Clinical Research Directory

Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults

This single institution, phase I clinical trial will determine the safety and feasibility of employing T-cell receptor (TCR) αβ+ and CD19+ (Cluster of Differentiation ) depleted hematopoietic stem cell transplantation (HSCT) using peripheral blood stem cells (PBMC) from closely matched unrelated donors or haploidentical donors to treat non-malignant hematologic diseases in children and young adults. Allogeneic hematopoietic stem cell transplantation has become a curative option for children and adolescents with a variety of otherwise fatal conditions. To reduce the incidence and severity of graft-versus-host disease (GVHD) associated with allogeneic hematopoietic stem cell transplantation, donor grafts are depleted of T cells, either using CD34+ selection or CD3+/CD19+ depletion of grafts. However, these selection processes also deplete the graft of protective cell subsets, such as γδ T cells, natural killer(NK) cells, monocytes and dendritic cells, which play important roles in the immune response to infectious agents. Moreover, the presence of NK cells and γδ T in donor grafts is associated with more rapid immune reconstitution after HSCT transplantation. In order to retain these protective immune cell subsets, this trial will use a novel, highly selective graft engineering process using the Miltenyi CliniMACS system that selectively depletes αβ-T cells and B cells which are responsible for GVHD and Epstein Barr Virus (EBV)-related post-transplantation lymphoproliferative disorder, respectively. Prior to transplantation, patients will be treated with a conditioning regimen, specific for the original disorder. The primary objective of this study is evaluation of the safety and feasibility of HSCT using TCRαβ+/CD19+ depleted hematopoietic stem cells to treat non-malignant hematologic diseases. This will be assessed by evaluating the incidence of graft failure, grade III-IV acute GVHD and chronic GVHD and TRM. Secondary objectives include the evaluation of immune reconstitution and incidence of post-transplant infections, adverse events, serious adverse events, overall and disease-free survival and the efficiency of graft processing by the CliniMACS System.

Diamond Blackfan Anemia Registry (DBAR)

The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA).

Role of Transcranial Doppler and Magnetic Resonance Angiography for Future Management of Sickle Cell Anemia CNS.

This study aimed to determine the Predictive Value of Transcranial Doppler and Magnetic Resonance Angiography for Future Management of Sickle Cell Anemia. Specific aims are: Demonstrate silent parenchymal and vascular brain changes that are incidentally observed in neurologically free SCD children using screening TCD and MRA in Pediatric Hematology unit at Assiut University Hospital Detect any abnormality with vasculopathy, arterial occlusion and old SCI. Strokes in children with SCD can be prevented by checking a transcranial Doppler (TCD) ultrasound,MRA and providing blood transfusions to children with abnormal blood flow on the TCD and detect Silent cerebral and cerebrovascular changes in SCD.

Collection and Distribution of Biospecimens for Novel Research Uses

iSpecimen aims to create a clinical partner network of hospitals, laboratories, academic institutions, and other healthcare organizations ("institutions") capable of providing researchers and educators ("researchers") with annotated biospecimens for use in biomarker discovery and validation; diagnostic test and instrumentation development and validation; therapeutics development; other medical research including the impact that various specimen collection and handling methods and conditions have on research results; and in education such as researcher or physician training (collectively "research").