Overview

Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide

Status:
Recruiting
Trial end date:
2026-12-31
Target enrollment:
Participant gender:
Summary
This is a Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), and inherited bone marrow failure syndromes (IBMFS).
Phase:
Phase 2
Details
Lead Sponsor:
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Treatments:
Alemtuzumab
Cyclophosphamide
Fludarabine
Fludarabine phosphate
Melphalan
Mycophenolic Acid
Tacrolimus