia (AML), as demonstrated in Phase II trials, showing promising results in patients with poor prognosis.

While gemtuzumab ozogamicin has an acceptable safety profile as a standalone treatment, there is a significant risk of veno-occlusive disease of the liver when used in combination with chemotherapy or during hematopoietic stem cell transplants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gemtuzumab ozogamicin in the treatment of acute myeloid leukemia. Stadtmauer, EA.[2019]

Gemtuzumab ozogamicin, the first antibody-drug conjugate approved for CD33-positive acute myeloid leukemia (AML), was initially withdrawn from the market due to lack of clinical benefit, but later studies with new dosing regimens demonstrated its efficacy.

Pharmacokinetic modeling revealed significant relationships between drug exposure and clinical outcomes, leading to its full FDA approval in 2017 for both newly diagnosed and relapsed AML in adults and children aged 2-17 years.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetic/Pharmacodynamic Modeling to Support the Re-approval of Gemtuzumab Ozogamicin. Fostvedt, LK., Hibma, JE., Masters, JC., et al.[2020]

Gemtuzumab ozogamicin is an effective treatment for older adults (≥60 years) with CD33-positive acute myeloid leukemia (AML) in first relapse, achieving complete remission in about 25% of patients.

While it has a tolerability profile that is generally acceptable, serious side effects like hepatotoxicity can occur, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Spotlight on gemtuzumab ozogamicin in acute myeloid leukaemia. Fenton, C., Perry, CM.[2018]

Gemtuzumab ozogamicin (GO) was effective in treating CD33-positive acute myeloid leukemia (AML), achieving a 26% remission rate among 277 patients, with a median recurrence-free survival of 6.4 months for those who achieved complete remission.

The safety profile of GO was generally acceptable, with high rates of expected side effects like neutropenia (98%) and thrombocytopenia (99%), but relatively low incidences of severe infections such as sepsis (17%) and pneumonia (8%).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Final report of the efficacy and safety of gemtuzumab ozogamicin (Mylotarg) in patients with CD33-positive acute myeloid leukemia in first recurrence. Larson, RA., Sievers, EL., Stadtmauer, EA., et al.[2022]

Gemtuzumab ozogamicin is an effective treatment for older patients (≥60 years) with CD33-positive acute myeloid leukaemia (AML) in first relapse, achieving complete remission in about 25% of cases.

While it has a tolerability profile that is generally acceptable, serious side effects like hepatotoxicity can occur, highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Gemtuzumab ozogamicin: a review of its use in acute myeloid leukaemia. Fenton, C., Perry, CM.[2022]

In a study of 142 patients with CD33-positive acute myeloid leukemia (AML) in first relapse, 30% achieved remission after treatment with Mylotarg (gemtuzumab ozogamicin), indicating its efficacy as a targeted chemotherapy agent.

While Mylotarg was associated with some adverse effects, such as myelosuppression and elevated liver enzymes, it had a favorable safety profile overall, with low incidences of severe nausea, vomiting, and no significant cardiotoxicity or hair loss.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of gemtuzumab ozogamicin in patients with CD33-positive acute myeloid leukemia in first relapse. Sievers, EL., Larson, RA., Stadtmauer, EA., et al.[2022]

Gemtuzumab ozogamicin is a targeted chemotherapy agent that effectively treats relapsed acute myeloid leukemia (AML) by specifically targeting the CD33 antigen on leukemic cells, which helps reduce overall toxicity compared to conventional chemotherapy.

This treatment has shown particular efficacy and tolerability in older patients (60 years and above) with relapsed AML, providing a crucial option for those who often have limited treatment alternatives.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nursing implications of mylotarg: a novel antibody-targeted chemotherapy for CD33+ acute myeloid leukemia in first relapse. Shannon-Dorcy, K.[2012]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10813452/

Gemtuzumab Ozogamicin in Acute Myeloid Leukemia

A study showed that when GO was administered as a single agent, it appeared to be an effective option for treating relapsed AML, with a 31.6% ...

2.

ashpublications.org

ashpublications.org/blood/article/142/20/1673/498629/Gemtuzumab-ozogamicin-in-AML-the-next-chapter

Gemtuzumab ozogamicin in AML: the next chapter | Blood

For patients with intermediate-risk AML, there was significant antileukemic efficacy, but allogeneic HCT played an important role in increasing ...

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S2213048925000275

Real-World Efficacy and Safety of Gemtuzumab ...

Gemtuzumab ozogamicin plus standard induction chemotherapy “3 + 7″ has been reported to improve outcomes of newly diagnosed intermediate cytogenetic risk AML, ...

4.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/16116598/

Final report of the efficacy and safety of gemtuzumab ...

The median recurrence-free survival was 6.4 months for patients who achieved CR and 4.5 months for patients who achieved CRp. Although expected ...

5.

nature.com

nature.com/articles/s41375-025-02700-9

Gemtuzumab ozogamicin in first-line treatment of CBF-AML

In a contemporary cohort, our data show an absolute survival advantage of 10% at 2 years with GO-containing induction therapy and acceptable ...

6.

ascopubs.org

ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.6516

Safety outcomes in patients with acute myeloid leukemia ...

Conclusions: The use of GO appears to be safe prior to HSCT in adults with AML. Rates for 100-day VOD and TRM were comparable to those ...

7.

haematologica.org

haematologica.org/article/view/8727

Gemtuzumab ozogamicin for de novo acute myeloid ...

The final results of this study indicate that GO added to standard chemotherapy significantly prolongs EFS in patients with newly diagnosed de novo AML and has ...