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

NCT05322850 | Suspended Phase 1 DMC FDA Drug

• 2 other identifiers: UF-PED-003OCR41968
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 4

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.

Conditions

Acute Myeloid Leukemia; Acute Lymphoid Leukemia; Mixed Phenotype Acute Leukemia; Acute Undifferentiated Leukemia; Chronic Myeloid Leukemia in Myeloid Blast Crisis; Chronic Myeloid Leukemia in Lymphoid Blast Crisis (Diagnosis); Chronic Myeloid Leukemia - Accelerated Phase

Keywords

hematologic malignancies; Orca-Q; hematopoietic cell transplant; engineered donor graft; pediatrics

Outcome Measures

Primary Outcomes (2)

• Primary graft failure

  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

  28 days

• Secondary graft failure

  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.

  100 days

Secondary Outcomes (10)

• Non-relapse mortality (12 months post-transplant)

  12 months

• Non-relapse mortality (24 months post-transplant)

  24 months

• Overall survival

  24 months

• Relapse rate (12 months post-transplant)

  12 months

• Relapse rate (24 months post-transplant)

  24 months

Study Arms (1)

Orca-Q

EXPERIMENTAL

Biological: Orca-Q

Interventions

Orca-Q BIOLOGICAL

All transplant recipients enrolled on this study in both the phase I and phase II portions will receive Orca-Q (Orca-Q Prime and Orca-Q Supplement) intravenously following myeloablative conditioning.

Orca-Q

Eligibility Criteria

• Age: Up to 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age \< 22 years of age at the time of diagnosis of malignancy
• Eligible for treatment at a pediatric HCT center
• Patients with the following histopathologically-confirmed diseases are eligible:
• Acute myeloid, lymphoid, mixed phenotype or undifferentiated leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi) as defined by:
• Marrow blasts \< 5% by morphologic examination
• Absolute neutrophil count \> 1.0 × 109/L
• Platelet count \> 100 × 109/L
• Absence of leukemic blasts in the peripheral blood by morphological examination, and
• No evidence of extramedullary disease
• CRi: All CR criteria except for residual neutropenia (\< 1.0 × 109/L) or thrombocytopenia (\< 100 × 109/L)
• Myeloid or lymphoid blast crisis or accelerated phase developing in the setting of chronic myeloid leukemia is an allowed diagnosis provided that patients are in CR or CRi with regard to the blast crisis
• Planned to undergo myeloablative allogeneic hematopoetic cell transplant (MA-alloHCT) with a myeloablative conditioning regimen
• Available donor willing to donate PBSCs:
• Related donor who is a 7 or 8/8 match for HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods
• Matched unrelated donor who is a 7 or 8/8 match at HLA-A, -B, -C, and -DRB1, all typed using DNA-based high-resolution methods

You may not qualify if:

• Prior myeloablative allogeneic HCT
• Currently receiving corticosteroids or other immunosuppressive therapy (topical corticosteroids or physiologic replacement hydrocortisone is allowed)
• Planned donor lymphocyte infusion (DLI)
• Planned pharmaceutical in vivo or ex vivo T cell depletion, e.g., post-transplant cyclophosphamide (Cy) or alemtuzumab. Anti-thymocyte globulin is allowed only as specified in the protocol.
• Positive anti-donor HLA antibodies against a mismatched allele in the selected donor determined by either:
• a positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing), or
• the presence of anti-donor HLA antibody to any of the following HLA loci: HLA-A, -B, -C, -DRB1, -DQB1, -DQA1, -DPB1, or -DPA1, with mean fluorescence intensity (MFI) \> 1000 by solid phase immunoassay
• Lansky play scale \< 70% or Karnofsky \<70%
• Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) \> 4
• Documented or suspected bridging fibrosis or liver cirrhosis
• Uncontrolled bacterial, viral or fungal infections (currently taking antimicrobial therapy and with progression or no clinical improvement) at time of enrollment
• Seropositive for HIV-1 or -2, HTLV-1 or -2
• Known allergy or hypersensitivity to, or intolerance of, tacrolimus
• Documented allergy or hypersensitivity to iron dextran or bovine, murine, algal or Streptomyces avidinii proteins
• Any uncontrolled autoimmune disease requiring active immunosuppressive treatment

Sponsors & Collaborators

• Orca Biosystems, Inc.: collaborator
• Florida Department of Health: collaborator
• University of Florida: lead

Study Sites (4)

University of Florida

Gainesville, Florida, 32608, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Biphenotypic, Acute; Disease; Leukemia, Myeloid, Accelerated Phase; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Neoplasms by Site

Study Officials

• Jordan Milner, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2022
• First Posted: April 12, 2022
• Study Start: August 16, 2022
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2027
• Last Updated: April 13, 2026

Record last verified: 2026-04

Locations

US (4)