Clinical Trial

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

Recruiting Phase 2
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Summary
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.
Protocol Amendment History 11 amendments
This ClinicalTrials.gov record has been amended 11 times since 2020-01-14; most recent amendment 2025-11-21.
Status change: Not Yet Recruiting → Recruiting 2020-01-17
Trial Details
NCT Number NCT04232085
Lead Sponsor Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators: Maryland Stem Cell Research Fund
Conditions Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length, Fanconi Anemia, Non Fanconi DNA-DSB Repair Disorder, Hoyeraal-Hreidarsson Syndrome, Dyskeratosis Congenita +2 more
Enrollment 27 participants
Start Date 2020-02-12
Primary Completion 2027-12-31 (estimated)
Study Completion 2028-12-31 (estimated)
Updated on ClinicalTrials.gov 2025-11-28