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Acute Myeloid LeukemiaPhase 1/2NCT03374332

Fractionated Gemtuzumab Ozogamicin Followed by Non-engraftment Donor Leukocyte Infusions for Relapsed/Refractory Acute Myeloid Leukemia

A Phase 1/2 study testing Gemtuzumab Ozogamicin (GO) and Donor Leukocytes in people with Acute Myeloid Leukemia, run by Brown University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study includes patients with relapsed acute leukemia who have previously been treated with standard treatment that is still present and there is no curative treatment option available. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Gemtuzumab Ozogamicin (GO) and Donor Leukocytes
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
1 siteUnited States
Study dates
31 Dec 2019to 31 Mar 2025
Phase
Phase 1/2
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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologic confirmation of acute myeloid leukemia (AML)
  • Recurrence or progression (including refractory disease) of AML after at least 1 prior standard treatment with progression within 6 months from last treatment.
  • No curative treatment option is available
  • ≥ 4-weeks since prior chemotherapy or radiation to cellular therapy infusion.
  • Age equal to or greater than 18 years.
  • Patients with a history of invasive second malignancy who are disease free for ≥ 2 years.
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  • Patients must have an expected life expectancy of at least 2 months at the time of initiation of treatment
  • No active systemic infections allowed.
  • Patients who have relapsed after standard autologous stem cell infusion are eligible as long as they meet all inclusion criteria and no exclusion criteria. These patients must be out more than 6 months from cell infusion to be eligible for enrollment.
  • DLCO ≥ 40% with no symptomatic pulmonary disease.
  • LVEF ≥ 40% by MUGA or echocardiogram.
  • Creatinine <1.5x ULN or any serum creatinine level associated with a measured or calculated creatinine clearance of >40 mL/min, AST and ALT < 2.5x ULN, Total Bilirubin < 1.5 x ULN
  • Women of childbearing potential and sexually active males must use 2 forms of effective contraception method from time of consent through 6 months after completing treatment (whether last treatment is infusion or drug).
  • Not pregnant or nursing. Women of child bearing potential must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 7 days prior to beginning of treatment (drug). Post-menopausal women (surgical menopause or lack of menses ≥12 months) do not need to have a pregnancy test, please document status.
  • Performance status ≤ 2 (or KPS 70)

Who cannot join

  • Evidence of HIV infection.
  • Any uncontrolled severe, concurrent illness which in the opinion of the treating physician would make this protocol treatment unreasonably hazardous for the patient.
  • Oxygen dependent obstructive pulmonary disease.
  • Failure to demonstrate adequate compliance with medical therapy and follow-up
  • Significant medical or psychiatric illness that would impair the ability to participate in protocol therapy.
  • Previous allogeneic stem cell transplant
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  • Patients who have had previous purine analog (fludarabine, pentostatin, 2-CDA) treatment or treatment with alemtuzumab within 1 year of entering the study
  • Previous history of Veno-occlusive disease/Sinusoidal Obstruction Syndrome
  • Active hepatitis B or C
  • Patients with known active central nervous system leukemia
  • Patients with prior treatment of Gemtuzumab ozogamicin
  • Patients eligible for allogeneic stem cell transplant
  • Donor Eligibility Criteria donor eligibility must be documented and assessed by a separate participating physician other than the treating physician of the recipient.
  • Donors agree to up to 3 donor leukocyte collections as documented by assessing physician (although it is not required that the same donor is used for all leukocyte infusions it is preferable, therefore agreement is important at screening). This must be documented in writing by treating physician.
  • Typing is required at the following loci: HLA A, B, C, and DRB1.
  • Patients with a HLA 0/6 to 3/6(using loci A, B, DR) donor match will be able to participate in this protocol.
  • Donors must be a first or second degree relative.
  • If the patient/recipient is CMV negative, only CMV negative donors are eligible. If the patient/recipient is CMV positive, CMV testing is not required for the donor.
  • Although ABO blood type matching is not required given use of apheresis product preference will be made to ABO compatible donors
  • Donors must be ≥18 years of age to donate.
  • Donor with a history of malignancy other than non-melanoma skin cancers are eligible only if they had a history of a solid tumor malignancy more than five years prior to lymphocyte donation, and are considered to be in longstanding complete remission as documented by a physician. The donor must be healthy and have all testing criteria completed and meet all criteria for stem cell donation.
  • The donor will also be assessed by physician who will document behavioral risk for exposure to infectious agents and diseases. Testing will include the following: Human transmissible spongiform encephalopathy, including Creutzfeldt-Jakob disease, communicable disease risks associated with xenotransplantation, Chlamydia trachomatis and for Neisseria gonorrhea, Treponema pallidum (syphilis).This will exclude the donor.
  • The donor must be in good general health and not have significant cardiopulmonary, renal, endocrine, or hepatic disease as documented in writing by physician.
  • B-HCG urine or serum negative if donor is of childbearing potential within 7 days of PBMC procurement. Not applicable for donors who are Post-menopausal (surgical menopause or lack of menses ≥12 months).
  • Donors must have adequate venous access so leukapheresis can be performed via standard peripheral IV without the need for placement of a central venous catheter.
  • Donor Exclusion Criteria:
  • Evidence of infection for HIV-1, HIV-2, syphilis, hepatitis B or C, HTLV 1 or 2, WNV, Chagas.
  • Uncontrolled diabetes mellitus. If donor has controlled diabetes this must be documented by physician.
  • Active or congestive heart failure syndrome from any cause per donor medical history. Approval for participation by Cardiologist required to be sent to BrUOG.
  • Full cardiac workup by a Cardiologist is required for any history of congestive heart failure syndrome, conduction abnormalities or history of arrhythmia other than treated atrial fibrillation.
  • Active angina pectoris. To be documented by physician
  • Oxygen dependent pulmonary disease. To be documented by physician
  • History of any lymphoid, myeloid or other non-solid malignancy. To be documented by physician
  • Failure to receive full informed consent.
  • Inadequate renal and/or hepatic function per medical history. To be documented by physician
  • Known history of cirrhosis or active liver disease per medical history. To be documented by physician
  • History of transplantation. To be documented by physician.
  • A donor in whom plasma dilution sufficient to affect the results of communicable disease testing is suspected, unless: (A) You test a specimen taken from the donor before transfusion or infusion and up to 7 days before recovery of cells or tissue; or (B) You use an appropriate algorithm designed to evaluate volumes administered in the 48 hours before specimen collection, and the algorithm shows that plasma dilution sufficient to affect the results of communicable disease testing has not occurred.
  • Clinical situations in which you must suspect plasma dilution sufficient to affect the results of communicable disease testing will exclude the donor. Such situations include, but are not limited to the following:(A) Blood loss is known or suspected in a donor over 12 years of age, and the donor has received a transfusion or infusion of any of the following, alone or in combination:
  • More than 2,000 milliliters (mL) of blood (e.g., whole blood, red blood cells) or colloids within 48 hours before death or specimen collection, whichever occurred earlier, or
  • More than 2,000 mL of crystalloids within 1 hour before death or specimen collection, whichever occurred earlier.

Trial locations and contacts

1 site in 1 country.

United States1 site
  • Rhode Island Hospital

    Providence, Rhode Island, 02903

Other trials in United States

About this study

This study includes patients with relapsed acute leukemia who have previously been treated with standard treatment that is still present and there is no curative treatment option available. Researchers are studying whether the drug Gemtuzumab Ozogamicin, followed by an infusion of blood cells called leukocytes from a donor, can stimulate the immune system to potentially fight the leukemia. Gemtuzmab ozogamicin is a class of drugs known as an antibody drug conjugate. The drug is given on days 1,4,7. It is infused, attaches to cells with a certain marker on the surface (the majority of which would be leukemia cells). The drug is then internalized and the chemotherapy drug becomes activated. Gemtuzumab is currently FDA approved for the treatment of acute myeloid leukemia.

The infusion of leukocytes to stimulate the immune system to fight your leukemia is investigational and has not been proven to cure cancer. This combination of Gemtuzumab Ozogamicin and donor leukocytes is not an FDA approved treatment and is investigational.

Initially a total of 6 patients will be included in the study to assess the safety of the treatment. Once 6 patients have been treated and no unacceptable toxicities are seen, more patients will be enrolled. The study will treat up to 18 patients on the study.

Being tested
Gemtuzumab Ozogamicin (GO), Donor Leukocytes
Keywords
Refractory, progression, recurrence
Medical subject headings
Leukemia, Myeloid, Acute, Disease Progression, Recurrence

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Gemtuzumab Ozogamicin (GO)Drug

    Also called Mylotarg

    Patients will be given gemtuzumab ozogamicin on days 1,4, and 7. Capped at 4.5mg individual doses. Total doses capped at 13.5mg.

  • Donor LeukocytesOther

    The product will be administered unprocessed on day 8/24 hours post GO (same day as leukapheresis) with a minimum of CD3+ cells and maximum of CD3+ cells/kg irrespective of the number of CD34+ cells.

Study groups in NCT03374332
Study groupRoleWhat they receive
Phase 1 Treatment 1: Gemtuzumab Ozogamicin and DLI Dose Level 1ExperimentalDay 8: The product will be administered unprocessed on day 8 (same day as leukapheresis) with a minimum of 1x10^7 CD3+ cells and maximum of 2x10^7 CD3+ cells/kg irrespective of the number of CD34+ cells. Patients who show a transient response to therapy can receive up to two additional donor leukocyte infusions with GO 6mg/m2 (2mg/m2 each day, capped at 4.5mg) for all patients regardless of age administered on days 1,4, and 7, no sooner than 35 days status post their last cellular infusion.
Receives: Drug: Gemtuzumab Ozogamicin (GO); Other: Donor Leukocytes
Phase 1 Treatment 2: Gemtuzumab Ozogamicin and DLI Dose Level 2ExperimentalDay 8: The product will be administered unprocessed on day 8 (same day as leukapheresis) with a minimum of 1x10^8 CD3+ cells and maximum of 2x10^8 CD3+ cells/kg irrespective of the number of CD34+ cells. Patients who show a transient response to therapy can receive up to two additional donor leukocyte infusions with GO 6mg/m2 (2mg/m2 each day, capped at 4.5mg) for all patients regardless of age administered on days 1,4, and 7, no sooner than 35 days status post their last cellular infusion.
Receives: Drug: Gemtuzumab Ozogamicin (GO); Other: Donor Leukocytes
Phase 2 Treatment 3: Gemtuzumab Ozogamicin and DLI Dose Level (MTD determined from Phase1)ExperimentalMaximum Tolerated Dose (MTD) determined from Phase 1 portion of study Day 8: The product will be administered unprocessed on day 8 (same day as leukapheresis) with a dose of 1x10^7 - 2x10^7 CD3+ cells OR 1x10^8 - 2x10^8 CD3+ cells/kg irrespective of the number of CD34+ cells. MTD AS DETERMINED BY PHASE 1 Patients who show a transient response to therapy can receive up to two additional donor leukocyte infusions with GO 6mg/m2 (2mg/m2 each day, capped at 4.5mg) for all patients regardless of age administered on days 1,4, and 7, no sooner than 35 days status post their last cellular infusion.
Receives: Drug: Gemtuzumab Ozogamicin (GO); Other: Donor Leukocytes

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Phase 1: Determine the RP2D or MTD of Nonengraftment Donor Leukocyte Infusions With Fractionated Gemtuzumab Ozogamicin in Patients With Relapsed/Refractory AML Defined as Dose Level Without Dose Limiting Toxicities.

    The MTD of non-engraftment donor leukocyte infusions administered with fractionated gemtuzumab ozogamicin in patients with relapsed/refractory AML is determined by the dose level achieved with no Dose limiting toxicities. Measure = participants without DLTs.

    The recommended phase 2 dose (RP2D) or maximum tolerated dose (MTD) was evaluated by utilizing a 3+3 design to determine if 1-2x108 CD3 cells/kg can be safely administered with fractionated Gemtuzumab Ozogamicin administered on days 1,4 and 7 (total dose capped at 13.5 mg). The leukocyte infusion is administered on day 8 after completion of GO day 7.

    Two DLI dose levels well be assessed:

    DLI Dose Level 1: 1-2x107 CD3+ cells/kg DLI Dose Level 2: 1-2x108 CD3+ cells/kg The RP2D/MTD is determined by the dose level achieved with no Dose limiting toxicities. Measure = participants without DLTs through 35 days post DLI.

    Measured: 35 days post infusion

  2. Response Rate

    Response rate of infusional gemtuzumab ozogamicin followed by nonengraftment donor leukocyte infusions in patients with refractory acute myeloid leukemia.

    Bone Marrow Biopsy to be done in patients thought to have responded.

    Complete remission or complete remission with incomplete count recovery, after one cycle of treatment. Rationale for including CRi: CRi has previously been used in gemtuzumab ozogamicin trials because of incomplete platelet recovery seen with this drug.

    Measured: 4 weeks post infusion

Secondary outcomes (3)
  1. Number of Participants Reaching 24 Months Progression Free Survival

    Progression free survival of infusional gemtuzumab ozogamicin followed by nonengraftment donor leukocyte infusions in patients with refractory acute myeloid leukemia.

    Measured: Through 2 years post end of treatment

  2. Number of Participants Who Met the Objective for Overall Survival at 2 Years

    Overall survival of infusional gemtuzumab ozogamicin followed by nonengraftment donor leukocyte infusions in patients with refractory acute myeloid leukemia.

    Measured: Through 2 years post end of treatment

  3. Number of Participants With Dose Limiting Toxicities as Defined by the Protocol

    Number of participants with dose limiting toxicities following treatment with gemtuzumab ozogamicin followed by nonengraftment donor leukocyte infusions in patients with refractory acute myeloid leukemia. DLT's defined in the protocol as

    • IBMTR Severity Index C \& D graft versus host disease
    • Grade 3 or 4 infusion related reaction
    • Any grade 3 non-hematological toxicities not directly resulting from patient's disease (with some exclusions specified in the protocol)
    • Any grade 4 related or unrelated non-hematological toxicity not directly resulting from patient's disease
    • Development of SOS
    • Discontinuation of treatment due to an adverse event of any grade is considered a DLT unless the AE is clearly and solely related to the underlying disease.

    All Grade 5 toxicities are considered DLTs unless clearly and solely related to the underlying disease.

    Measured: Start of treatment through 30 days post the final infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Dec 2017
  2. Study start31 Dec 2019
  3. Primary completion26 Sep 2023
  4. Study completion31 Mar 2025

Resources

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03374332
Sponsor's protocol ID
BrUOG 345
Status last verified
Jun 2026
First submitted
29 Nov 2017
Last updated
23 Jul 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Gemtuzumab Ozogamicin (GO) and Donor Leukocytes in people with Acute Myeloid Leukemia, run by Brown 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 15 inclusion and 41 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 23 Jul 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 combined 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.

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 23 Jul 2026). 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?

Phase 1: Determine the RP2D or MTD of Nonengraftment Donor Leukocyte Infusions With Fractionated Gemtuzumab Ozogamicin in Patients With Relapsed/Refractory AML Defined as Dose Level Without Dose Limiting Toxicities. (time frame: 35 days post infusion). The study lists 2 primary outcomes in all. 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 23 Jul 2026.

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