Actively Recruiting
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Led by Masonic Cancer Center, University of Minnesota · Updated on 2026-01-30
48
Participants Needed
1
Research Sites
529 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
CONDITIONS
Official 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 (FA) and age under 65 years
- Presence of severe aplastic anemia, myelodysplastic features, high risk genotype, or immunodeficiency with recurrent infections for FA patients
- Karnofsky performance status \u2265 70% if 16 years or older or Lansky play score \u2265 50% if under 16 years
- Adequate lung, heart, and liver function
- Voluntary written consent (and minor assent if appropriate) before study procedures
- Diagnosis of telomere biology disorder (TBD) and age under 70 years
- Presence of severe aplastic anemia or myelodysplastic features for TBD patients
- Donor matches including HLA-A, B, DRB1 matched sibling or related donor, mismatched related donor, or 7-8/8 HLA matched unrelated donor
- Donor age at least 12 years and weight at least 40 kilograms
- Donor willing and able to undergo mobilized peripheral blood apheresis
- Donor in general good health with adequate organ function (hematologic, liver, and kidney)
- Donor infectious disease screening negative for HIV, hepatitis B and C, and other infections
- Female donors of childbearing potential with negative pregnancy test prior to mobilization
- Voluntary written consent from donors before research procedures
You will not qualify if you...
- Pregnant or breastfeeding women; females of childbearing potential must have a negative pregnancy test within 14 days before study
- Active, uncontrolled infection within 1 week before starting study therapy
- Malignant solid tumor cancer within previous 2 years
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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