A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

Summary

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia).

This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA.

The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms.

This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

Conditions

• Severe Aplastic Anemia

Interventions

DRUG

cyclosporine

cyclosporine

PROCEDURE

Matched Unrelated Donor Hematopoetic Stem Cell Transplant

Matched Unrelated Donor (MUD) Hematopoietic Stem Cell Transplantation (HSCT)

DRUG

horse anti-thymocyte globulin (ATG)

horse anti-thymocyte globulin (ATG)

DRUG

rabbit anti-thymocyte globulin (ATG)

rabbit anti-thymocyte globulin (ATG)

DRUG

Methotrexate

methotrexate

DRUG

Fludarabine

fludarabine

DRUG

Cyclophosphamide

cyclophosphamide

RADIATION

low-dose total body irradiation (TBI)

low-dose total body irradiation (TBI)

PROCEDURE

Immunosuppressive Therapy (IST)

Immunosuppressive Therapy (IST)

Sponsors & Collaborators

• Center for International Blood and Marrow Transplant Research

  collaborator NETWORK

• National Institutes of Health (NIH)

  collaborator NIH

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• North American Pediatric Aplastic Anemia Consortium

  collaborator UNKNOWN

• Pediatric Transplantation and Cellular Therapy Consortium

  collaborator OTHER

• Blood and Marrow Transplant Clinical Trials Network

  collaborator NETWORK

• Boston Children's Hospital

  lead OTHER

Principal Investigators

• David Williams, MD · Boston Children's Hospital

• Michael Pulsipher, MD · University of Utah

• Bronwen Shaw, MD · CIBMTR/Medical College of Wisconsin (MCW)

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

0 Years

Max Age

25 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-01-25

Primary Completion

2029-12-31

Completion

2029-12-31

Countries

• United States
• Canada

Study Locations