Actively Recruiting
T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure Disorders
Led by Masonic Cancer Center, University of Minnesota · Updated on 2026-01-30
48
Participants Needed
1
Research Sites
105 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell transplantation in patients with inherited bone marrow failure (BMF) disorders. The goal is to avoid routine immune suppression for graft-versus-host disease (GVHD), allowing earlier immune recovery and potentially reducing severe infection risks after transplantation. This trial is sponsored by the Masonic Cancer Center, University of Minnesota. Participants receive one of several treatment plans involving drugs such as total body irradiation (TBI), cyclophosphamide, fludarabine, methylprednisolone, rituximab, busulfan, alemtuzumab, melphalan, and a T cell receptor α/β depleted peripheral blood stem cell infusion on day 0. The conditioning regimens vary based on disease features, donor type, and patient tolerance, with some patients receiving TBI and others alternative drug combinations. G-CSF is administered after transplant to support neutrophil recovery. During the study, participants undergo monitoring for immune recovery and complications including acute and chronic GVHD, engraftment of neutrophils and platelets, infections, regimen-related toxicity, and overall survival up to one year after transplant. Evaluations include clinical assessments and laboratory tests at defined time points such as day 42, day 100, and one year post-transplant. The study aims to describe outcomes for approximately 3 to 5 patients treated annually under these protocols.
CONDITIONS
Brief Title
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Fanconi anemia with age under 65 years
- Severe aplastic anemia, myelodysplastic features, high risk genotype, or immunodeficiency with recurrent infections
- Karnofsky performance status ≥ 70% if 16 years or older, or Lansky play score ≥ 50% if younger than 16
- Adequate pulmonary, cardiac, and liver function
- Voluntary written consent (minor assent if appropriate)
- Diagnosis of telomere biology disorder with age under 70 years
- Same risk factors and performance status as for Fanconi anemia patients
- Adequate organ function
- Donor matching criteria including HLA-matched sibling or related donors, mismatched related donors, or matched unrelated donors
- Donor weight at least 40 kilograms and age at least 12 years
- Donor able to undergo mobilized peripheral blood apheresis and in good health
- Donor organ function within specified laboratory limits
- Donor negative for infections including HIV, hepatitis, and others
- Negative pregnancy test for females of childbearing potential
- Voluntary written consent for donors (parent/guardian and minor assent if under 18)
You will not qualify if you...
- Pregnant or breastfeeding females
- Active uncontrolled infection within one week before starting treatment
- Malignant solid tumor cancer within two years prior to study
- Donors who are pregnant or positive for certain infections
- Donors not meeting organ function or health requirements
- Inability to provide voluntary written consent or minor assent if appropriate
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 2 weeks
Participants receive conditioning drugs and undergo T cell receptor alpha/beta depleted peripheral blood stem cell transplantation.
Multiple visits from day -6 through day 0 for conditioning and transplantation
Duration - Up to 1 year after transplant
Participants are monitored for graft versus host disease, engraftment, infections, and overall survival after transplantation.
Regular visits for assessments up to 1 year post-transplant
Trial Site Locations
Total: 1 location
1
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
Research Team
M
Margaret MacMillan, MD, Msc, FRCPC
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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