Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures (NCT04232085)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (38 items) and avoid the listed exclusions (26 items).
Location: Johns Hopkins University — Baltimore, Maryland
Age range: 4 Months to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion criteria
- Patients will not be excluded on the basis of sex, racial or ethnic background.
- Positive leukocytotoxic crossmatch.
- Prior allogeneic stem cell transplant.
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment. The investigators recognize that patients with CAEBV may have ongoing EBV viremia at the time of initiating pre-transplant therapy, but other patients should have no uncontrolled bacterial, viral, or fungal infections.
- Diagnosis of idiopathic aplastic anemia
- Seropositivity for the human immunodeficiency virus (HIV)
- Active Hepatitis B or C determined by serology and/or NAT
- Female patients who are diagnosed as pregnant by beta bHCG testing (per institutional practice) or who are breast-feeding.
- Active malignancy or within the timeframe for significant concern for relapse of prior malignancy
- For Cohort B and C: liver biopsy (if performed, not required) with moderate-severe fibrosis/cirrhosis Donor Eligibility
- Donor must be medically, socially, and psychologically fit to donate
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (1 Sites)
- Johns Hopkins University — Baltimore, Maryland
📞 Contact the Study Coordinator
Phone: (410) 502-9961
Email: hsymons2@jhmi.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
- 💵 Compensation indicated: Growth factor support Patients will receive Granulocyte-colony stimulating factor (GCSF) (Filgrastim®) 5µg/kg/d subcutaneous (SC) or IV starting at Day 5 and continuing until the absolute neutrophi...
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💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.