Haploidentical HCT for Severe Aplastic Anemia

PHASE2RecruitingINTERVENTIONAL
Enrollment

21

Participants

Timeline

Start Date

January 21, 2021

Primary Completion Date

July 1, 2029

Study Completion Date

July 1, 2030

Conditions
Aplastic AnemiaBone Marrow Failure Syndrome
Interventions
DRUG

Anti-Thymocyte Globulin (Rabbit)

Given intravenously (IV)

DRUG

Fludarabine

Given intravenously (IV)

DRUG

Cyclophosphamide

Given intravenously (IV)

DRUG

Mesna

Given intravenously (IV)

DRUG

G-CSF

Filgrastim is a human granulocyte colony-stimulating factor (G-CSF), produced by recombinant DNA technology. Dosage and Route of Administration: 5mcg/kg subcutaneous or intravenous daily until ANC \>2000 for 2 consecutive days, or as clinically indicated

RADIATION

Total Lymphoid Irradiation (TLI)

TLI will be given at 800 cGy total dose in 4 fractions.

DEVICE

CliniMACS

The mechanism of action of the CliniMACS Cell Selection System is based on magnetic-activated cell sorting (MACS). The CliniMACS device is a powerful tool for the isolation of many cell types from heterogeneous cell mixtures, (e.g. apheresis products). These can then be separated in a magnetic field using an immunomagnetic label specific for the cell type of interest, such as CD3+ human T cells.

BIOLOGICAL

HPC, A Infusion

Given intravenously Day 0-HPC, A Infusion (TCR αβ+/CD19+-depleted graft)

BIOLOGICAL

CD45RA-depleted DLI

CD45RA-depleted DLI will be given at least ONE week after engraftment

Trial Locations (1)

38105

RECRUITING

St. Jude Children's Research Hospital, Memphis

All Listed Sponsors
lead

St. Jude Children's Research Hospital

OTHER