What side effects are associated with this type of intervention?

Common treatments for Acute Undifferentiated Leukemia, particularly HLA-haploidentical related hematopoietic cell transplant (Haplo-HCT), are associated with several significant side effects. These include graft-versus-host disease (GVHD), which can be chronic and extensive, increased susceptibility to infections due to immunosuppression, and organ toxicity affecting the liver and kidneys. Patients may also experience pancytopenia, leading to higher risks of bleeding and infection, and there is a notable risk of treatment-related mortality. Long-term complications can include secondary malignancies.

What prior FDA approvals exist?

The FDA has approved several treatments for acute leukemias, including chemotherapeutic agents and targeted therapies. For treatments similar to HLA-haploidentical related hematopoietic cell transplant (Haplo-HCT), which involves replacing diseased bone marrow with healthy donor cells, the FDA has approved various conditioning regimens and supportive therapies. These include fludarabine and melphalan, often used in reduced-intensity conditioning regimens, and total body irradiation (TBI). Additionally, drugs like azacitidine and decitabine are used as hypomethylating agents in the treatment of acute myeloid leukemia (AML), which shares some treatment approaches with acute undifferentiated leukemia.

What other types of research exist?

Promising novel alternatives to HLA-haploidentical related hematopoietic cell transplant (Haplo-HCT) for Acute Undifferentiated Leukemia (AUL) patients include: 1. Chimeric Antigen Receptor T-cell (CAR-T) therapy, which has shown efficacy in various hematologic malignancies and is being explored for AUL. 2. Bispecific T-cell Engagers (BiTEs), which are designed to direct the body's immune system to target and destroy leukemia cells. 3. Targeted therapies, such as FLT3 inhibitors or IDH inhibitors, which are tailored to specific genetic mutations found in leukemia cells. 4. Nonmyeloablative allogeneic stem cell transplantation, which uses less intensive conditioning regimens to reduce toxicity while maintaining efficacy.

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Alkylating agents

Bone Marrow Transplant for Blood Cancers

Phase 2

Waitlist Available

Led By Hany Elmariah, MD, MS

Research Sponsored by H. Lee Moffitt Cancer Center and Research Institute

Eligibility Criteria Checklist

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

Must have

Adequate performance status is defined as Karnofsky score ≥ 70%

Natural killer cell malignancies

Must not have

Active HIV infection

Active central nervous system malignancy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 18 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a stem cell transplant for patients who need a transplant but don't have a perfect match. The process uses a less intense preparation method and aims to see if patients remain disease-free over time.

Who is the study for?

This trial is for people aged 55+ or with certain health conditions, lacking a fully matched sibling donor, and with specific blood cancers in remission or sensitive to chemotherapy. They must have good organ function, agree to birth control if applicable, and have an HLA-haploidentical relative as a donor.

What is being tested?

The study tests a transplant of blood cells from half-matched relatives using lower doses of Fludarabine and Melphalan drugs plus whole-body radiation. The goal is to see how many patients are disease-free after 18 months.

What are the potential side effects?

Potential side effects include weakened immune system leading to infections, nausea, mouth sores due to chemotherapy; skin irritation from radiation; and possible organ damage related to the treatment.

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 and carry out daily activities.

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My condition is related to natural killer cell malignancies.

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I have CML in the accelerated phase and TKIs haven't worked for me or I can't tolerate them.

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My leukemia is in remission.

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My blood disorder is severe but with less than 5% cancer cells.

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My blood cancer has less than 5% blast cells, or I've had treatment to reduce it to this level.

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My Burkitt's lymphoma is in its second or later remission.

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My selected donor and I have undergone detailed HLA typing and share a HLA haplotype.

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My AML is in remission with less than 5% of cells being blasts.

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My lymphoma has returned after treatment, is responsive to chemotherapy, but I can't have a stem cell transplant.

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My T-cell lymphoma has returned but responds to chemotherapy and I can't have a stem cell transplant.

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I am 55 or older, or I have significant health issues.

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I do not have a fully matched family donor for a transplant.

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My leukemia/lymphoma is in its second or more remission, or I had a bad reaction to initial treatment.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently living with HIV.

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I have an active brain or spinal cord tumor.

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My AML is considered low risk based on specific genetic features.

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I currently have an infection that hasn't been treated.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 18 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 18 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 18 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Disease Free Survival

Secondary study objectives

Graft vs Host Disease (GVHD) free survival

Overall Survival (OS)

Relapse Free Survival (RFS)

+2 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: Conditioning Regimen + TransplantExperimental Treatment3 Interventions

All participants will receive a conditioning regimen of Fludarabine, Melphalan and Total Body Irradiation prior to transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 4

~1860

Melphalan

2008

Completed Phase 3

~1500

Total Body Irradiation

2006

Completed Phase 3

~820

0 / 18Eligibility 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 Undifferentiated Leukemia (AUL) include hematopoietic cell transplantation (HCT), which replaces diseased or damaged bone marrow with healthy donor cells. This process is crucial for AUL patients as it helps to restore normal blood cell production and immune function. Reduced-intensity conditioning regimens, such as those using fludarabine and melphalan, are often employed to make the transplant process more tolerable, especially for older or medically unfit patients. These regimens work by suppressing the patient's immune system to prevent rejection of the donor cells while minimizing toxicity. The healthy donor cells then engraft in the patient's bone marrow, leading to the production of healthy blood cells and potentially curing the leukemia.

Influence of Donor Type (Sibling versus Matched Unrelated Donor versus Haploidentical Donor) on Outcomes after Clofarabine-Based Reduced-Intensity Conditioning Allograft for Myeloid Malignancies.Haploidentical vs identical-sibling transplant for AML in remission: a multicenter, prospective study.Place of HSCT in treatment of childhood AML.