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Clinical Trials Search at Vanderbilt-Ingram Cancer Center



Treosulfan-Based Conditioning Regimen before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

Multiple Cancer Types

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.
Hematologic, Pediatrics
II
Connelly, James
NCT04965597
VICCPED2192

Conditioning SCID Infants Diagnosed Early

Multiple Cancer Types

The investigators want to study if lower doses of chemotherapy will help babies with SCID to
achieve good immunity with less short and long-term risks of complications after
transplantation. This trial identifies babies with types of immune deficiencies that are most
likely to succeed with this approach and offers them transplant early in life before they get
severe infections or later if their infections are under control. It includes only patients
receiving unrelated or mismatched related donor transplants.

The study will test if patients receiving transplant using either a low dose busulfan or a
medium dose busulfan will have immune recovery of both T and B cells, measured by the ability
to respond to immunizations after transplant. The exact regimen depends on the subtype of
SCID the patient has. Donors used for transplant must be unrelated or half-matched related
(haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance
of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T
cells removed, using a newer, experimental approach of a well-established technology. Once
the stem cell transplant is completed, patients will be followed for 3 years. Approximately
9-18 months after the transplant, vaccinations will be administered, and a blood test
measuring whether your child's body has responded to the vaccine will be collected.
Hematologic, Pediatrics
II
Connelly, James
NCT03619551
VICCNCPED18122

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

Pediatrics

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.
Pediatrics
III
Connelly, James
NCT05600426
VICCPED2295

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