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Study identifier: NCT04232085 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

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

Condition: Primary Immune Deficiency Disorder · Immune Deficiency Disease · Bone Marrow Failure  ·  Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

PhasePhase 2
Planned participants27
Who can joinAll sexes, 4 Months to 50 Years
Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Heather J Symons, MD, MHS  ·  4105029961  ·  hsymons2@jhmi.edu

Megan Petrycki, RN  ·  4109555068  ·  mpetryc1@jhmi.edu

Always discuss trial participation with your own doctor first.

Locations (1)

Johns Hopkins UniversityBaltimore, Maryland, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT04232085