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Acute Myeloid LeukemiaPhase 1/2NCT05322850

Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)

A Phase 1/2 study testing Orca-Q in people with Acute Myeloid Leukemia and Acute Lymphoid Leukemia, run by University of Florida at 4 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This is a first in children prospective study of allogeneic hematopoietic cell transplant using a centrally manufactured engineered donor graft (Orca-Q). The study will assess safety and efficacy of Orca-Q in pediatric patients with hematologic malignancies. Read more about this study

Who is it for?
Ages Up to 50 yearsHealthy volunteers can take part
What is being tested?
Orca-Q
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
4 sitesUnited States
Study dates
16 Aug 2022to Sep 2027
Phase
Phase 1/2
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 Up to 50 years
  • All sexes
  • Healthy volunteers accepted
  • Child, Adult

Who may be able to join

  • Age < 22 years of age at the time of diagnosis of malignancy
  • Eligible for treatment at a pediatric HCT center
  • Patients with the following histopathologically-confirmed diseases are eligible:
  • Acute myeloid, lymphoid, mixed phenotype or undifferentiated leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi) as defined by:
  • Marrow blasts < 5% by morphologic examination
  • Absolute neutrophil count > 1.0 × 109/L
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  • Platelet count > 100 × 109/L
  • Absence of leukemic blasts in the peripheral blood by morphological examination, and
  • No evidence of extramedullary disease
  • CRi: All CR criteria except for residual neutropenia (< 1.0 × 109/L) or thrombocytopenia (< 100 × 109/L)
  • Myeloid or lymphoid blast crisis or accelerated phase developing in the setting of chronic myeloid leukemia is an allowed diagnosis provided that patients are in CR or CRi with regard to the blast crisis
  • Planned to undergo myeloablative allogeneic hematopoetic cell transplant (MA-alloHCT) with a myeloablative conditioning regimen
  • Available donor willing to donate PBSCs:
  • Related donor who is a 7 or 8/8 match for HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods
  • Matched unrelated donor who is a 7 or 8/8 match at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods
  • Haploidentical related donor who is a ≥ 4/8 but < 7/8 match at HLA-A, -B, -C, and -DRB1 (typed using DNA-based high-resolution methods), with at most one mismatch per locus
  • Serum creatinine within normal range for age and estimated GFR by Schwartz equation >70 ml/min/1.73m2. If serum creatinine is higher than normal, obtain creatinine clearance or nuclear medicine glomerular filtration rate. GFR of ≥ 70 mL/minute/1.73m2 is required.
  • Cardiac ejection fraction at rest ≥ 50% by echocardiogram or radionuclide scan (MUGA)
  • Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 70% or pulse ox on RA >95% for patients who cannot cooperate with the PFTs
  • Total bilirubin < 1.5 times upper limit of normal (ULN) (< 3 times if attributed to Gilbert's syndrome) and ALT/AST < 4 times ULN
  • Recipients in screening must screen negative for SARS-CoV-2 RNA using a PCR-based test prior to enrollment as follows:
  • The SARS-CoV-2 testing screening test should be timed such that the results are available prior to the start of the recipient's conditioning regimen. Efforts should be made to minimize the window of time between test result availability and the start of conditioning.
  • Recipients in screening who test positive for SARS-CoV-2 are ineligible but may be considered eligible for future trial participation provided that they are cleared for transplantation per the most current ASTCT guidelines (https://www.astct.org/connect/astct-response-to-covid-19).
  • Subjects must not have more than one active malignancy at the time of enrollment (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen [as determined by the treating physician and approved by the PI] may be included).
  • Written informed consent obtained from the subject and the subject or parental/legal representative permission and subject's assent, if applicable, stating that the subject agrees to comply with all the study-related procedures.
  • Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy throughout the study and for one year after transplantation to minimize the risk of pregnancy.
  • Males with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g ., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for one year after transplantation.
  • Age ≥ 18 and ≤ 50 years at time of enrollment
  • Match to the patient as follows:
  • Matched related donor who is a 7 or 8/8 match for HLA-A, -B, -C, and -DRB1, all typed using DNA-based high resolution methods
  • Matched unrelated donor who is a 7 or 8/8 match at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods
  • Haploidentical related donor who is a ≥ 4/8 but < 7/8 match at HLA-A, -B, -C, and -DRB1 (typed using DNA-based high-resolution methods), with at most one mismatch per locus
  • Willing to donate PBSC for up to two consecutive days
  • Able to donate within North America or Hawaii at a site that will employ a Spectra Optia Apheresis System for post-mobilization apheresis.
  • Meets federal eligibility criteria for donors of viable, leukocyte-rich cells or tissues as defined by 21 CFR § 1271 2018 and all relevant FDA Guidance for Industry.
  • Donors determined to be ineligible, based on the results of required testing and/or screening, may nonetheless be included if either apply, as per 21 CFR § 1271.65 2018:
  • The donor is a first-degree or second-degree blood relative of the recipient
  • Urgent medical need, meaning no comparable human cell product is available and the Recipient is likely to suffer death or serious morbidity without the human cell product, as attested by the investigator.
  • Meets any other criteria for donation as specified by standard NMDP guidelines (NMDP donors) or institutional/FACT standards (non-NMDP donors)

Who cannot join

  • Prior myeloablative allogeneic HCT
  • Currently receiving corticosteroids or other immunosuppressive therapy (topical corticosteroids or physiologic replacement hydrocortisone is allowed)
  • Planned donor lymphocyte infusion (DLI)
  • Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy) or alemtuzumab. Anti-thymocyte globulin is allowed only as specified in the protocol.
  • Positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:
  • a positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing), or
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  • the presence of anti-donor HLA antibody to any of the following HLA loci: HLA-A, -B, -C, -DRB1, -DQB1, -DQA1, -DPB1, or -DPA1, with mean fluorescence intensity (MFI) > 1000 by solid phase immunoassay
  • Lansky play scale < 70% or Karnofsky <70%
  • Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) > 4
  • Documented or suspected bridging fibrosis or liver cirrhosis
  • Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment
  • Seropositive for HIV-1 or -2, HTLV-1 or -2
  • Known allergy or hypersensitivity to, or intolerance of, tacrolimus
  • Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal or Streptomyces avidinii proteins
  • Any uncontrolled autoimmune disease requiring active immunosuppressive treatment
  • Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected
  • Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire study period and for one year following transplantation.
  • Females who are known to be pregnant or breastfeeding and unwilling to cease breastfeeding.
  • Any serious medical condition or abnormality in clinical laboratory tests that, in the Investigator's or Medical Monitor's judgment, precludes the Recipient's safe participation in and completion of the study, or which could affect compliance with the protocol or interpretation of results
  • Administration of a vaccine containing live virus within 30 days prior to the first dose of trial treatment (conditioning regimen). Note: Most flu vaccines are killed viruses, with the exception of the intra-nasal vainer (Flu-Mist) which is an attenuated live virus and therefore prohibited for 30 days prior to first dose. Non-live versions of the COVID-19 vaccine are allowed.
  • Prisoners or subjects who are involuntarily incarcerated, wards of state, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.
  • Evidence of active infection
  • Seropositive for HIV -1 or -2, HTLV-1 or -2
  • Positive for active or chronic Hepatitis B (HBV) based on serology [HBV surface antigen (HBsAg), total anti-Hepatitis B core antibody (HBcAb, IgG and IgM), and Hepatitis B surface antibody (HBsAb)] or by nucleic acid testing (NAT) testing. Donors with ambiguous HBV serology results should be tested for HBV by NAT.
  • Positive for anti-Hepatitis C (HCV) antibody or HCV NAT
  • Potential for Zika virus infection as defined as any of the following:
  • Medical diagnosis of Zika virus infection in the past 6 months
  • Residence in, or travel to, an area with active Zika virus transmission within the past 6 months.
  • Sex within the past 6 months with a person who is known to have either of the risk factors listed above
  • Donors determined to be ineligible based on the results of Zika virus screening may be determined to be eligible if:
  • The donor has no signs or symptoms consistent with active Zika virus infection and either of the following is true: ii. The donor is a first-degree or second-degree blood relative of the recipient iii. Urgent medical need, meaning no comparable human cell product is available and the recipient is likely to suffer death or serious morbidity without the human cell product, as attested by the Investigator
  • Aberrant CD45RA isoform expression by central laboratory testing
  • Females who are pregnant or breastfeeding and unwilling to cease breastfeeding

Trial locations and contacts

4 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Alex Leggett, MS contact

352-273-9785

PMO@cancer.ufl.edu

United States4 sites, 1 recruiting

  • University of Florida

    Gainesville, Florida, 32608

    Beate Greer, 352-294-8744, bgreer01@ufl.edu

    Jordan Milner, MD

    Recruiting

  • University of Miami

    Miami, Florida, 33136

    Suspended

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

    Suspended

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701

    Suspended

Other trials in United States

About this study

Official title: Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT) Recipients With Hematologic Malignancies (HM)

This is a first in children prospective study of allogeneic hematopoietic cell transplant using a centrally manufactured engineered donor graft (Orca-Q). The study will assess safety and efficacy of Orca-Q in pediatric patients with hematologic malignancies.

Being tested
Orca-Q
Keywords
hematologic malignancies, Orca-Q, hematopoietic cell transplant, engineered donor graft, pediatrics
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Biphenotypic, Acute, Disease, Leukemia, Myeloid, Accelerated Phase, Hematologic Neoplasms

What participants receive

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

  • Orca-QBiological

    All transplant recipients enrolled on this study in both the phase I and phase II portions will receive Orca-Q (Orca-Q Prime and Orca-Q Supplement) intravenously following myeloablative conditioning.

Study groups in NCT05322850
Study groupRoleWhat they receive
Orca-QExperimentalNot described
Receives: Biological: Orca-Q

What is being measured

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

  1. Primary graft failure

    Evaluate primary graft failure through day +28, defined as being alive without recovery of neutrophils (achieving an absolute neutrophil count >500/µL for 3 consecutive days) by day +28

    Measured: 28 days

  2. Secondary graft failure

    Evaluate secondary graft failure through day +100, defined as neutrophil engraftment followed by subsequent decline in absolute neutrophil counts to <500 µL, unresponsive to growth factor therapy and in the absence of alternative explanations such as recurrence of the underlying malignant disorder, infections, microangiopathy, medications causing bone marrow suppression or immune-mediated cytopenia.

    Measured: 100 days

Secondary outcomes (10)

  1. Non-relapse mortality (12 months post-transplant)

    Evaluate non-relapse mortality, defined as death without evidence of disease recurrence, at 12 months post-transplant

    Measured: 12 months

  2. Non-relapse mortality (24 months post-transplant)

    Evaluate non-relapse mortality, defined as death without evidence of disease recurrence, at 24 months post-transplant.

    Measured: 24 months

  3. Overall survival

    Determine the overall survival at 24 months post-transplant, as defined as the time from the date of transplant to the date of death from any cause or, for surviving patients, to the date of last follow-up.

    Measured: 24 months

  4. Relapse rate (12 months post-transplant)

    Determine the relapse rate, as defined as the incidence of disease relapse through 12 months post-transplant. Disease relapse is defined as any of the following: ≥ 5% blasts in the bone marrow or peripheral blood, reappearance of pre-transplant cytogenetic abnormality, new evidence or redevelopment of extramedullary disease. Institution of any therapy to treat relapsed disease, such as withdrawal of immunosuppression or donor lymphocyte infusion (DLI), will be considered evidence of relapse regardless of whether any of these are met.

    Measured: 12 months

  5. Relapse rate (24 months post-transplant)

    Determine the relapse rate, as defined as the incidence of disease relapse through 24 months post-transplant. Disease relapse is defined as any of the following: ≥ 5% blasts in the bone marrow or peripheral blood, reappearance of pre-transplant cytogenetic abnormality, new evidence or redevelopment of extramedullary disease. Institution of any therapy to treat relapsed disease, such as withdrawal of immunosuppression or donor lymphocyte infusion (DLI), will be considered evidence of relapse regardless of whether any of these are met.

    Measured: 24 months

  6. Rate of acute GVHD

    Determine the rate of acute GVHD at 6 months post-transplant

    Measured: 6 months

  7. Rate of chronic GVHD

    Determine the rate of chronic GVHD through 24 months post-transplant.

    Measured: 24 months

  8. Serious infection rate

    Determine the rate of serious infections by 12 months post-transplant. A serious infection is defined as any new viral, bacterial, fungal or parasitic infection Common Terminology Criteria for Adverse Events v5.0 grade 2 or higher.

    Measured: 12 months

  9. Treatment-Emergent Adverse Events

    Describe treatment-emergent adverse events through 24 months post-transplant.

    Measured: 24 months

  10. Graft-versus-host disease (GVHD)-free, relapse-free survival

    Evaluate the GVHD-free, relapse-free survival at 12 months post-transplant. GVHD-free, relapse-free survival is defined as freedom from grade III or IV acute GVHD, moderate or severe chronic GVHD, or disease progression or relapse.

    Measured: 12 months

Timeline

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

  1. First posted12 Apr 2022
  2. Study start16 Aug 2022
  3. Primary completionSep 2026 (estimated)
  4. Study completionSep 2027 (estimated)

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05322850
Sponsor's protocol ID
UF-PED-003
Other IDs
OCR41968
Status last verified
Aug 2026
First submitted
31 Mar 2022
Last updated
1 Sep 2026
Results posted
No
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 Orca-Q in people with Acute Myeloid Leukemia and Acute Lymphoid Leukemia, run by University of Florida at 4 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 39 inclusion and 33 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 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 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 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?

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 Alex Leggett, MS (352-273-9785, PMO@cancer.ufl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Primary graft failure (time frame: 28 days). 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?

On the ClinicalTrials.gov record for NCT05322850.

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 1 Sep 2026.

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