NCT04013685

Brief Summary

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
255

participants targeted

Target at P75+ for phase_1

Timeline
11mo left

Started Nov 2019

Longer than P75 for phase_1

Geographic Reach
1 country

21 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress88%
Nov 2019Apr 2027

First Submitted

Initial submission to the registry

July 3, 2019

Completed
7 days until next milestone

First Posted

Study publicly available on registry

July 10, 2019

Completed
4 months until next milestone

Study Start

First participant enrolled

November 21, 2019

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 30, 2025

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2027

Expected
Last Updated

May 2, 2025

Status Verified

May 1, 2025

Enrollment Period

5.4 years

First QC Date

July 3, 2019

Last Update Submit

May 1, 2025

Conditions

Keywords

hematopoietic stem cell transplantationacute leukemiaMyelodysplastic syndromesmatched related donormatched unrelated donor

Outcome Measures

Primary Outcomes (2)

  • The incidence of primary graft failure

    The incidence of primary graft failure

    365 days

  • The incidence of grade 3 or 4 aGVHD

    The incidence of grade 3 or 4 aGVHD

    180 days

Secondary Outcomes (5)

  • 1-year overall survival (OS)

    365 days

  • 1 year graft-versus-host-disease-free and relapse-free survival (GRFS)

    365 days

  • incidence and severity of acute and chronic graft vs host disease (GvHD)

    365 days

  • incidence of serious infections

    365 days

  • incidence of engraftment

    28 days

Study Arms (1)

Subjects with Acute Leukemia or Myelodysplastic Syndrome, or BPDCN

EXPERIMENTAL

This is a non-randomized, single-arm study. All enrolled subjects will receive an allogeneic HCT with the Orca-T product.

Biological: Orca-T

Interventions

Orca-TBIOLOGICAL

an allogeneic stem cell and T-cell immunotherapy biologic

Subjects with Acute Leukemia or Myelodysplastic Syndrome, or BPDCN

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Recipients must meet all of the following criteria:
  • Patients must diagnosed with one of the following histopathologically confirmed diseases, for which a myeloablative hematopoietic stem cell transplant (HCT) is planned:
  • A) Acute myeloid, lymphoid, or mixed phenotype/undifferentiated leukemia who are not in CR or CRi (active disease) and/or MDS with \>10% to \<20% bone marrow blast burden (ages 18 to 75 years)
  • B) Acute leukemia in CR/CRi or MDS that is DRI intermediate to high risk (ages 66 to 75 years)
  • C) BPDCN (ages 18 to 65 years)
  • D) Participants aged 18 to 65 who would be eligible for the Phase 3 component of Precision-T except for mild impairments of renal and/or hepatic function as defined by an eGFR of 50 to \<60 mL/min and/or a total bilirubin of \>ULN to ≤2 x ULN and diagnosed with either of the following:
  • i. Acute myeloid, lymphoid, or mixed phenotype/undifferentiated leukemia that is in CR/CRi and DRI intermediate to high risk
  • a) MDS that is DRI intermediate to high risk
  • E) Acute or chronic leukemia in remission that is DRI low risk (ages 18 to 65 years), including the following:
  • i. CML in chronic phase but with a history of accelerated phase or blast crisis or who are resistant to or intolerant of more than 1 first- and second-generation tyrosine kinase inhibitors
  • ii. Acute myeloid leukemia (AML) with inv(16) without accompanying complex cytogenetics
  • Patients must be matched to a 8/8 HLA-matched related or unrelated donor
  • Estimated glomerular filtration rate (eGFR) \> 50 mL/minute
  • Cardiac ejection fraction at rest ≥ 45% or shortening fraction of ≥ 27% by echocardiogram or radionuclide scan (MUGA)
  • Diffusing capacity of the lung for carbon monoxide (DLCO) (adjusted for hemoglobin) ≥ 50%
  • +1 more criteria

You may not qualify if:

  • History of prior allogeneic HCT
  • Currently receiving corticosteroids or other immunosuppressive therapy. Topical corticosteroids or oral systemic corticosteroid doses less than or equal to 10 mg/day are allowed.
  • Pre-planned donor lymphocyte infusion (DLI)
  • Planned pharmaceutical in vivo or ex vivo T cell depletion
  • Positive for anti-donor HLA antibodies against an allele in the selected donor
  • Karnofsky performance score \< 70%
  • Hematopoietic cell transplantation-specific Comorbidity Index (HCT-CI) \> 4
  • 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 antibody, HTLV-1 or -2 antibody, Hepatitis B sAg, or Hepatitis C antibody
  • Any uncontrolled autoimmune disease requiring active immunosuppressive treatment
  • Concurrent malignancies or active disease within 1 year, except non-melanoma skin cancers that have been curatively resected
  • Women who are pregnant or breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (21)

City of Hope

Duarte, California, 91010, United States

Location

Ronald Reagan UCLA Medical Center

Los Angeles, California, 90095, United States

Location

UC Davis

Sacramento, California, 95817, United States

Location

Stanford Health Care

Stanford, California, 94305, United States

Location

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States

Location

University of Miami Hospital and Clinics - Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Winship Cancer Institute - Emory University

Atlanta, Georgia, 30322, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

The University of Kansas Hospital

Kansas City, Kansas, 66160, United States

Location

Massachusetts

Boston, Massachusetts, 02114, United States

Location

University of Michigan Health System - Michigan Medicine

Ann Arbor, Michigan, 48109, United States

Location

Weill Cornell Medicine - New York-Presbyterian Hospital

New York, New York, 10021, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

OU Health Stephenson Cancer Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oregon Health & Sciences University - Knight Cancer Institute

Portland, Oregon, 97239, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Vanderbilt University

Nashville, Tennessee, 37232, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77054, United States

Location

University of Utah - Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, AcutePrecursor Cell Lymphoblastic Leukemia-LymphomaMyelodysplastic SyndromesBlastic Plasmacytoid Dendritic Cell NeoplasmLeukemia, Myelogenous, Chronic, BCR-ABL Positive

Condition Hierarchy (Ancestors)

Leukemia, MyeloidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLeukemia, LymphoidLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesBone Marrow DiseasesHistiocytic Disorders, MalignantLymphomaHematologic NeoplasmsNeoplasms by SiteSkin NeoplasmsSkin DiseasesSkin and Connective Tissue DiseasesMyeloproliferative DisordersChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Design

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

Study Record Dates

First Submitted

July 3, 2019

First Posted

July 10, 2019

Study Start

November 21, 2019

Primary Completion

April 30, 2025

Study Completion (Estimated)

April 1, 2027

Last Updated

May 2, 2025

Record last verified: 2025-05

Locations