Status: Recruiting
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
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), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.
Participants needed: 27
Trial details
Phase: Phase 2Age: 4-50Biological sex: AllType: InterventionalSponsor: Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUpdated: Nov 28, 2025Locations: 1
Eligibility criteria
Chronic granulomatous disease (CGD) [+35]
Patients will not be excluded on the basis of sex, racial or ethnic background. [+22]