A Phase 3 interventional study of Orca-T and Standard-of-Care in Acute Myeloid Leukemia, Acute Lymphoid Leukemia and Mixed Phenotype Acute Leukemia, sponsored by Orca Biosystems, Inc.. Active, not recruiting at 19 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-03-04.
Sponsored by Orca Biosystems, Inc. · Phase 3, Interventional, and Treatment
This study will evaluate the safety, tolerability, and efficacy of Orca-T, an allogeneic stem cell and T-cell immunotherapy biologic manufactured for each patient (transplant recipient) from the mobilized peripheral blood of a specific, unique donor. It is composed of purified hematopoietic stem and progenitor cells (HSPCs), purified regulatory T cells (Tregs), and conventional T cells (Tcons) in participants undergoing myeloablative allogeneic hematopoietic cell transplant transplantation for hematologic malignancies.
This posting represents the Phase III component of Precision-T. The Precision-T Ph1b component is described under NCT04013685.
Cross reference NCT04013685
Key Inclusion Criteria:
Diagnosed with one of the following diseases:
Planned to undergo MA-alloHCT including one of the following myeloablative conditioning regimens:
Key Exclusion Criteria:
For patients randomized to the Orca-T arm, Orca-T will be administered after myeloablative conditioning regimen. Single-agent GVHD prophylaxis with tacrolimus will be administered following Tcon infusion (generally Day +3).
Biological: Orca-T
For patients randomized to the standard-of-care control arm, an unmanipulated allograft derived from the peripheral blood of a matched donor will be administered after a myeloablative conditioning regimen. Dual-agent prophylaxis consisting of tacrolimus plus methotrexate will be administered starting on Day -3.
Biological: Standard-of-Care
an allogeneic stem cell and T-cell immunotherapy biologic
unmanipulated donor allograft
Also known as: SOC
Event-free at 12 Months for Moderate or Severe Chronic Graft-versus-Host-Disease-free Survival (cGFS) Per Endpoint Adjudication Committee (EAC)
Event-free rate estimated at 12 months for cGFS per EAC, which is defined as the time from date of allogeneic hematopoietic cell transplantation (alloHCT) (ie, day 0) to date of death from any cause or first onset of moderate or severe chronic graft-versus-host disease (cGVHD) (graded per NIH consensus criteria), whichever was earliest, within 2 years after day 0. cGVHD was assessed and graded by EAC blinded to treatment assignment. Each participant in the study is followed for up to 730 days after transplant. Primary analysis was event driven (ie, when 56 participants had died or had moderate or severe cGVHD) and was not based on duration of follow-up. The analysis was conducted using all available data at the time that the 56th event occurred, and event-free rate at 12 months was estimated regardless of length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
Time frame: Day 0 through 730 days after transplantation
Event Rate at 12 Months for Time to Moderate or Severe cGVHD Per EAC
Event rate estimated at 12 months for time from alloHCT to first onset of moderate or severe cGVHD defined by NIH consensus criteria within 2 years after day 0. Death within 2 years after day 0 without prior moderate or severe cGVHD was considered a competing event. cGVHD was assessed and graded by an independent EAC. Each participant in the study is followed for up to 730 days after transplant. The primary analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe chronic GVHD) and was not based on the duration of follow-up. Therefore, the analysis was conducted using all available data at the time that the 56th cGFS event occurred, and the event rate of moderate or severe cGVHD at 12 months was estimated regardless of the length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
Time frame: Day 0 through 730 days after transplantation
Event-free at 12 Months for Overall Survival (OS)
Event-free rate estimated at 12 months for OS, which is defined as time from randomization to death from any cause. Each participant in the study is followed for up to 730 days after transplant. The analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe chronic GVHD) and was not based on the duration of follow-up. Therefore, the analysis was conducted using all available data at the time that the 56th cGFS event occurred, and the event-free rate of OS at 12 months was estimated regardless of the length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
Time frame: Up to 730 days after end of enrollment
Event-free Rate at 12 Months for Graft-versus-host Disease-free and Relapse-free Survival (GRFS) Per EAC
Event-free rate estimated at 12 months for GRFS, which is defined as time from alloHCT to death from any cause, relapse, the first onset of grade 3 or 4 acute GVHD (graded per Mount Sinai aGVHD International Consortium \[MAGIC\] criteria), or the first onset of moderate or severe cGVHD (graded per NIH consensus criteria), whichever is earliest, within 2 years from day 0 as assessed by independent EAC. Each participant in the study is followed for up to 730 days after transplant. The analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe cGVHD) and not based on duration of follow-up. Therefore, analysis was conducted using all available data at the time that the 56th cGFS event occurred, and event-free rate of GRFS at 12 months was estimated regardless of length of follow-up for individual participants. At time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
Time frame: Day 0 through 730 days after transplantation
187 participants were randomized to treatment from 19 study centers, of which 182 participants were treated
| Milestone | Orca-T | Standard of Care |
|---|---|---|
| Started | 93 | 94 |
| Participants treated | 88 | 94 |
| Completed | 0 | 2 |
| Not completed | 93 | 92 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Death | 3 | 9 |
| Withdrew: Lost to follow-up | 1 | 0 |
| Withdrew: Physician decision | 1 | 4 |
| Withdrew: Withdrawal by subject | 3 | 5 |
| Withdrew: Progression/relapse | 14 | 9 |
| Withdrew: Received standard of care | 1 | 0 |
| Withdrew: Ongoing | 70 | 64 |
Event-free rate estimated at 12 months for cGFS per EAC, which is defined as the time from date of allogeneic hematopoietic cell transplantation (alloHCT) (ie, day 0) to date of death from any cause or first onset of moderate or severe chronic graft-versus-host disease (cGVHD) (graded per NIH consensus criteria), whichever was earliest, within 2 years after day 0. cGVHD was assessed and graded by EAC blinded to treatment assignment. Each participant in the study is followed for up to 730 days after transplant. Primary analysis was event driven (ie, when 56 participants had died or had moderate or severe cGVHD) and was not based on duration of follow-up. The analysis was conducted using all available data at the time that the 56th event occurred, and event-free rate at 12 months was estimated regardless of length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
| Percentage of participants | Orca-T | Standard of Care |
|---|---|---|
| Event-free at 12 Months for Moderate or Severe Chronic Graft-versus-Host-Disease-free Survival (cGFS) Per Endpoint Adjudication Committee (EAC) | 78.0 (65.0 to 86.6) | 38.4 (26.2 to 50.5) |
Event rate estimated at 12 months for time from alloHCT to first onset of moderate or severe cGVHD defined by NIH consensus criteria within 2 years after day 0. Death within 2 years after day 0 without prior moderate or severe cGVHD was considered a competing event. cGVHD was assessed and graded by an independent EAC. Each participant in the study is followed for up to 730 days after transplant. The primary analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe chronic GVHD) and was not based on the duration of follow-up. Therefore, the analysis was conducted using all available data at the time that the 56th cGFS event occurred, and the event rate of moderate or severe cGVHD at 12 months was estimated regardless of the length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
| Percentage of participants | Orca-T | Standard of Care |
|---|---|---|
| Event Rate at 12 Months for Time to Moderate or Severe cGVHD Per EAC | 12.6 (5.3 to 23.1) | 44.0 (31.3 to 56.1) |
Event-free rate estimated at 12 months for OS, which is defined as time from randomization to death from any cause. Each participant in the study is followed for up to 730 days after transplant. The analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe chronic GVHD) and was not based on the duration of follow-up. Therefore, the analysis was conducted using all available data at the time that the 56th cGFS event occurred, and the event-free rate of OS at 12 months was estimated regardless of the length of follow-up for individual participants. At the time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
| Percentage of participants | Orca-T | Standard of Care |
|---|---|---|
| Event-free at 12 Months for Overall Survival (OS) | 93.9 (85.8 to 97.4) | 83.1 (72.9 to 89.8) |
Event-free rate estimated at 12 months for GRFS, which is defined as time from alloHCT to death from any cause, relapse, the first onset of grade 3 or 4 acute GVHD (graded per Mount Sinai aGVHD International Consortium \[MAGIC\] criteria), or the first onset of moderate or severe cGVHD (graded per NIH consensus criteria), whichever is earliest, within 2 years from day 0 as assessed by independent EAC. Each participant in the study is followed for up to 730 days after transplant. The analysis was triggered by the 56th cGFS event (ie, when 56 participants had died or had moderate or severe cGVHD) and not based on duration of follow-up. Therefore, analysis was conducted using all available data at the time that the 56th cGFS event occurred, and event-free rate of GRFS at 12 months was estimated regardless of length of follow-up for individual participants. At time of analysis, not all participants have reached 730 days of follow-up because they enrolled at different times.
| Percentage of participants | Orca-T | Standard of Care |
|---|---|---|
| Event-free Rate at 12 Months for Graft-versus-host Disease-free and Relapse-free Survival (GRFS) Per EAC | 63.1 (50.0 to 73.6) | 30.9 (20.0 to 42.4) |
Collected over A treatment-emergent AE (TEAE) was any AE that started on or after alloHCT infusion (Orca-T or SoC) through study completion (day +730).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Orca-T | 3/88 (3.4%) | 34/88 (38.6%) | 88/88 (100%) |
| Standard of Care | 9/94 (9.6%) | 53/94 (56.4%) | 94/94 (100%) |
| Event | Orca-T | Standard of Care |
|---|---|---|
| Acute graft-versus-host diseaseImmune system disorders | 6/88 | 12/94 |
| SepsisInfections and infestations | 7/88 | 6/94 |
| PneumoniaInfections and infestations | 1/88 | 6/94 |
| Chronic graft-versus-host diseaseImmune system disorders | 1/88 | 6/94 |
| PyrexiaGeneral disorders | 5/88 | 1/94 |
| COVID-19Infections and infestations | 0/88 | 5/94 |
| Bacterial sepsisInfections and infestations | 4/88 | 4/94 |
| Acute kidney injuryRenal and urinary disorders | 2/88 | 4/94 |
| Venoocclusive liver diseaseHepatobiliary disorders | 3/88 | 1/94 |
| Septic shockInfections and infestations | 1/88 | 3/94 |
| Event | Orca-T | Standard of Care |
|---|---|---|
| NauseaGastrointestinal disorders | 79/88 | 79/94 |
| DiarrhoeaGastrointestinal disorders | 71/88 | 83/94 |
| FatigueGeneral disorders | 50/88 | 48/94 |
| HypomagnesaemiaMetabolism and nutrition disorders | 49/88 | 53/94 |
| StomatitisGastrointestinal disorders | 32/88 | 50/94 |
| ThrombocytopeniaBlood and lymphatic system disorders | 46/88 | 44/94 |
| Decreased appetiteMetabolism and nutrition disorders | 40/88 | 47/94 |
| VomitingGastrointestinal disorders | 43/88 | 43/94 |
| Febrile neutropeniaBlood and lymphatic system disorders | 32/88 | 42/94 |
| Chronic graft-versus-host diseaseImmune system disorders | 13/88 | 40/94 |
Intent-to-Treat (ITT): All enrolled participants who were randomized to either Orca-T or standard of care (SoC), regardless of whether they received Orca-T/SoC or not; participants were analyzed according to their randomized treatment assignment
| Age, Categorical(Participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 93 | 94 | 187 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| Female | 40 | 44 | 84 |
| Male | 53 | 50 | 103 |
| Ethnicity (NIH/OMB)(Participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| Hispanic or Latino | 26 | 24 | 50 |
| Not Hispanic or Latino | 62 | 59 | 121 |
| Unknown or Not Reported | 5 | 11 | 16 |
| Race (NIH/OMB)(Participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| American Indian or Alaska Native | 1 | 2 | 3 |
| Asian | 10 | 8 | 18 |
| Native Hawaiian or Other Pacific Islander | 1 | 1 | 2 |
| Black or African American | 1 | 2 | 3 |
| White | 76 | 63 | 139 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 4 | 18 | 22 |
| Region of Enrollment(participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| United States | 93 | 94 | 187 |
| Primary Disease(Participants) | Orca-T | Standard of Care | Total |
|---|---|---|---|
| Acute Lymphoid Leukemia (ALL) | 30 | 27 | 57 |
| Acute Myeloid Leukemia (AML) | 49 | 51 | 100 |
| High-risk Myelodysplastic Syndrome (MDS) | 12 | 11 | 23 |
| Mixed Phenotype Acute Leukemia (MPAL) | 2 | 5 | 7 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No
This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Orca Biosystems, Inc.