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A Study of Engineered Donor Grafts (TregGraft/Orca-T) in Recipients Undergoing Allogeneic Transplantation for Hematologic Malignancies

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ClinicalTrials.gov Identifier: NCT04013685
Recruitment Status : Recruiting
First Posted : July 10, 2019
Last Update Posted : November 1, 2021
Information provided by (Responsible Party):
Orca Biosystems, Inc.

Brief Summary:
This study will evaluate the safety, tolerability, and efficacy of an engineered donor graft ("TregGraft"/"Orca-T", a T-cell-Depleted Graft With Additional Infusion of Conventional T Cells and Regulatory T Cells) in participants undergoing myeloablative allogeneic hematopoietic cell transplant transplantation for hematologic malignancies.

Condition or disease Intervention/treatment Phase
Acute Myeloid Leukemia Acute Lymphoid Leukemia Myelodysplastic Syndromes Acute Leukemia Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Biological: TregGraft (Orca-T) Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 84 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter Phase Ib Trial for Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Cell Transplantation With TregGraft (Orca-T), a T-cell-Depleted Graft With Additional Infusion of Conventional T Cells and Regulatory T Cells
Actual Study Start Date : November 21, 2019
Estimated Primary Completion Date : September 30, 2022
Estimated Study Completion Date : November 30, 2022

Arm Intervention/treatment
Experimental: Subjects with Acute Leukemia or Myelodysplasic Syndrome, Myelofibrosis, or BPDCN

This is a non-randomized, single-arm study. Patients will be grouped based on their underlying disease:

Group 1 will enroll subjects planning to undergo myeloablative allogeneic hematopoietic cell transplantation (MA-alloHCT) for the treatment of either acute myeloid, lymphoid or mixed phenotype leukemia in complete remission (CR) or CR with incomplete hematologic recovery (CRi), and with no known minimal residual disease positivity.

Group 2 will enroll subjects Subjects planning to undergo MA-alloHCT for acute myeloid, lymphoid or mixed phenotype leukemia that is either:

  • not in morphologic CR with bone marrow infiltration by leukemic blasts of <= 10%, or
  • in morphologic CR with evidence of minimal residual positivity by either multiparameter flow cytometric analysis or by a nucleic acid-based technique.

Group 3 will enroll subjects planning to MA-alloHCT for high or very high risk myelodysplasic syndrome (MDS) myelodysplastic syndromes, myelofibrosis or BPDCN

Biological: TregGraft (Orca-T)
engineered donor allograft

Primary Outcome Measures :
  1. TregGraft (Orca-T), with single agent GVHD prophylaxis [ Time Frame: 365 days ]

Secondary Outcome Measures :
  1. 1-year overall survival (OS) [ Time Frame: 365 days ]
    1-year overall survival (OS)

  2. 1 year graft-versus-host-disease-free and relapse-free survival (GRFS) [ Time Frame: 365 days ]
    1 year graft-versus-host-disease-free and relapse-free survival (GRFS)

  3. incidence and severity of acute and chronic graft vs host disease (GvHD) [ Time Frame: 365 days ]
    incidence and severity of acute and chronic graft vs host disease (GvHD)

  4. incidence of serious infections [ Time Frame: 365 days ]
    incidence of serious infections

  5. incidence and timing of engraftment [ Time Frame: 28 days ]
    incidence and timing of engraftment of platelets and neutrophils

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

Recipients must meet all of the following criteria:

  1. Patients must diagnosed with one of the following histopathologically confirmed diseases, for which a myeloablative hematopoietic stem cell transplant (HCT) is planned:

    • acute myeloid, lymphoid or mixed phenotype leukemia
    • high or very high risk myelodysplastic syndromes
    • Myelofibrosis
    • Blastic Plasmacytoid Dendritic Cell Neoplasm
  2. Patients with active acute leukemia (i.e. not in morphologic complete response) must have bone marrow infiltration by leukemic blasts of <= 10%,
  3. Patients must be matched to a related or unrelated donor
  4. Estimated glomerular filtration rate (eGFR) > 50 mL/minute
  5. Cardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA)
  6. Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50%
  7. Total bilirubin < 2 times upper limit of normal (ULN) (patients with Gilbert's syndrome may be included where hemolysis has been excluded) and ALT/AST < 3 times ULN

Key Exclusion Criteria:

Recipients meeting any of the following exclusion criteria will not be eligible:

  1. History of prior allogeneic HCT
  2. Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed.
  3. Pre-planned donor lymphocyte infusion (DLI)
  4. Planned pharmaceutical in vivo or ex vivo T cell depletion
  5. Positive for anti-donor HLA antibodies against an allele in the selected donor
  6. Karnofsky performance score < 70%
  7. Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) > 4
  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 HIV-1 or -2 antibody, HTLV-1 or -2 antibody, Hepatitis B sAg, or Hepatitis C antibody
  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
  12. Women who are pregnant or breastfeeding

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04013685

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Contact: James S McClellan, MD PhD 530-414-9743 info@orcabiosystems.com

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United States, California
City of Hope Recruiting
Duarte, California, United States, 91010
Contact: Amandeep Salhotra         
Ronald Reagan UCLA Medical Center Recruiting
Los Angeles, California, United States, 90095
Contact: Caspian Oliai         
UC Davis Recruiting
Sacramento, California, United States, 95817
Contact: Rasmus Hoeg         
Stanford Health Care Recruiting
Stanford, California, United States, 94305
Contact: Everett Meyer         
United States, Georgia
Winship Cancer Institute - Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Edmund Waller         
United States, Illinois
University of Chicago Recruiting
Chicago, Illinois, United States, 60637
Contact: Satyajit Kosuri         
United States, Kansas
The University of Kansas Hospital Recruiting
Kansas City, Kansas, United States, 66160
Contact: Joseph McGuirk         
United States, New York
Weill Cornell Recruiting
New York, New York, United States, 10065
Contact: Koen Van Besien         
United States, Oregon
Oregon Health & Sciences University - Knight Cancer Institute Recruiting
Portland, Oregon, United States, 97239
Contact: Arpita Gandhi         
United States, Tennessee
Sarah Cannon Research Institute Recruiting
Nashville, Tennessee, United States, 37203
Contact: Jeremy Pantin         
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Bhagirathbhai Dholaria         
United States, Texas
University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77054
Contact: Rohtesh Mehta         
Texas Transplant Institute Recruiting
San Antonio, Texas, United States, 78229
Contact: Behyar Zoghi         
United States, Utah
University of Utah - Huntsman Cancer Institute Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Sagar Patel         
Sponsors and Collaborators
Orca Biosystems, Inc.
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Responsible Party: Orca Biosystems, Inc.
ClinicalTrials.gov Identifier: NCT04013685    
Other Study ID Numbers: TRGFT-201
First Posted: July 10, 2019    Key Record Dates
Last Update Posted: November 1, 2021
Last Verified: October 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Orca Biosystems, Inc.:
hematopoietic stem cell transplantation
acute leukemia
Myelodysplastic syndromes
matched related donor
matched unrelated donor
Additional relevant MeSH terms:
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Hematologic Neoplasms
Leukemia, Lymphoid
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms by Site
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases