Phase I/II Trial: Engineered Donor Graft (Orca Q) for Pediatric Hematopoietic Cell Transplant (HCT)

Phase 1

Recruiting

• Conditions: Chronic Myeloid Leukemia in Lymphoid Blast Crisis (Diagnosis); Acute Undifferentiated Leukemia; Acute Myeloid Leukemia; Acute Lymphoid Leukemia; Chronic Myeloid Leukemia in Myeloid Blast Crisis; Chronic Myeloid Leukemia - Accelerated Phase; Mixed Phenotype Acute Leukemia
• Interventions: Biological: Orca-Q

• Registration Number: NCT05322850
• Lead Sponsor: University of Florida

Brief Summary

This is a first in children prospective study of allogeneic hematopoietic cell transplant using a centrally manufactured engineered donor graft (Orca-Q). The study will assess safety and efficacy of Orca-Q in pediatric patients with hematologic malignancies.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-31
• Study First Submitted QC: 2022-04-11
• Study First Posted: 2022-04-12
• Study Start: 2022-08-16
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-12
• Last Update Posted: 2025-06-13
• Primary Completion: 2026-09
• Study Completion: 2027-09

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Orca-Q	Orca-Q	-

Primary Outcome Measures

Name	Time	Method
Primary graft failure	28 days	Evaluate primary graft failure through day +28, defined as being alive without recovery of neutrophils (achieving an absolute neutrophil count \>500/µL for 3 consecutive days) by day +28
Secondary graft failure	100 days	Evaluate secondary graft failure through day +100, defined as neutrophil engraftment followed by subsequent decline in absolute neutrophil counts to \<500 µL, unresponsive to growth factor therapy and in the absence of alternative explanations such as recurrence of the underlying malignant disorder, infections, microangiopathy, medications causing bone marrow suppression or immune-mediated cytopenia.

Secondary Outcome Measures

Name	Time	Method
Non-relapse mortality (12 months post-transplant)	12 months	Evaluate non-relapse mortality, defined as death without evidence of disease recurrence, at 12 months post-transplant
Relapse rate (12 months post-transplant)	12 months	Determine the relapse rate, as defined as the incidence of disease relapse through 12 months post-transplant. Disease relapse is defined as any of the following: ≥ 5% blasts in the bone marrow or peripheral blood, reappearance of pre-transplant cytogenetic abnormality, new evidence or redevelopment of extramedullary disease. Institution of any therapy to treat relapsed disease, such as withdrawal of immunosuppression or donor lymphocyte infusion (DLI), will be considered evidence of relapse regardless of whether any of these are met.
Treatment-Emergent Adverse Events	24 months	Describe treatment-emergent adverse events through 24 months post-transplant.
Overall survival	24 months	Determine the overall survival at 24 months post-transplant, as defined as the time from the date of transplant to the date of death from any cause or, for surviving patients, to the date of last follow-up.
Relapse rate (24 months post-transplant)	24 months	Determine the relapse rate, as defined as the incidence of disease relapse through 24 months post-transplant. Disease relapse is defined as any of the following: ≥ 5% blasts in the bone marrow or peripheral blood, reappearance of pre-transplant cytogenetic abnormality, new evidence or redevelopment of extramedullary disease. Institution of any therapy to treat relapsed disease, such as withdrawal of immunosuppression or donor lymphocyte infusion (DLI), will be considered evidence of relapse regardless of whether any of these are met.
Non-relapse mortality (24 months post-transplant)	24 months	Evaluate non-relapse mortality, defined as death without evidence of disease recurrence, at 24 months post-transplant.
Rate of acute GVHD	6 months	Determine the rate of acute GVHD at 6 months post-transplant
Rate of chronic GVHD	24 months	Determine the rate of chronic GVHD through 24 months post-transplant.
Serious infection rate	12 months	Determine the rate of serious infections by 12 months post-transplant. A serious infection is defined as any new viral, bacterial, fungal or parasitic infection Common Terminology Criteria for Adverse Events v5.0 grade 2 or higher.
Graft-versus-host disease (GVHD)-free, relapse-free survival	12 months	Evaluate the GVHD-free, relapse-free survival at 12 months post-transplant. GVHD-free, relapse-free survival is defined as freedom from grade III or IV acute GVHD, moderate or severe chronic GVHD, or disease progression or relapse.

Trial Locations

Locations (4)

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of Miami; 🇺🇸 Miami, Florida, United States

Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States

Johns Hopkins All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States