A Phase 1 Study of Orca-Q in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

NCT ID: NCT03802695

Last Updated: 2025-12-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

300 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-04-08

Study Completion Date

2027-12-31

Brief Summary

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This study will evaluate the safety, tolerability, and efficacy of engineered donor grafts ("OrcaGraft"/"Orca-Q") in participants undergoing allogeneic hematopoietic cell transplant (alloHCT) transplantation for hematologic malignancies.

Detailed Description

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Conditions

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Acute Myeloid Leukemia Myelodysplastic Syndromes Mixed Phenotype Acute Leukemia Acute Lymphoblastic Leukemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Arm A

Recipients with human leukocyte antigen (HLA)-identical related or unrelated or 1-allele mismatched (7/8 alleles) unrelated donor undergoing myeloablative conditioning (MAC); with single- or dual-agent graft-versus-host disease (GVHD) prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Arm B

Recipients with haploidentical-related donors undergoing MAC; with single- or dual-agent GVHD prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Arm C

Recipients with an HLA-identical related or unrelated donor undergoing MAC; no GVHD prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Arm D

Recipients with an HLA-identical related or unrelated donor undergoing non-myeloablative (NMA)/reduced intensity conditioning (RIC); with dual agent GVHD prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Arm E

Recipients with 1-allele mismatched (7/8 alleles) unrelated donor undergoing NMA/RIC; with dual-agent GVHD prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Arm F

Recipients with haploidentical-related donors undergoing NMA/RIC; with dual-agent GVHD prophylaxis given

Group Type EXPERIMENTAL

OrcaGraft (Orca-Q)

Intervention Type BIOLOGICAL

engineered donor allograft

Interventions

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OrcaGraft (Orca-Q)

engineered donor allograft

Intervention Type BIOLOGICAL

Eligibility Criteria

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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)

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
Minimum Eligible Age

12 Years

Maximum Eligible Age

78 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Orca Biosystems, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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James S McClellan, MD, PhD

Role: STUDY_DIRECTOR

Orca Biosystems, Inc.

Locations

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UC Davis

Sacramento, California, United States

Site Status RECRUITING

City of Hope

Duarte, California, United States

Site Status RECRUITING

Stanford Health Care

Stanford, California, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status ACTIVE_NOT_RECRUITING

The University of Kansas Hospital

Kansas City, Kansas, United States

Site Status WITHDRAWN

Ohio State University

Columbus, Ohio, United States

Site Status ACTIVE_NOT_RECRUITING

University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Froedtert Memorial Lutheran Hospital

Milwaukee, Wisconsin, United States

Site Status WITHDRAWN

Countries

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United States

Central Contacts

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Tamara Zharkevich, MD, PhD

Role: CONTACT

650-246-9601

James S McClellan, MD PhD

Role: CONTACT

650-246-9601

Facility Contacts

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Amandeep Salhotra, MD

Role: primary

Mehrdad Abedi, MD

Role: primary

Robert Lowsky, MD

Role: primary

Samer Srour, MD

Role: primary

Other Identifiers

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OGFT001-001

Identifier Type: -

Identifier Source: org_study_id

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