A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies

NCT ID: NCT05589896

Last Updated: 2025-06-25

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/PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-08-16

Study Completion Date

2026-12-31

Brief Summary

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The goal of this clinical trial is to determine the safety and feasibility of allogeneic transplantation with bone marrow from a deceased donor in patients with acute and chronic leukemias, myelodysplastic syndrome, and certain lymphomas. Patients will either receive myeloablative conditioning or reduced intensity conditioning regimen prior to the transplant. Patients will be followed for 56 days for safety endpoints and remain in follow-up for one year.

Detailed Description

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Conditions

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Acute Leukemia Acute Lymphoblastic Leukemia Acute Myeloid Leukemia Acute Biphenotypic Leukemia Acute Undifferentiated Leukemia CLL (Chronic Lymphocytic Leukemia) Chronic Myeloid Leukemia (CML) MDS (Myelodysplastic Syndrome) Non-Hodgkin Lymphomas Hodgkins Lymphoma Cutaneous T Cell Lymphomas (CTCL)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

All patients will receive Ossium HPC, Marrow product. Study arms will be based on order of enrollment and planned conditioning regimen.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

Bone Marrow Transplant with Ossium HPC, Marrow

Pre-transplant myeloablative conditioning treatment with:

Regimen A(MAC): Busulfan and Fludarabine

\[OR\]

Regimen B(MAC): Fludarabine and Total Body Irradiation

\[OR\]

Regimen C(RIC): Fludarabine, Cyclophosphamide, and Total Body Irradiation

\[OR\]

Regimen D (RIC): Fludarabine, Melphalan, and Total Body Irradiation

Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim

Group Type EXPERIMENTAL

Ossium HPC Marrow, Bone Marrow Transplant

Intervention Type OTHER

Hematopoetic Cell Transplantation

Pre-transplant conditioning - Myeloablative (MAC)

Intervention Type OTHER

Regimen A or Regimen B

Pre-transplant conditioning - Reduced Intensity (RIC)

Intervention Type OTHER

Regimen C or Regimen D

Post-transplant treatment

Intervention Type OTHER

Post-transplant treatment

Cohort 2

\*Open to enrollment after DSMB review of Cohort 1 safety events through Day 56\*

Bone Marrow Transplant with Ossium HPC, Marrow

Pre-transplant conditioning treatment with:

Regimen A(MAC): Busulfan and Fludarabine

\[OR\]

Regimen B(MAC): Fludarabine and Total Body Irradiation

\[OR\]

Regimen C(RIC): Fludarabine, Cyclophosphamide, and Total Body Irradiation

\[OR\]

Regimen D(RIC): Fludarabine, Melphalan, and Total Body Irradiation

Post-Transplant treatment with Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, and Filgrastim

Group Type EXPERIMENTAL

Ossium HPC Marrow, Bone Marrow Transplant

Intervention Type OTHER

Hematopoetic Cell Transplantation

Pre-transplant conditioning - Myeloablative (MAC)

Intervention Type OTHER

Regimen A or Regimen B

Pre-transplant conditioning - Reduced Intensity (RIC)

Intervention Type OTHER

Regimen C or Regimen D

Post-transplant treatment

Intervention Type OTHER

Post-transplant treatment

Interventions

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Ossium HPC Marrow, Bone Marrow Transplant

Hematopoetic Cell Transplantation

Intervention Type OTHER

Pre-transplant conditioning - Myeloablative (MAC)

Regimen A or Regimen B

Intervention Type OTHER

Pre-transplant conditioning - Reduced Intensity (RIC)

Regimen C or Regimen D

Intervention Type OTHER

Post-transplant treatment

Post-transplant treatment

Intervention Type OTHER

Other Intervention Names

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Busuflex Fludara TBI Fludara Cytoxan Mesnex TBI Melphalan Cytoxan Mesnex CellCept Filgrastim

Eligibility Criteria

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Inclusion Criteria

* Patient has the ability to provide informed consent according to the applicable regulatory and local institutional requirements
* Male or female, aged ≥18 and ≤65 years for patients receiving MAC (Regimen A or Regimen B); aged ≥18 and ≤75 years for patients receiving RIC (Regimen C or D)
* Patient must require allogeneic HCT per the discretion of the treating physician
* Patient must be high-resolution, HLA partially or fully matched (4-8/8 allele matched at HLA-A, -B, -C, DRB1) to an available Ossium HPC, Marrow product
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Diagnosed with malignant hematologic disease including:

1. Acute leukemia \[acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), acute biophenotypic leukemia (ABL), or acute undifferentiated leukemia (AUL)\], MDS without fibrosis, or chronic leukemia (CLL, CML) in the first remission or beyond with ≤5% marrow blasts documented by bone marrow assessment and no circulating blasts or extra-medullary disease within 42 days prior to anticipated start of conditioning
2. Chemosensitive non-Hodgkin's lymphomas, Hodgkin's lymphoma, or cutaneous T cell lymphomas in the first remission or beyond documented by PET/CT imaging and bone marrow assessment within 42 days prior to anticipated start of conditioning
* Karnofsky performance status score ≥70% (MAC) or ≥60% (RIC)
* HCT comorbidity index (HCT-CI) ≤5
* Adequate organ function defined as:

1. Cardiac: LVEF at rest ≥40% (RIC) or LVEF at rest ≥45% (MAC)
2. Pulmonary: DLCO, FEV1, FVC ≥50% predicted by pulmonary function tests (PFTs). DLCO value may be corrected for hemoglobin.
3. Hepatic: total bilirubin ≤2.0 mg/dL, and ALT, AST, and ALP \<3 x upper limit normal (ULN), unless ALT, AST, and/or ALP are disease related
4. Renal: SCr within 1.5x normal range for age. If SCr is outside normal range for age, CrCl\> 60 mL/min/1.73m2 must be obtained (measured by 24-hour urine specimen or nuclear glomerular filtration rate (GFR), or calculated GFR)

Exclusion Criteria

* Availability of suitable graft from living donor (defined as 7/8 or 8/8 HLA-matched related or unrelated donors, haploidentical donors, or cord blood donors)
* Prior autologous or allogeneic HCT
* Pregnancy or lactation
* Ongoing treatment with an investigational drug used for disease-related treatment within 5 half-lives of the drug
* Current uncontrolled bacterial, viral or fungal infection defined as currently taking medication with evidence of progression of clinical symptoms or radiologic findings
* Any condition(s) or diagnosis, both physical or psychological, or physical exam finding that in the investigator's opinion precludes participation
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Center for International Blood and Marrow Transplant Research

NETWORK

Sponsor Role collaborator

Ossium Health, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jeffery Auletta, MD

Role: STUDY_CHAIR

Center for International Blood and Marrow Transplant Research

Locations

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City of Hope

Duarte, California, United States

Site Status RECRUITING

Moffitt Cancer Center

Tampa, Florida, United States

Site Status RECRUITING

Henry Ford Cancer Institute

Detroit, Michigan, United States

Site Status RECRUITING

Columbia University - Herbert Irving Comprehensive Cancer Center

New York, New York, United States

Site Status RECRUITING

Oregon Health and Science University

Portland, Oregon, United States

Site Status RECRUITING

TriStar Bone Marrow Transplant

Nashville, Tennessee, United States

Site Status RECRUITING

St. David's South Austin Medical Center

Austin, Texas, United States

Site Status RECRUITING

Methodist Hospital, Texas Transplant

San Antonio, Texas, United States

Site Status RECRUITING

University of Utah - Huntsman Cancer Institute

Salt Lake City, Utah, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Matthew Flores

Role: CONTACT

(763)406-3060

Preethi Prasad, MSc.

Role: CONTACT

Facility Contacts

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Miranda Garcia

Role: primary

Eric Hornfeck

Role: primary

Abhinav Krishnaswami

Role: primary

Elizabeth Shelton, MPH

Role: primary

Elisa Minerich

Role: primary

Sydney Fitzgerald

Role: primary

Stephanie Goldin

Role: primary

Tatiana Aguirre Robles Gil, MD

Role: primary

Mia Kowalczyk

Role: primary

Other Identifiers

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PRESERVE I

Identifier Type: -

Identifier Source: org_study_id

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