TCR Alpha/Beta and CD19-deplete Haplo-HSCT

University of Colorado Denver (CU Denver)

Status and phase: Withdrawn; Phase 2

Conditions: Hematologic Malignancy; Other Hematologic Condition; Pediatric Patients

Treatments: Device: CliniMACS Plus Instrument

Study type: Interventional

Funder types: Other

Identifiers

NCT05288595

20-2336.cc

Details and patient eligibility

About

This is an open label, interventional, non-randomized, phase II trial of TCR alpha/beta and CD19-depeleted allogeneic HCT in pediatric patients with hematologic disease.

Full description

This is a single-site, open label, interventional, non-randomized, phase II trial of TCRαβ/CD19 deplete allogeneic HCT as donor source and sole GVHD prophylaxis in pediatric patients with either malignant or non-malignant hematologic disease who are eligable for allogeneic HCT, but lack a HLA-matched sibling donor.

Sex: All

Ages: 31 days to 30 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. Age 31 days to <30 years
2. Have a malignant or non-malignant hematologic disease, defined as disease resulting from abnormal function of a cell of the hematopoietic stem cell lineage, that could benefit from an allogeneic HCT. Examples include acute and chronic leukemias, myelodysplastic syndrome, lymphoma, severe acquired and congenital cytopenias/marrow failure, white blood cell abnormalities, red blood cell abnormalities, and platelet abnormalities.
3. Clinical remission for patients with acute leukemia (MDS/AML excluded) or lymphoma
4. Lack a healthy and willing HLA-identical related donor, with the exception of patients with FA who will be eligible with a willing HLA-identical related donor given the standard use of T-cell depletion in matched sibling donor HCT in FA
5. Have a related or an unrelated donor who meets the donor selection criteria, is healthy, willing, and able to receive GCSF with or without Plerixafor, and undergo apheresis through placement of catheters in the antecubital veins or a temporary central venous catheter
6. Able to give informed consent if ≥ 18 years, or with legal guardian capable of giving informed consent if < 18 years
7. Provision of signed and dated informed consent form

Exclusion criteria

1. Uncontrolled, active infection at time of HCT
2. HIV positivity
3. Cardiac ejection fraction <45%
4. Creatinine clearance <60 mL/min/1.72 mL
5. Pulmonary diffusion capacity (adjusted for hemoglobin), FEV1, or FVC <60% of predicted or an O2 saturation <94% on room air if unable to perform pulmonary function testing
6. Serum ALT >5x upper limit of normal or bilirubin >2
7. Performance score (Lansky or Karnofsky) <50
8. Pregnant or lactating females, as many medications necessary for a successful HCT are potentially harmful to unborn babies and infants.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Pediatric patients with malignant or non-malignant hematologic condition

Experimental group

Description:

The infusion of the final TCRαβ/CD19 depleted product will be given through the recipient's central venous catheter and will be administered fresh, without cryopreservation whenever possible. If the product must be cryopreserved and then thawed, this will be done according to institutional standards.

Treatment:

Device: CliniMACS Plus Instrument