Immunosuppression vs. Stem Cell Transplant for Severe Aplastic Anemia

Palo Alto (17 mi)

Age: < 65

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: N/A

Waitlist Available

Sponsor: Michael Pulsipher, MD

No Placebo Group

Trial Summary

What is the purpose of this trial?The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for pediatric acquired severe aplastic anemia.

Eligibility Criteria

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.

Inclusion Criteria

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

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

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

I am 25 years old or younger.

Exclusion Criteria

I am not pregnant or breast-feeding.

My bone marrow test shows early signs of MDS.

I have had a stem cell transplant from a donor.

I have had a solid organ transplant.

I am HIV positive.

Treatment Details

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.

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.

Cyclophosphamide is already approved in United States, European Union, Canada, Japan for the following indications:

🇺🇸 Approved in United States as Cytoxan for: