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RecruitingInterventionalPhase 1/Phase 2

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: NCT05589896Sponsor: Ossium Health, Inc.Last updated: 2026-02-24

Summary

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.

Arms & interventions

  • OtherOssium HPC Marrow, Bone Marrow Transplant

    Hematopoetic Cell Transplantation

  • OtherPre-transplant conditioning - Myeloablative (MAC)

    Regimen A or Regimen B

  • OtherPre-transplant conditioning - Reduced Intensity (RIC)

    Regimen C or Regimen D

  • OtherPost-transplant treatment

    Post-transplant treatment

Outcome measures

Primary

  • Neutrophil Engraftment

    Neutrophil engraftment is defined as achieving an absolute neutrophil count (ANC) of greater than or equal to 500/µL for 3 consecutive measurements on 3 different days by Day 28.

    Time frame: Day 28

  • Serious Adverse Events

    Occurrence of any event classified as SAE. The time of occurrence of each serious adverse event will be recorded.

    Time frame: Day 56

  • CTCAE Grade 3/4 Adverse Events (AEs)

    Occurrence of any event classified as grade 3/4 AE attributed to Ossium HPC, Marrow per the CTCAE v5.0 guidelines. The time of the occurrence of each event will be recorded.

    Time frame: Day 56

  • CTCAE Grade 3/4 Adverse Events (AEs) attributed to infusion of Ossium HPC, Marrow

    Occurrence of any event classified as grade 3 or higher attributed to Ossium HPC, Marrow infusion per the CTCAE v5.0 guidelines. The time of the occurrence will be recorded.

    Time frame: Day 28

  • Death

    The time of death will be recorded for each expired patient.

    Time frame: Day 56

Secondary

  • Cumulative incidences of neutrophil engraftment

    Time frame: Day 28

  • Cumulative incidences of platelet recovery

    Time frame: Day 56

  • Cumulative incidence of disease relapses

    Time frame: Day 365

  • Transplant-related mortality (TRM)

    Time frame: Day 100 and Day 365

  • Cumulative incidences of acute (aGVHD) Graft Versus Host Disease

    Time frame: Day 100, Day 180, and Day 365

  • Cumulative incidences of chronic (cGVHD) Graft Versus Host Disease

    Time frame: Day 100, Day 180, and Day 365

  • Incidence of clinically-significant infections

    Time frame: Day 100 and Day 365

Eligibility criteria

Sex: AllAge: 18 Years to 75 YearsHealthy volunteers: No
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

Study locations (9)

City of Hope

Duarte, California, 91010

Recruiting
Sunmin Park, MD · Principal Investigator

Moffitt Cancer Center

Tampa, Florida, 33612

Recruiting
Taiga Nishihori, MD · Principal Investigator

Emory University - Winship Cancer Institute

Atlanta, Georgia, 30322

Recruiting
Colin Vale, MD · Principal Investigator

Henry Ford Cancer Institute

Detroit, Michigan, 48202

Recruiting
Muneer Abidi, MD · Principal Investigator

Oregon Health and Science University

Portland, Oregon, 97239

Recruiting
Arpita Gandhi, MD · Principal Investigator

TriStar Bone Marrow Transplant

Nashville, Tennessee, 37203

Recruiting
Jeremy Pantin, MD · Principal Investigator

St. David's South Austin Medical Center

Austin, Texas, 78745

Recruiting
Uttam Rao, MD · Principal Investigator

Methodist Hospital, Texas Transplant

San Antonio, Texas, 78229

Recruiting
Nosha Farhadfar, MD · Principal Investigator

University of Utah - Huntsman Cancer Institute

Salt Lake City, Utah, 84112

Recruiting
Sagar Patel, MD · Principal Investigator