Engineered donor cells aim to make stem cell transplants safer for blood cancer patients
NCT ID NCT03802695
First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times
Summary
This study tests a new way to prepare donor stem cells (called Orca-Q) for people with blood cancers like leukemia or MDS who need a transplant. The goal is to see if this engineered graft is safe and helps the body accept the new cells with fewer complications. About 300 participants will receive the treatment and be monitored for side effects and how well the new cells take hold.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 300 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2019
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 78 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Key Inclusion Criteria: 1. Age at the time of enrollment: 1. For MAC with fully matched donor (Arm A with 8/8 donor and Arm C) and NMA/RIC: Age ≥ 12 and ≤ 78 years 2. For MAC with mismatched donors (Arm A with 7/8 donor and Arm B): Age ≥ 12 and ≤ 65 years 2. Diagnosed acute myeloid, lymphoblastic or mixed phenotype leukemia, or high or very high risk myelodysplastic syndrome (MDS) either in complete remission (CR) or with ≤ 10 percent of blast cells in bone marrow (BM) 3. Indicated for allogeneic hematopoietic stem cell transplant (alloHCT) 4. Matched to a 8/8 or 7/8 related or unrelated donor, or to a related haploidentical donor 5. Estimated glomerular filtration rate (eGFR) \> 50 mL/minute (MAC with tacrolimus) or \> 30 mL/minute (NMA/RIC or MAC without tacrolimus) 6. Cardiac parameters: Cardiac ejection fraction ≥ 45 percent (MAC) or ≥ 40 percent (NMA/RIC) 7. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50 percent for MAC or ≥ 40 percent for NMA/RIC 8. Liver function: Total bilirubin \< 1.5 times upper limit of normal (ULN) (MAC) or \< 3 times ULN (NMA/RIC); alanine transaminase (ALT)/aspartate transaminase (AST) \< 3 times ULN (MAC) or \< 5 times ULN (NMA/RIC) 9. Participants enrolling on NMA/RIC-alloHCT arms must be deemed unfit for a myeloablative alloHCT per assessment of the principal investigator (PI) Key Exclusion Criteria: 1. Prior alloHCT 2. Currently receiving corticosteroids or other immunosuppressive therapy except for approved disease-specific therapy for the patient's underlying hematologic malignancy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed 3. Planned donor lymphocyte infusion (DLI) 4. Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy) or alemtuzumab 5. Positive anti-donor HLA antibodies against a mismatched allele in the selected donor 6. Low performance score: For MAC: Karnofsky Performance Score (KPS) \< 70 percent, For NMA/RIC: \<60 percent 7. High HCT-specific Comorbidity Index (HCT-CI): For MAC \> 4, For NMA/RIC \>6 8. Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment 9. Seropositive for human immunodeficiency virus (HIV)-1 or -2, human T-lymphotropic virus (HTLV)-1 or -2 or Hepatitis B surface antigen (HbsAg) or anti-Hepatitis C virus (HCV) antibody (Ab) 10. Any uncontrolled autoimmune disease requiring active immunosuppressive treatment 11. Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected. Patients with concurrent indolent hematologic malignancies that do not require active treatment and are under active surveillance only (such as CLL, low-grade lymphomas, smoldering MM, MZL) may be included with the approval of Medical Monitor 12. History of idiopathic or secondary myelofibrosis 13. Women who are pregnant or breastfeeding
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
8 sites. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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City of Hope
RECRUITINGDuarte, California, 91010, United States
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Emory University
ACTIVE_NOT_RECRUITINGAtlanta, Georgia, 30322, United States
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Fred Hutchinson Cancer Center
RECRUITINGSeattle, Washington, 98109, United States
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Froedtert Memorial Lutheran Hospital
WITHDRAWNMilwaukee, Wisconsin, 53226, United States
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John Theurer Cancer Center at Hackensack University Medical Center
RECRUITINGHackensack, New Jersey, 07601, United States
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Memorial Sloan Kettering Cancer Center
RECRUITINGNew York, New York, 10065, United States
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Moffitt Cancer Center
RECRUITINGTampa, Florida, 33612, United States
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Ohio State University
ACTIVE_NOT_RECRUITINGColumbus, Ohio, 43210, United States
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Stanford Health Care
RECRUITINGStanford, California, 94305, United States
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The University of Kansas Hospital
WITHDRAWNKansas City, Kansas, 66160, United States
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UC Davis
RECRUITINGSacramento, California, 95817, United States
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University of Texas MD Anderson Cancer Center
RECRUITINGHouston, Texas, 77054, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding venetoclax make donor stem cell transplants safer for High-Risk blood cancers?
- Can an HDAC inhibitor wipe out residual leukemia cells?
- Can an experimental pill block a cancer-driving enzyme in hard-to-treat leukemia?
- Engineered immune cells aim to wipe out stubborn leukemia
- Two-Drug combo targets leukemia that outsmarted its first treatment
- Tweaking donor cells may shield older transplant patients from a dangerous complication