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Alkylating agents

Immunosuppression vs. Stem Cell Transplant for Severe Aplastic Anemia

N/A

Waitlist Available

Research Sponsored by Michael Pulsipher, MD

Eligibility Criteria Checklist

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

Must have

Confirmed diagnosis of idiopathic SAA with bone marrow cellularity <25%, or <30% hematopoietic cells and two out of three of the following (in peripheral blood): neutrophils <0.5 x109/L, platelets <20 x109/L, reticulocyte count <60 x109/L with hemoglobin <8g/dL.

No suitable fully matched related donor available (minimum 6/6 match for Human Leukocyte antigen (HLA) -A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing).

Must not have

Female patients who are pregnant or breast-feeding.

Clonal cytogenetic abnormalities or fluorescence In Situ Hybridization (FISH) pattern consistent with pre-myelodysplastic syndrome (pre-MDS) or MDS on marrow examination (see section 4.2.3.1 for details of the required MDS FISH panel).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 years

Awards & highlights

No Placebo-Only Group

Summary

This trial will compare outcomes of immunosuppressive therapy vs. stem cell transplant to treat pediatric severe aplastic anemia.

Who is the study for?

This trial is for children and young adults up to 25 years old with severe aplastic anemia, who don't have a closely matched family donor for stem cell transplant. They should have specific low blood counts, no inherited bone marrow failure syndromes or certain other conditions, and at least two potential unrelated donors.

What is being tested?

The study compares two treatments: immunosuppressive therapy (IST) versus a stem cell transplant from an unrelated donor (MUD HSCT). It includes drugs like fludarabine and cyclophosphamide, anti-thymocyte globulin from rabbits or horses, methotrexate, cyclosporine, and low-dose total body irradiation.

What are the potential side effects?

Possible side effects include allergic reactions to medications especially horse ATG if known allergy exists; damage to organs such as the liver or kidneys; increased risk of infections due to immune suppression; mouth sores; nausea; diarrhea.

Eligibility Criteria

Inclusion Criteria

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

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I have severe aplastic anemia with very low blood cell counts.

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I don't have a perfectly matched family donor for a transplant.

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I have at least two unrelated bone marrow donors who match me closely.

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I am 25 years old or younger.

Exclusion Criteria

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

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I am not pregnant or breast-feeding.

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My bone marrow test shows early signs of MDS.

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I have had a stem cell transplant from a donor.

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I have had a solid organ transplant.

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I am HIV positive.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Percentage of patients randomized to HSCT that actually complete HSCT

Secondary study objectives

Paroxysmal nocturnal hemoglobinuria

Incidence of significant infection in both treatment arms

Number of patients that fail to receive their primary assigned therapy (HSCT or IST).

+15 more

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Active Control

Group I: Matched Unrelated Stem Cell TransplantActive Control7 Interventions

Patient will under go matched unrelated donor transplant of hematopoietic stem cells as their therapy using fludarabine, cyclophosphamide, rabbit anti-thymocyte globulin (ATG), and low-dose total body irradiation (TBI) as preparative regimen and cyclosporine and methotrexate for graft versus host disease (GVHD) prevention.

Group II: Immunosuppressive TherapyActive Control3 Interventions

Patient will receive standard immunosuppressive therapy combination of drugs: horse anti-thymocyte globulin (ATG) and cyclosporine.

0 / 9Eligibility criteria met