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

NCT05589896 | Recruiting Phase 1 DMC

• 1 other identifier: PRESERVE I
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 9

Brief 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.

Conditions

Acute Lymphoblastic Leukemia; Acute Myeloid Leukemia; Acute Biphenotypic Leukemia; Acute 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)

Keywords

Leukemia; Hematologic Diseases; ALL; AML; ABL; AUL; Bone Marrow Transplant; Lymphoma; MDS; CLL; CML

Outcome Measures

Primary Outcomes (5)

• 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.

  Day 28

• Serious Adverse Events

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

  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.

  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.

  Day 28

• Death

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

  Day 56

Secondary Outcomes (7)

• Cumulative incidences of neutrophil engraftment

  Day 28

• Cumulative incidences of platelet recovery

  Day 56

• Cumulative incidence of disease relapses

  Day 365

• Transplant-related mortality (TRM)

  Day 100 and Day 365

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

  Day 100, Day 180, and Day 365

Other Outcomes (2)

• Length of Stay in Hospital

  Day 100 and Day 365

• Time to provide Ossium product to the patient from product availability request

  Day 365

Study Arms (2)

Cohort 1

EXPERIMENTAL

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

Other: Ossium HPC Marrow, Bone Marrow Transplant; Other: Pre-transplant conditioning - Myeloablative (MAC); Other: Pre-transplant conditioning - Reduced Intensity (RIC); Other: Post-transplant treatment

Cohort 2

EXPERIMENTAL

\*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

Other: Ossium HPC Marrow, Bone Marrow Transplant; Other: Pre-transplant conditioning - Myeloablative (MAC); Other: Pre-transplant conditioning - Reduced Intensity (RIC); Other: Post-transplant treatment

Interventions

Pre-transplant conditioning - Myeloablative (MAC) OTHER

Regimen A or Regimen B

Also known as: Busuflex, Fludara, TBI

Cohort 1; Cohort 2

Pre-transplant conditioning - Reduced Intensity (RIC) OTHER

Regimen C or Regimen D

Also known as: Fludara, Cytoxan, Mesnex, TBI, Melphalan

Cohort 1; Cohort 2

Ossium HPC Marrow, Bone Marrow Transplant OTHER

Hematopoetic Cell Transplantation

Cohort 1; Cohort 2

Post-transplant treatment OTHER

Post-transplant treatment

Also known as: Cytoxan, Mesnex, CellCept, Filgrastim

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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
• 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:
• Cardiac: LVEF at rest ≥40% (RIC) or LVEF at rest ≥45% (MAC)
• Pulmonary: DLCO, FEV1, FVC ≥50% predicted by pulmonary function tests (PFTs). DLCO value may be corrected for hemoglobin.
• 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
• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Ossium Health, Inc.: lead

Study Sites (9)

City of Hope

Duarte, California, 91010, United States

RECRUITING

Moffitt Cancer Center

Tampa, Florida, 33612, United States

RECRUITING

Emory University - Winship Cancer Institute

Atlanta, Georgia, 30322, United States

RECRUITING

Henry Ford Cancer Institute

Detroit, Michigan, 48202, United States

RECRUITING

Oregon Health and Science University

Portland, Oregon, 97239, United States

RECRUITING

TriStar Bone Marrow Transplant

Nashville, Tennessee, 37203, United States

RECRUITING

St. David's South Austin Medical Center

Austin, Texas, 78745, United States

RECRUITING

Methodist Hospital, Texas Transplant

San Antonio, Texas, 78229, United States

RECRUITING

University of Utah - Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States

RECRUITING

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Myeloid, Acute; Leukemia, Biphenotypic, Acute; Leukemia, B-Cell; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Anemia, Refractory, with Excess of Blasts; Hodgkin Disease; Lymphoma, T-Cell, Cutaneous; Leukemia; Hematologic Diseases; Lymphoma

Interventions

Bone Marrow Transplantation; fludarabine phosphate; Cyclophosphamide; Mesna; Melphalan; Mycophenolic Acid; Filgrastim

Condition Hierarchy (Ancestors)

Leukemia, Lymphoid; Neoplasms by Histologic Type; Neoplasms; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Leukemia, Myeloid; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Anemia, Refractory; Anemia; Myelodysplastic Syndromes; Lymphoma, T-Cell; Lymphoma, Non-Hodgkin

Intervention Hierarchy (Ancestors)

Tissue Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfhydryl Compounds; Sulfur Compounds; Sulfonic Acids; Sulfur Acids; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Proteins; Biological Factors

Central Study Contacts

Preethi Prasad, M.Sc.

CONTACT

628-842-6562; preethi.prasad@ossiumhealth.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: October 18, 2022
• First Posted: October 21, 2022
• Study Start: August 16, 2024
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): March 31, 2027
• Last Updated: February 24, 2026

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)