Orca-T for Blood Cancers

(Orca-T Trial)

Recruiting at 20 trial locations

Overseen By Anna Pavlova, MD PhD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Orca Biosystems, Inc.

Must not be taking: Corticosteroids, Immunosuppressive therapy

Disqualifiers: Prior allogeneic HCT, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

174 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires that participants stop taking corticosteroids or other immunosuppressive therapy, except for topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day.

Eligibility Criteria

This trial is for people with certain blood cancers or pre-leukemia who are in remission, have a matched donor for stem cell transplant, and whose major organs function well. They must not be pregnant, breastfeeding, or unwilling to use birth control. People with recent other cancers, prior transplants, severe infections or certain viruses like HIV can't join.

Inclusion Criteria

Total bilirubin ≤ upper limit of normal (ULN)

Negative serum or urine beta-HCG test in females of childbearing potential

Estimated glomerular filtration rate (eGFR) ≥ 60 mL/minute

Exclusion Criteria

Psychosocial circumstances that preclude the patient from going through transplant or participating responsibly in follow-up care

Known allergy or hypersensitivity to, or intolerance of, tacrolimus

I am on low-dose corticosteroids or other immunosuppressive therapy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning and Transplantation

Participants undergo a myeloablative conditioning regimen followed by transplantation with either Orca-T or standard-of-care allograft

3-4 weeks

Initial Treatment and Prophylaxis

Participants receive GVHD prophylaxis with tacrolimus (and methotrexate for standard-of-care arm) starting around Day -3 to Day +3

4 weeks

Follow-up

Participants are monitored for safety and effectiveness, including survival and GVHD outcomes

730 days

Treatment Details

Interventions

• Orca-T
• Standard-of-Care

Trial Overview The study compares Orca-T (an engineered graft of T cells) versus standard care in patients undergoing myeloablative allogeneic hematopoietic cell transplantation for blood cancers. It aims to assess the safety and effectiveness of this new treatment approach.

Participant Groups

Group I: Orca-T Experimental Treatment 1 Intervention

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).

Group II: Standard of Care alloHCT Control Active Control 1 Intervention

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.

Orca-T is already approved in United States for the following indications:

Approved in United States as Orca-T for:

• Acute Lymphoblastic Leukemia (ALL)
• Acute Myeloid Leukemia (AML)
• Myelodysplastic Syndrome (MDS)
• Myelofibrosis

Who Is Running the Clinical Trial?

Orca Biosystems, Inc.

Lead Sponsor

Trials

8

Recruited

750+