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Engineered donor cells aim to make stem cell transplants safer for blood cancer patients

NCT ID NCT03802695

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 300 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2019

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

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

12 to 78 years

Sex

Anyone

Healthy volunteers

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

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.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    8 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. 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

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • City of Hope

    RECRUITING

    Duarte, California, 91010, United States

  • Emory University

    ACTIVE_NOT_RECRUITING

    Atlanta, Georgia, 30322, United States

  • Fred Hutchinson Cancer Center

    RECRUITING

    Seattle, Washington, 98109, United States

  • Froedtert Memorial Lutheran Hospital

    WITHDRAWN

    Milwaukee, Wisconsin, 53226, United States

  • John Theurer Cancer Center at Hackensack University Medical Center

    RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Memorial Sloan Kettering Cancer Center

    RECRUITING

    New York, New York, 10065, United States

  • Moffitt Cancer Center

    RECRUITING

    Tampa, Florida, 33612, United States

  • Ohio State University

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43210, United States

  • Stanford Health Care

    RECRUITING

    Stanford, California, 94305, United States

  • The University of Kansas Hospital

    WITHDRAWN

    Kansas City, Kansas, 66160, United States

  • UC Davis

    RECRUITING

    Sacramento, California, 95817, United States

  • University of Texas MD Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77054, United States

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