What's the purpose of this trial?

This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) hematopoietic cell transplantation (HCT) transplantation in patients with Fanconi anemia (FA) 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.

This trial is currently open and accepting patients.

What will happen during the trial?

You may be able to join this trial if you:

The following criteria is a partial list of reasons why patients may be eligible to participate in this clinical trial. Further evaluation with a medical professional is required.

Patient Selection:

Inclusion Criteria:

* Diagnosis of Fanconi anemia
* Less than 65 years of age
* Karnofsky performance status of ≥ 70% or, for children \< 16 years of age, Lansky Play Score ≥ 50
* Presence of at least one of the following risk factors:

* Severe aplastic anemia (SAA) defined as: Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions:

* platelet count \<20 x 109/L
* absolute neutrophil count of \<5 x 108/L
* hemoglobin \<8 g/dL
* Myelodysplastic syndrome (MDS) or acute leukemia
* High risk genotype
* Adequate organ function defined as:

* Bilirubin, AST or ALT, ALP \<5 x normal, Cardiac: left ventricle ejection fraction (LEFV) ≥45% by ECHO
* Pulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
* Identification of a suitable donor for peripheral blood cells per match criteria found in Section 5.
* Females of childbearing potential and males with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
* Able to provide written voluntary consent prior to the performance of any research related tests or procedures with parental/guardian consent for minor (and assent as appropriate)

Exclusion Criteria:

* 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

Donor Selection (Inclusion Criteria): meets one of the following match criteria:

* 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

Additional Trial Information

Phase 2

Enrollment: 48 patients (estimated)

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University of Minnesota - Masonic Cancer Center

Minneapolis, MN

Open and Accepting
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