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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
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
H
Heather J Symons, MD, MHS
M
Megan Petrycki, RN
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Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Lead Sponsor
Maryland Stem Cell Research Fund
Collaborating Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | Investigational preparative regimen (trial) | Current reduced‑intensity conditioning regimen |
|---|---|---|
| What it is | A multi‑drug reduced‑intensity conditioning regimen combining alemtuzumab, fludarabine, melphalan, low‑dose total body irradiation followed by GVHD prophylaxis (cyclophosphamide, tacrolimus, mycophenolate mofetil) for allogeneic hematopoietic cell transplant | Reduced‑intensity conditioning (RIC) regimens combining chemotherapy agents and low‑dose radiation are commonly used in clinical practice for IBMFS to balance efficacy and reduce regimen‑related toxicity |
| Role in the study | Assigned preparative regimen administered before transplant to all trial participants across cohorts | Represents clinical approach outside trial rather than a study arm |
| Primary purpose | To prepare recipients for donor cell engraftment while reducing toxicity | To enable engraftment in fragile genetic disease populations with lower toxicity compared to traditional myeloablative regimens |
| How it is administered | Given as a sequential regimen of chemotherapy drugs and low‑dose radiation before transplant, followed by standard GVHD prophylaxis | Administered using milder chemotherapy and radiation protocols adapted for inherited syndrome patients to reduce regimen‑related toxicity |
| Supporting evidence | Current trial evaluates safety and engraftment outcomes but lacks published results | Published literature provides evidence that reduced‑intensity regimens improve transplant tolerate‑ability in IBMFS |
| Important limitations | Safety and long‑term outcomes are not yet established | Does not address non‑hematologic disease manifestations and may still carry late toxicity risks |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
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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
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/Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Lead Sponsor
Maryland Stem Cell Research Fund
Collaborating Sponsor
