National Institutes of Health

Bethesda, MD

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Accepting patients

Natural History Study

The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes.
  • Observational Trial

Accepting patients

GATA2 Genetic Testing

The Natural History of GATA2 Deficiency and Related Disorders
  • Genetic Sequencing
  • Observational Trial

Accepting patients

Post-Transplant Cyclophosphamide

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
  • Chemotherapy
  • Allogeneic Stem Cell Transplant
  • Post-Allogeneic Stem Cell Transplant
  • Phase 2

Accepting patients

Allo Stem Cell Transplant for GATA2 Mutations

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome
  • Allogeneic Stem Cell Transplant
  • Phase 2

Accepting patients

Pacritinib

Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/​Myeloproliferative Neoplasms
  • Kinase Inhibitor
  • Phase 1/2

Accepting patients

Safety Study of Cord Blood Units

Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated Transplantation
  • Allogeneic Stem Cell Transplant
  • Phase 2

Accepting patients

Collection of Biological Samples

Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS
  • Observational Trial

Accepting patients

Collection of Tissue Samples for Cancer Research

Tissue Procurement Protocol for the Developmental Therapeutics Clinic, National Cancer Institute (NCI)
  • Observational Trial

Accepting patients

G-CSF Mobilized CD34+ Cells in Transplant

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-Cells
  • Allogeneic Stem Cell Transplant
  • CD34
  • Phase 2

Accepting patients

Autologous T Cells

A Phase I Study of Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Other Hematologic Malignancies
  • T Cell
  • Phase 1