|Brief Description||Principal Investigator|
|To characterize the pharmacokinetic profile of rivaroxaban administered as dry powder for suspension formulation.||Hinson, Ashley Rebekah Presar|
I. To maintain a Childhood Cancer Registry for infants, children, adolescents, and young adults with cancer.
II. To utilize clinical and biological data to help determine eligibility or stratification, based on childhood cancer disease classification schemas, for potential enrollment of research subjects onto Children's Oncology Group (COG) therapeutic clinical trials.
III. To develop a well annotated childhood cancer biobank for current and future research through the collection of biospe
|Kaplan, Joel Adam|
|To determine the maximum tolerated dose (MTD), the dose limiting toxicities(DLT) and the dose of FT-1101 recommended for future phase 2 studies (RP2D) for patients with relapsed refractory acute leukemia or high-risk MDS.||Grunwald, Michael Richard|
Primary: Assess the efficacy of ruxolitinib in combination with corticosteroids in subjects with Grade IIB-IVD acute graft-versus-host disease (GVHD).
Secondary: Assess additional response and longer-term efficacy outcomes in the study population. Assess the incidence and severity of adverse events (AEs) and serious AEs. Evaluate the pK of ruxolitinib when administered in combination with corticosteroids.
Exploratory: Incidence of secondary graft failure. Incidence of GVHD flares. Utilizatio
|Grunwald, Michael Richard|
To collect high quality specimens from patients with hematologic disorders and normal volunteers.
Future specimen analyses will be performed to determine the differences between sick and normal cells. Data used for these comparisons will include, but are not limited to: gene expression data, DNA, RNA, epigenetics, proteomics, metabolomics, and pathway analysys.
|Avalos, Belinda Rene|
Primary: To collect high quality biospecimens to study plasma cell disorders.
Exploratory: 1. To determine the differences between precurser states and multiple myeloma using gene expression data, DNA sequencing, RNA studies, epigenetics, proteomics, metabolomics, pathway analysis, and immunophenotypic analysis. 2. To standardize MRD assays and validate prognostic MRD thesholds for MRD monitoring as a tool for risk-adapted clinical trials. 3. To evaluate biomarkers and immune markers associated
|The primary objective of this study is to examine the incidence of neutrophil recovery of > or = to 500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed.||Huo, Jeffrey|
|The primary objective is to assess the incidence rate of FVIII inhibitors during long-term prevention and treatment of bleeds with turoctocog alfa in previously FVIII treated patients with severe and moderately severe haemophilia A.||Hinson, Ashley Rebekah Presar|
|To estimate the incidence of non-relapse mortality at 180 days following myeloablative haploidentical BMT for children and young adults with high risk hematologic malignancies.||Huo, Jeffrey|
|To determine the impact that abatacept will make on the incidence of early, severe acute GVHD (aGvHD), when it is added to a standard GvHD prophylaxis regimen during unrelated-donor hematopoietic stem cell transplantation (HSCT) for patients with hematologic malignancies.||Huo, Jeffrey|
|The purpose of this study is to determine if treatment with alemtuzumab, fludarabine and melphalan followed by related/unrelated bone marrow, peripheral blood stem cell, or umbilical cord blood cell transplant will result in donor engraftment on day +100. It is also to determine major toxicities from this conditioning regimen, within the first 100 days after transplantation.||Huo, Jeffrey|