What side effects are associated with this type of intervention?

The most common treatments for Acute Leukemia, particularly allogeneic bone marrow transplantation, are associated with several significant side effects. These include graft-versus-host disease (GVHD), where the donor's immune cells attack the recipient's body, secondary malignancies, chronic immunosuppression leading to increased infection risk, and cardiac and pulmonary toxicity. Chemotherapy, often used before transplantation, can cause pancytopenia (a reduction in blood cell counts), infection, hepatic impairment, and neuropathy. These side effects highlight the need for careful monitoring and supportive care during and after treatment.

What prior FDA approvals exist?

The FDA has approved several treatments for Acute Leukemia, particularly focusing on hypomethylating agents such as azacitidine and decitabine. Azacitidine is approved by the European Medicines Agency (EMA) for AML arising from myelodysplastic syndromes (MDS), while the FDA has approved an oral form of azacitidine (CC-486) for post-remission maintenance therapy of AML. Decitabine is used in both outpatient and inpatient settings for AML treatment. These agents are well-tolerated and have shown comparable efficacy. Additionally, allogeneic bone marrow transplantation, which involves replacing diseased bone marrow with healthy donor marrow, is a critical treatment option for achieving long-term remission in patients with hematologic malignancies.

What other types of research exist?

Promising novel alternatives to allogeneic bone marrow transplantation for acute leukemia patients include: 1) CAR T-cell therapy, which involves modifying a patient's own T-cells to target and kill leukemia cells; 2) Bispecific T-cell engagers (BiTEs), which are designed to direct the body's immune system to attack leukemia cells; 3) Targeted therapies such as FLT3 inhibitors for patients with specific genetic mutations; and 4) Immune checkpoint inhibitors, which help to enhance the body's immune response against leukemia cells. These therapies are being actively researched and have shown encouraging results in clinical trials.

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Bone Marrow Transplant

Bone Marrow Transplant for Leukemia

Phase 1 & 2

Recruiting

Research Sponsored by Ossium Health, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Karnofsky performance status score ≥70% (MAC) or ≥60% (RIC)

Cardiac: LVEF at rest ≥45% (RIC) or LVEF at rest ≥40% (MAC)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 100, day 180, and day 365

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the safety of using bone marrow from a deceased donor to treat patients with severe leukemia. The goal is to see if this new bone marrow can help produce healthy blood cells. Patients will be monitored closely for any side effects and overall effectiveness over several months.

Who is the study for?

This trial is for adults aged 18-55 (or up to 70 for a specific regimen) with certain types of acute leukemia in remission, who need a bone marrow transplant and match the donor's tissue type at least half-way. They must be able to consent, have decent heart function and overall health status, and commit to study procedures for one year.

What is being tested?

The study tests the safety of bone marrow transplants from deceased donors in patients with blood cancers. It compares two pre-transplant conditioning regimens: myeloablative (stronger) or reduced intensity (milder), followed by close monitoring for nearly two months post-transplant.

What are the potential side effects?

Potential side effects include reactions related to chemotherapy drugs like Busulfan and Fludarabine such as nausea, fatigue, mouth sores; complications from radiation therapy; immune suppression risks from Tacrolimus and Mycophenolate Mofetil; infection risk due to low white cell counts.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am mostly able to care for myself but may need help.

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My heart's pumping ability is within the required range.

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I am partially or fully matched for a bone marrow transplant.

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My lung function tests are at least half of what is expected for someone healthy.

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I have acute leukemia in remission with minimal bone marrow involvement.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 100, day 180, and day 365

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 100, day 180, and day 365

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 100, day 180, and day 365 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

CTCAE Grade 3/4 Adverse Events (AEs)

Death

Neutrophil Engraftment

+1 more

Secondary study objectives

Cumulative incidence of disease relapses

Graft-vs-Host Disease

Cumulative incidences of chronic (cGVHD) Graft Versus Host Disease

+4 more

Other study objectives

Length of Stay in Hospital

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

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment4 Interventions

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

Group II: Cohort 1Experimental Treatment3 Interventions

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

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Acute Leukemia include chemotherapy, targeted therapy, and allogeneic bone marrow transplantation. Chemotherapy works by using potent drugs to kill rapidly dividing cancer cells, but it also affects normal cells, leading to significant side effects. Targeted therapy involves drugs that specifically target cancer cell molecules, minimizing damage to normal cells. Allogeneic bone marrow transplantation replaces diseased bone marrow with healthy donor marrow, which can restore normal blood cell production and provide a new immune system capable of attacking residual leukemia cells. This is particularly important for Acute Leukemia patients as it offers a potential cure by eradicating the diseased marrow and re-establishing healthy hematopoiesis.