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Alpha/Beta TCD HCT for Inherited Bone Marrow Failure Disorders

This study is testing a new type of stem cell transplant called T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation for people with inherited bone marrow failure (BMF) disorders like Fanconi Anemia, Severe Aplastic Anemia, and Myelodysplastic Syndromes. The goal is to reduce the need for medicines that prevent graft-versus-host disease (GVHD), which is when the new cells attack your body. This could help your immune system recover faster and lower your risk of serious infections after the transplant. You would receive treatments like Total Body Irradiation (TBI), Cyclophosphamide (CY), Fludarabine (FLU), and Methylprednisolone (MP) before the stem cell transplant. The study is looking at how often severe GVHD happens by Day 100 after the transplant. This study is for individuals up to 65 years old.

Study design
This is a Phase II study involving up to 48 participants. It is testing a specific type of stem cell transplant.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study measures a key outcome (acute graft versus host disease) at Day 100 after the transplant.

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NCT03579875

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Recruiting
PHASE2Up to 65InterventionalTreatment
Masonic Cancer Center, University of Minnesota
~48 participants
Updated 2026-01-30 on ClinicalTrials.gov
What's tested:Total Body Irradiation (TBI) (Plan 1)Cyclophosphamide (CY) (Plan 1)Fludarabine (FLU)Methylprednisolone (MP)Donor mobilized PBSC infusionG-CSF

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Grade II-IV acute graft versus host disease (GVHD)
Measured over Day 100

Conditions

Fanconi Anemia
Severe Aplastic Anemia
Myelodysplastic Syndromes
T Cell Receptor Alpha/Beta Depletion
Telomere Biology Disorder
Bone Marrow Failure
Dyskeratosis Congenita
Telomere Biology Disorders

Explore related trials

Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic SyndromePancytopeniaSevere Aplastic AnemiaTelomere Biology Disorder

Where it's being run

1 sites across 1 states
Minnesota1

Study leadership

  • Margaret MacMillan, MD, Msc, FRCPC · PRINCIPAL_INVESTIGATOR · Masonic Cancer Center, University of Minnesota

Who to contact

Margaret MacMillan, MD, Msc, FRCPC
[email protected]
612-626-2961
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Diagnosis of Fanconi anemia
Age \<65 years of age
Has one of the following risk factors:
Severe aplastic anemia (SAA)
Myelodysplastic features
High risk genotype
Immunodeficiency associated with history of recurrent infections
Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \<16 years of age
Adequate pulmonary, cardiac and liver function
Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
Age \<70 years of age
Has one of the following risk factors:
Severe aplastic anemia (SAA)
Myelodysplastic features
Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score
Adequate pulmonary, cardiac and liver function
Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
an HLA-A, B, DRB1 matched sibling donor (matched sibling)
an HLA-A, B, DRB1 matched related donor (other than sibling)
a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen
7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.
Body weight of at least 40 kilograms and at least 12 years of age
Willing and able to undergo mobilized peripheral blood apheresis
In general good health as determined by the medical provider
Adequate organ function defined as:
Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)
Hepatic: ALT \< 2 x upper limit of normal
Renal: serum creatinine \< 1.8 mg/dl
Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1/2 Antibody, HTLVA 1/2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B
Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start
Voluntary written consent (parent/guardian and minor assent, if \< 18 years) prior to the performance of any research related procedure

Exclusion

Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
Active, uncontrolled infection within 1 week prior to starting study therapy
Malignant solid tumor cancer within previous 2 years

What this trial measures

  • Grade II-IV acute graft versus host disease (GVHD)Day 100

    incidence of grade II-IV acute graft versus host disease (GVHD)