Category:Pediatric
Status:Active
This trial will assess the efficacy and safety of autologous CD34+ hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector, for the treatment of childhood cerebral adrenoleukody...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 2 / 3
Protocol Number:
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease and a predisposition to cancer are also frequent causes...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 2
Protocol Number: 16-294
Gut Decontamination In Pediatric Allogeneic Hematopoietic
This research study is for participants who are undergoing allogeneic hematopoietic stem cell transplantation (HSCT) and are at risk for developing acute graft-versus-host disease (GVHD). GVHD is a...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 2
Protocol Number: 15-394
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease
Chronic Granulomatous Disease (CGD) is an inherited immunodeficiency disorder which results from defects that prevent white blood cells from effectively killing bacteria, fungi and other microorgan...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase: 1 / 2
Protocol Number:
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
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 s...
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase:
Protocol Number:
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral bl...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase:
Protocol Number:
This research study is studying a targeted therapy drug as a possible treatment for IDH2 mutant acute myeloid leukemia or chronic myelomonocytic leukemia while undergoing hematopoietic stem cell tr...
Diagnosis: Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase:
Protocol Number: 18-022
Letermovir Treatment for Refractory or Resistant Cytomegalovirus Infection
The trial will evaluate the safety and efficacy of letermovir antiviral treatment of active cytomegalovirus infection or cytomegalovirus disease in patients with infections that are refractory or r...
Diagnosis: Hematopoietic Stem Cell Transplant, Pediatric Hematopoietic Stem Cell Transplant (HSCT)
Phase:
Protocol Number: 18-348