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Acute LeukemiaPhase 1NCT05170828

Cryopreserved MMUD BM With PTCy for Hematologic Malignancies

A Phase 1 study testing Cyclophosphamide, Fludarabine and Total Body Irradiation in people with Acute Leukemia and Myelodysplastic Syndromes, run by Ossium Health, Inc..

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

Multicenter single arm study to assess the safety and efficacy of allogeneic transplantation using cryopreserved bone marrow from deceased MMUD and PTCy, sirolimus and MMF for GVHD prophylaxis. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Cyclophosphamide, Fludarabine and Total Body Irradiation
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
Sep 2022to Nov 2024
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Provision of signed and dated informed consent form
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • Male or female, aged ≥ 18 and < 71 years (Note: HIV-negative subjects with MDS must be aged <50 at the time of signing the informed consent form)
  • Diagnosed with
  • Acute leukemias or T-lymphoblastic lymphoma (T-LBL) in 1st or subsequent CR
  • Acute lymphocytic leukemia (ALL) or T-LBL as defined by the following:
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  • < 5% blasts in the bone marrow
  • Normal maturation of all cellular components in the bone marrow
  • No currently active extramedullary disease (EMD) (e.g., central nervous system (CNS), soft tissue disease)
  • ANC ≥ 1,000/mm3
  • Acute myeloid leukemia (AML) defined by the following:
  • < 5% blasts in the bone marrow
  • No blasts with Auer rods
  • Normal maturation of all cellular components in the bone marrow
  • No currently active EMD (e.g., CNS, soft tissue disease)
  • ANC ≥ 1,000/mm3
  • Acute biphenotypic leukemia (ABL)/Acute undifferentiated leukemia (AUL) defined by the following:
  • < 5% blasts in the bone marrow
  • Normal maturation of all cellular components in the bone marrow
  • No currently active EMD (e.g., CNS, soft tissue disease)
  • ANC ≥ 1,000/mm3
  • Myleodysplastic Syndromes (MDS), fulfilling the following criteria:
  • Subjects with de novo MDS who have or have previously had Intermediate-2 or High-risk disease as determined by the IPSS. Current Intermediate-2 or High- risk disease is not a requirement
  • Subjects must have < 20% bone marrow blasts, assessed within 60 days of informed consent
  • Subjects may have received prior therapy for the treatment of MDS prior to enrollment
  • Performance status: Karnofsky ≥ 60%
  • Adequate organ function defined as:
  • Cardiac: LVEF at rest ≥ 35% (RIC cohort) or LVEF at rest ≥ 40% (FIC cohort), or LVFS ≥ 25%
  • Pulmonary: DLCO, FEV1, FVC ≥ 50% predicted by pulmonary function tests (PFTs). DLCO value may be corrected or uncorrected for hemoglobin
  • Hepatic: total bilirubin ≤ 2.5 mg/dL, and ALT, AST, and ALP < 5 x ULN (unless ALT, AST, and/or ALP are disease related)
  • Renal: SCr within normal range for age (see table 5.1B). If SCr is outside normal range for age, CrCl > 40 mL/min/1.73m2 must be obtained (measured by 24-hour (hr) urine specimen or nuclear glomerular filtration rate (GFR), or calculated GFR (by Cockcroft-Gault formula)
  • Subjects must have the ability to give informed consent according to applicable regulatory and local institutional requirements
  • Availability of deceased HLA MMUD cryopreserved product through Ossium cryobank
  • HLA MMUD defined as 4-7/8 HLA-allele matching at MHC class (A, B, or C) or MHC class II (DRB1)
  • Additional MHC class II HLA (DP and DQ) typing will be collected, but not incorporated in donor selection

Who cannot join

  • Pediatric patients (17 years or younger)
  • Suitable HLA-matched related or 8/8 allele matched (HLA-A, -B, -C, -DRB1) unrelated donor excluding Ossium product
  • Autologous HCT < 3 months prior to the time of signing the informed consent form
  • Pregnancy or lactation
  • Treatment with an investigational drug or other interventional GVHD clinical trials
  • Current uncontrolled bacterial, viral or fungal infection (currently taking medication with evidence of progression of clinical symptoms or radiologic findings)
Show all 10Show fewer
  • Prior allogeneic HCT
  • Primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia vera
  • Subjects with MDS may not receive RIC and must be < 50 years of age at the time of signing the informed consent form
  • Any condition(s) or diagnosis, both physical or psychological, or physical exam finding that in the investigator's opinion precludes participation

About this study

Official title: PRESERVE: A Multi-Center Trial Using Banked, Cryopreserved HLA-Mismatched Unrelated Donor Bone Marrow and Post-Transplantation Cyclophosphamide in Allogeneic Transplantation for Patients With Hematologic Malignancies

Multicenter single arm study to assess the safety and efficacy of allogeneic transplantation using cryopreserved bone marrow from deceased MMUD and PTCy, sirolimus and MMF for GVHD prophylaxis.

Keywords
Leukemia, MDS, T-LBL, ALL, AML, ABL, AUL, Bone marrow transplant
Medical subject headings
Myelodysplastic Syndromes, Precursor T-Cell Lymphoblastic Leukemia-Lymphoma, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CyclophosphamideDrug

    Also called Cytoxan

    pre-transplant conditioning treatment

  • FludarabineDrug

    Also called Fludara

    pre-transplant conditioning treatment

  • Total Body IrradiationRadiation

    Also called TBI

    pre-transplant conditioning treatment

  • BusulfanDrug

    Also called Busulfelx

    pre-transplant conditioning treatment

  • MesnaDrug

    Also called Mesnex

    given with cyclophosphamide

  • SirolimusDrug

    post-transplant treatment for GVHD

  • Mycophenolate MofetilDrug

    Also called MMF

    post-transplant treatment for GVHD

  • FilgrastimDrug

    Also called G-CSF

    post-transplant treatment for GVHD

  • CyclophosphamideDrug

    Also called Cytoxan

    post-transplant treatment for GVHD

  • Bone Marrow TransplantProcedure or surgery

    Also called Hematopoietic Stem Cell Transplant

    Transplant with investigational bone marrow product

Study groups in NCT05170828
Study groupRoleWhat they receive
Regmin A (RIC)OtherPre-transplant conditioning treatment with Fludarabine, Cyclophosphamide, and Total Body Irradiation (TBI)
Receives: Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total Body Irradiation; Drug: Mesna; Drug: Sirolimus; Drug: Mycophenolate Mofetil; Drug: Filgrastim; Procedure: Bone Marrow Transplant
Regimen B (FIC)OtherPre-transplant conditioning treatment with Busulfan and Cyclophosphamide OR Fludarabine
Receives: Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Busulfan; Drug: Mesna; Drug: Sirolimus; Drug: Mycophenolate Mofetil; Drug: Filgrastim; Procedure: Bone Marrow Transplant
Regimen C (FIC)OtherPre-transplant conditioning treatment with Cyclophosphamide and Total Body Irradiation (TBI)
Receives: Drug: Cyclophosphamide; Radiation: Total Body Irradiation; Drug: Mesna; Drug: Sirolimus; Drug: Mycophenolate Mofetil; Drug: Filgrastim; Procedure: Bone Marrow Transplant

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Neutrophil engraftment

    Run-in safety phase primary endpoint; Neutrophil engraftment is defined as neutrophil recovery by Day 35. Neutrophil recovery is defined as donor-derived absolute neutrophil count (ANC) ≥ 500/mm3 for three consecutive days with evidence of donor chimerism in bone marrow or peripheral blood. Full donor chimerism is >95%, and mixed donor chimerism is defined as 5-95%.

    Measured: Day 35 Post HCT

  2. Cumulative incidence and kinetics of neutrophil and platelet recovery

    Main phase primary endpoint; Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/mm3 for three consecutive measurements on three different days. The first of the three days will be designated the day of neutrophil recovery. The competing event is death without neutrophil recovery. For subjects who never drop ANC below 500/mm3, the date of neutrophil recovery will be Day 1 post-transplant. Platelet recovery is defined by two different metrics: the first day of a sustained platelet count greater than or equal to 20,000/mm3 or greater than or equal to 50,000/mm3 with no platelet transfusions in the preceding seven days. The first day of sustained platelet count above these thresholds will be designated the day of platelet engraftment. For subjects who never drop their platelet count below 20,000/mm3, the date of platelet recovery will be Day 1 post HCT.

    Measured: Day 35 Post HCT

  3. Overall survival (OS)

    Main phase primary endpoint; The primary endpoint for the study is OS at 1-year post-HCT. The event is death from any cause. The time to this event is the time from HCT to death, loss to follow-up, or end of study (whichever comes first). Subjects who are lost to follow-up prior to 1-year will be censored at the time of the last observation, and the OS probability will be estimated using the Kaplan-Meier method.

    Measured: 1-year Post HCT

Secondary outcomes (10)

  1. Cumulative incidences of aGVHD and cGVHD

    aGVHD is defined as any skin, gastrointestinal or liver abnormalities fulfilling the criteria of grades II-IV or grades III-IV. cGVHD is defined per National Institutes of Health (NIH) Consensus Criteria and includes organ involvement and severity, and overall global composite score (mild/moderate/severe).

    Measured: 1-year Post HCT

  2. Transplant-related mortality (TRM)

    TRM is defined as death without evidence of disease progression or recurrence. TRM will be evaluated at Day 100, 180, and 1-year (Day 365).

    Measured: Day 100 and 1-year Post HCT

  3. NK, B- and T-cell immune reconstitution

    Quantitative assessments of peripheral blood CD3, CD4, CD8, CD19 positive lymphocytes will be done through flow cytometric analysis at Days 35, 100, 180, and 365 post-HCT. Qualitative assessments will be tabulated according to time from transplant.

    Measured: Days 35, 100, 180, and 1-year Post HCT

  4. Progression free survival (PFS)

    Progression free survival will be estimated at 1-year (Day 365) post-transplant. An event for this time to event outcome is defined as disease relapse or progression, or death by any cause.

    Measured: 1-year Post HCT

  5. Event free survival (EFS)

    EFS will be estimated at 12 months post-HCT. An event for this time to event outcome includes graft failure (PGF by Day 35 or secondary graft failure), relapse or progression of underlying disease, death, grade III-IV acute GVHD, or NIH-severe chronic GVHD.

    Measured: 1-year Post HCT

  6. GVHD relapse free survival (GRFS)

    GRFS will be estimated at 1-year (Day 365) post-transplant. An event for this time to event outcome is defined as relapse or progression of underling disease, graft failure, grade III-IV acute GVHD, chronic GVHD requiring systemic immune suppression, or death by any cause.

    Measured: 1-year Post HCT

  7. Cumulative incidence of cytokine release syndrome (CRS)

    Overall incidence of CRS of any grade and grade 3 or 4 CRS post-transplant will be reported on all patients. CRS will be defined and graded using the American Society for Transplantation and Cellular Therapy (ASTCT) consensus grading criteria.

    Measured: Day 14 Post HCT

  8. Donor chimerism

    Peripheral blood chimerism (% of donor chimerism) in whole blood (unsorted) will be described on Day 35, 56, 100, 180, and 1-year (Day 365). Donor cell engraftment will be assessed with donor/recipient chimerism studies at Day 100. Chimerism may be assessed in CD3 blood cell fraction. For the purpose of this protocol, mixed chimerism is defined as the presence of donor cells, as a proportion of total cells to be less than 95% but at least 5% in the bone marrow or peripheral blood. Full donor chimerism is defined as greater than or equal to 95% of donor cells. Mixed and full chimerism will be evidence of donor cell engraftment. Donor cells of less than 5% by Day 35 will be considered as graft rejection. The proportion of patients with each level of chimerism described above will be described as part of this outcome. For sorted blood cell fractions, CD3+ donor cell chimerism will be used to define the donor/recipient chimerism status.

    Measured: Days 35, 56, 100, 180, and 1-year Post HCT

  9. Cumulative incidences of viral reactivations and infections

    The incidence of Cytomegalovirus (CMV), Epstein-Barr virus (EBV), BK virus, Adenovirus (ADV), and Human herpesvirus (HHV-6) viral activations and infections at Days 100, 180 and 1-year (Day 365).

    Measured: Days 100, 180, and 1-year Post HCT

  10. Cumulative incidence of primary disease relapse/progression

    Testing for recurrent malignancy in the blood, bone marrow or other sites will be used to assess relapse and progression post-HCT. For the purpose of this study, relapse is defined by either morphological, cytogenetic/molecular or radiological evidence of disease consistent with pre-HCT features, in line with institutional standards. The event for this endpoint is the time interval from HCT to relapse/recurrence of disease or to last follow-up through 1-year (Day 365) post-HCT. Death in remission is considered a competing risk.

    Measured: 1-year Post HCT

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Dec 2021
  2. Study startSep 2022 (estimated)
  3. Primary completionMar 2024 (estimated)
  4. Study completionNov 2024 (estimated)

Why it stopped: Change in Study Design

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05170828
Sponsor's protocol ID
PRESERVE
Status last verified
Feb 2026
First submitted
12 Nov 2021
Last updated
24 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Johnstone BH, Woods JR, Goebel WS, Gu D, Lin CH, Miller HM, Musall KG, Sherry AM, Bailey BJ, Sims E, Sinn AL, Pollok KE, Spellman S, Auletta JJ, Woods EJ. Characterization and Function of Cryopreserved Bone Marrow from Deceased Organ Donors: A Potential Viable Alternative Graft Source. Transplant Cell Ther. 2023 Feb;29(2):95.e1-95.e10. doi: 10.1016/j.jtct.2022.11.010. Epub 2022 Nov 17.

    PubMed 36402456

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cyclophosphamide, Fludarabine and Total Body Irradiation in people with Acute Leukemia and Myelodysplastic Syndromes, run by Ossium Health, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 35 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 24 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Neutrophil engraftment (time frame: Day 35 Post HCT). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05170828, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2026.

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