Actively Recruiting
Led by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Updated on 2025-11-28
27
Participants Needed
1
Research Sites
52 weeks
Total Duration
S
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Lead Sponsor
M
Maryland Stem Cell Research Fund
Collaborating Sponsor
Researchers are evaluating a Phase II trial to study donor engraftment rates using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) combined with post-transplant cyclophosphamide (PTCy) in patients with primary immune deficiencies, immune dysregulatory syndromes, inherited bone marrow failure syndromes, short telomere syndromes, Fanconi anemia, and related DNA repair disorders. This approach aims to improve donor cell engraftment and outcomes while reducing transplant-related complications. The study addresses challenges like graft failure, transplant mortality, and graft-versus-host disease by using a novel conditioning and prophylaxis regimen. Participants receive a preparative regimen including alemtuzumab, fludarabine, melphalan, and low-dose total body irradiation (for certain patients), followed by bone marrow transplantation on day 0. Post-transplant cyclophosphamide is administered on days 3 and 4 to prevent graft-versus-host disease, followed by immunosuppressive drugs tacrolimus and mycophenolate mofetil starting on day 5. Patients receive supportive care including growth factor, transfusions, infection prophylaxis, and anti-ovulatory treatment for menstruating females. The study includes a single treatment arm with detailed dosing schedules and monitoring. Participants undergo extensive evaluations before transplant, including organ function and disease status assessments. During and after transplant, they are closely monitored for donor engraftment, survival, immune recovery, and complications. The primary outcome is donor engraftment at 60 days post-transplant, with secondary outcomes including one-year survival and disease-free survival. Follow-up continues through the post-transplant period and after discharge. The total participation duration varies per patient depending on clinical course and recovery.
CONDITIONS
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 14 days
Participants receive a reduced intensity conditioning regimen including Alemtuzumab, Fludarabine, Melphalan, and for some, low dose total body irradiation to prepare for bone marrow transplantation.
Daily visits for approximately 14 days for drug infusions and radiation
Duration - 1 day
Participants undergo bone marrow infusion on day 0, following preparative therapy.
1 visit (in-person on day 0)
Duration - Up to approximately 180 days depending on donor type and clinical status
Participants receive post-transplant cyclophosphamide on days 3 and 4, followed by immunosuppressive drugs Tacrolimus and Mycophenolate Mofetil starting on day 5 to prevent graft-versus-host disease and support engraftment.
Multiple visits weekly during the first 2 months, then less frequent follow-ups up to 6 months
Duration - Up to 1 year or longer as per clinical needs
Participants are monitored for immune recovery, graft function, and complications including infections and graft-versus-host disease after transplantation and immunosuppressive therapy.
Regular follow-up visits with frequency decreasing over time
Total: 1 location
1
Johns Hopkins University
Baltimore, Maryland, United States, 21287
Actively Recruiting
H
Heather J Symons, MD, MHS
M
Megan Petrycki, RN
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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Orly R Klein, Samantha Bapty, Howard M Lederman...
https://pubmed.ncbi.nlm.nih.gov/33159275