Acute Myeloid LeukemiaPhase 1NCT06551584
Trial for Patients w/ Advanced Hematologic Malignancies Undergoing Allogeneic HCT
A Phase 1 study testing ORCA-T in people with Acute Myeloid Leukemia and Acute Lymphoid Leukemia, run by Stanford University at 1 site in United States.
Trial summary
The study goal is to characterize the safety of the combination of Orca-T with dual agent GVHD prophylaxis. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Mixed Phenotype Acute Leukemia
- What is being tested?
- ORCA-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2025to Dec 2026
- 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
- Eligible diseases:
- Acute myeloid, lymphoid or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi) as defined in Section 6.1.3; with or without the presence of known minimal residual disease, or
- Myelodysplasic syndrome (MDS) myelodysplastic syndromes eligible for alloHSCT and/or treatment-related MDS <10% blasts
- Age ≥ 18 and ≤ 70 years at the time of enrollment.
- Eligible for myeloablative alloHCT including one of two the myeloablative conditioning regimens (fractionated total body irradiation plus cyclophosphamide or busulfan, fludarabine, and thiotepa)
- Has a related or unrelated donor available who is 7/8 match (single allele mismatched) at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods.
Show all 15Show fewer
- Estimated glomerular filtration rate (eGFR) ≥ 50 mL/minute or creatinine < 2 mg/dL.
- Cardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA).
- Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50%.
- Total bilirubin < 2 times upper limit of normal (ULN) (patients with Gilbert's syndrome may be included once hemolysis has been excluded).
- Ability to understand and the willingness to provide written informed consent.
- Negative serum or urine beta-HCG test in females of childbearing potential (FCBP) within 3 weeks of enrollment.
- A female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
- Able to give informed consent. Legal authorized representative (LAR) is permitted if subject is cognitively able to provide verbal assent.
- Karnofsky Performance Score ≥70%
Who cannot join
- Prior allogeneic HCT.
- Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed.
- Planned donor lymphocyte infusion (DLI).
- Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy), peri-transplant anti-thymocyte globulin (ATG), or alemtuzumab. For patients that have previously been exposed to a T cell-depleting agent, a 5 half-life washout of the agent must occur prior to planned Day 0 (day of infusion of Orca-T HSPC and Tregs ).
- Recipient positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:
- Positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing), or
Show all 18Show fewer
- 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.
- Uncontrolled bacterial, viral, or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment including known, active tuberculosis infection.
- Seropositive for HIV-1 or -2, HTLV-1 or -2, Hepatitis B sAg, and/or Hepatitis C antibody.
- History of hepatitis B or hepatitis C is permitted if viral load is undetectable per quantitative PCR and/or NAT. In this case, monitoring for hepatitis B or hepatitis C by PCR at 3, 6, and 12 months is recommended.
- Known allergy or hypersensitivity to, or intolerance of, any investigational agent or ingredient therein, or planned GVHD prophylactic medications.
- Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal or Streptomyces avidinii proteins.
- Any uncontrolled autoimmune disease requiring active immunosuppressive treatment.
- Concurrent malignancy diagnosed within 12 months of enrollment, except non-melanoma skin cancers that have been curatively resected.
- Females of childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use effective forms of birth control or abstinence for one year after transplantation.
- (FCBP definition: A female of childbearing potential (FCBP) is a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months).
- History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment. History of stroke or pulmonary embolism within 6 months of enrollment.
- Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator'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.
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
Stanford University
Palo Alto, California, 94304
Alyssa Kenegai, 650-736-1596, akanegai@stanford.edu
Lori Muffly, MD, MS
About this study
Official title: Trial for Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation From an HLA-Mismatched Donor (7/8) With Orca-T
The study goal is to characterize the safety of the combination of Orca-T with dual agent GVHD prophylaxis.
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndromes
- Being tested
- ORCA-T
- Keywords
- Orca-T, Hematopoietic Cell Transplantation, Advanced Hematologic Malignancies
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Biphenotypic, Acute, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ORCA-TDrug
On Day 0, participants will receive an infusion of Orca-T HSPCs and Orca-T Tregs. On Day +2 or +3 (between approximately 48 to 72 hours of Day 0), patients will receive an infusion of the Orca-T Tcons. There is no dose escalation or de-escalation planned for the Orca-T investigational product.
| Study group | Role | What they receive |
|---|---|---|
| ORCA-T + (tacrolimus and ruxolitinib) | Experimental | Dual-agent tacrolimus starting on the day after Tcon infusion (Day +3 or Day +4) and ruxolitinib starting on the day after tacrolimus (Day +4 or Day +5). Receives: Drug: ORCA-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Patients Without Severe Transplant-Related Adverse Events at Day +100
The proportion of patients who, at Day +100 have not experienced any severe transplant-related adverse events including non-relapse mortality (NRM), primary graft failure, acute GVHD (grade 2-4), acute GVHD grade 3-4, or opportunistic infections (grade 4-5).
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2024
- Study start1 Dec 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (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 Stanford UniversityThe 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
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Incyte Corporation
- Responsible party
- Sponsor
- Investigators
- Lori Muffly, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06551584
- Sponsor's protocol ID
- IRB-73393
- Other IDs
- NCI-2025-01029
- Status last verified
- Jun 2026
- First submitted
- 9 Aug 2024
- Last updated
- 23 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial for Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation From an HLA-Mismatched Donor (7/8) With Orca-T
- ClinicalTrials.gov ID
- NCT06551584
- Acronym / sponsor ID
- IRB-73393
- Conditions
- Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Mixed Phenotype Acute Leukemia, Myelodysplastic Syndromes
- Keywords
- Orca-T, Hematopoietic Cell Transplantation, Advanced Hematologic Malignancies
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Incyte Corporation
- Study start
- 1 Dec 2025 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 13 Aug 2024 (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 study goal is to characterize the safety of the combination of Orca-T with dual agent GVHD prophylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ORCA-T + (tacrolimus and ruxolitinib) | Experimental | Drug: ORCA-TDual-agent tacrolimus starting on the day after Tcon infusion (Day +3 or Day +4) and ruxolitinib starting on the day after tacrolimus (Day +4 or Day +5). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Eligible diseases:
- Age ≥ 18 and ≤ 70 years at the time of enrollment.
- Eligible for myeloablative alloHCT including one of two the myeloablative conditioning regimens (fractionated total body irradiation plus cyclophosphamide or busulfan, fludarabine, and thiotepa)
- Has a related or unrelated donor available who is 7/8 match (single allele mismatched) at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods.
- Estimated glomerular filtration rate (eGFR) ≥ 50 mL/minute or creatinine < 2 mg/dL.
Key exclusion criteria (18 listed)
- Prior allogeneic HCT.
- Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed.
- Planned donor lymphocyte infusion (DLI).
- Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy), peri-transplant anti-thymocyte globulin (ATG), or alemtuzumab. For patients that have previously been exposed to a T cell-depleting agent, a 5 half-life washout of the agent must occur prior to planned Day 0 (day of infusion of Orca-T HSPC and Tregs ).
- Recipient positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:
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 |
|---|---|---|
| ORCA-TOn Day 0, participants will receive an infusion of Orca-T HSPCs and Orca-T Tregs. On Day +2 or +3 (between approximately 48 to 72 hours of Day 0), patients will receive an infusion of the Orca-T Tcons. There is no dose escalation or de-escalation planned for the Orca-T investigational product. | Drug | ORCA-T + (tacrolimus and ruxolitinib) |
Primary outcome
Proportion of Patients Without Severe Transplant-Related Adverse Events at Day +100
Time frame: 18 months
The proportion of patients who, at Day +100 have not experienced any severe transplant-related adverse events including non-relapse mortality (NRM), primary graft failure, acute GVHD (grade 2-4), acute GVHD grade 3-4, or opportunistic infections (grade 4-5).
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 23 Jun 2026.
Related trials for Acute Myeloid Leukemia
RecruitingObservational study
A Long-term Follow-up Study of Patients With ARD103 CAR-T Cell Therapies
Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia, in Relapsed or Refractory
RecruitingPhase 2
MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
RecruitingPhase 1
225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
RecruitingPhase 2
Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MYELOMATCH Treatment Trial)
Core Binding Factor Acute Myeloid Leukemia
RecruitingPhase 1/2
Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)
Acute Myeloid Leukemia, Acute Lymphoid Leukemia
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.
Lori Muffly principal investigator
Stanford University
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 ORCA-T in people with Acute Myeloid Leukemia and Acute Lymphoid Leukemia, run by Stanford 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 18 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 23 Jun 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 Alyssa Kanegai ((650) 736-1596, akanegai@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Patients Without Severe Transplant-Related Adverse Events at Day +100 (time frame: 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 23 Jun 2026.