Pediatric Hematopoietic Stem Cell Transplant (HSCT) Clinical Trials

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)

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

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Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

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

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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)

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Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

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)

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IDH2 Inhibition Using Enasidenib as Maintenance Therapy for IDH2-mutant Myeloid Neoplasms Following Allogeneic Stem Cell Transplantation

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)

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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)

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Protocol Number: 18-348