National Institutes of Health (Bethesda)

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Accepting patients

Collection of Biological Samples

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

Accepting patients

Omidubicel (former CordIn)

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, to Expedite Engraftment and Improve Transplant Outcome
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  • Cord Blood
  • Phase 1/2
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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
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  • Allogeneic Stem Cell Transplant
  • CD34
  • Phase 2

Accepting patients

Safety Study of Cord Blood Units

A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBUs) Manufactured by the National Cord Blood Program (NCBP) and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
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  • Allogeneic Stem Cell Transplant
  • Phase 2

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
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  • Allogeneic Stem Cell Transplant
  • Chemotherapy
  • Phase 2

Accepting patients

GATA2 Genetic Testing

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

Accepting patients

Allo Stem Cell Transplant for GATA2 Mutations

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

Accepting patients

Eltanexor Plus Inqovi

A Phase I/II Trial of Eltanexor (KPT-8602) With Inqovi (Decitabine-Cedazuridine) in High-Risk Myelodysplastic Syndromes
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  • Nucleoside Metabolic Inhibitor
  • SINE
  • Phase 1/2

Accepting patients

JSP191-Based Conditioning

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency
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  • Allogeneic Stem Cell Transplant
  • Monoclonal Antibody
  • Phase 2

Not yet accepting

Pacritinib

Phase I/II Trial of 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
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  • Kinase Inhibitor
  • Phase 1/2