This trial will study how well a reduced intensity conditioning hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) work in patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
1 Primary · 20 Secondary · Reporting Duration: 2 years
Experimental Treatment
27 Total Participants · 2 Treatment Groups
Primary Treatment: IBMFS · No Placebo Group · Phase 2
Age 4 - 40 · All Participants · 10 Total Inclusion Criteria
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