Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Recruiting Phase 2 Interventional Study
Severe Aplastic Anemia (SAA) Hypo-Plastic Myelodysplastic Syndrome (MDS) Paroxysmal Nocturnal Hemoglobinuria (PNH)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
4 – 75
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
56 (estimated)
Sponsor
National Heart, Lung, and Blood Institute (NHLBI) · NIH
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About This Trial
Background: Severe aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA - RECIPIENT: * Diagnosed with severe aplastic anemia with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or neutropenia (absolute neutrophil count less than or equal to 1000 cells/ microL or for patients receiving…
Contacts

Jennifer A Farren

(301) 318-2095

jennifer.farren@nih.gov

CONTACT

Richard W Childs, M.D.

(301) 768-9433

childsr@nhlbi.nih.gov

CONTACT