Interferon γ-Primed Mesenchymal Stromal Cells as Prophylaxis for Acute Graft v Host Disease

• Status: Recruiting
• Trial end date: 2023-08-01
• Target enrollment: 0
• Participant gender: All

Summary

The protocol is a phase I open label study evaluating the safety and feasibility of peri-transplant infusion of freshly expanded interferon gamma primed MSCs in adult and pediatric patients undergoing HCT for acute leukemia and myelodysplastic syndrome (MDS).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Edwin Horwitz
• Treatments: Interferon-gamma; Interferons

Criteria

Inclusion Criteria:

• All transplant patients who undergo HCT with a myeloablative (MA) or Fludarabine/Melphalan (RIC) conditioning regimen and a HLA A- B- C- DR-matched unrelated donor as treatment for hematologic malignancy or MDS.

• Age ≥ 1 year at the time that the informed consent document is signed.

• Patients with acute leukemia must be in complete remission (defined as an M1 marrow -<5% blasts- no evidence of extramedullary disease, and an absolute neutrophil count ≥1.0 x 109/L. Cases where the absolute neutrophil count (ANC) is < 1.0 x 109/L and rising will also be considered. The PI will need to approve such cases for enrollment complete remissions without platelet recovery (CRp) will be considered remissions.

• Planned GVHD prophylaxis with a calcineurin inhibitor and methotrexate per institutional standards.

• Subject or parent/guardian must sign an informed consent document, and if appropriate, children must sign an assent document.

Exclusion Criteria:

• Patients who are to receive a non-myeloablative conditioning regimen.

• Patients receiving another investigational drug for acute GVHD prevention during the conditioning regimen or a planned investigational drug for the first year after transplant (there are no restrictions on GVHD treatment).

• Any medical or psychological condition or situation deemed by the Investigators to put the patient at increased risk of complications or non-compliance.

• Patient with a secondary malignancy who would be otherwise eligible for study, but for whom remission from the primary disease cannot be conclusively confirmed or for whom the chance of relapse of the primary disease is significant.

• Pregnancy (positive serum b-HCG) or breastfeeding.

• Estimated glomerular filtration rate (GFR) of < 50 mL/min/1.73m2.

• Cardiac ejection fraction < 50 (using M-Mode if assessment is done by Echocardiogram)

• T bilirubin > 2 × upper limit of normal or alanine aminotransferase (ALT) > 4 × upper limit of normal or aspartate aminotransferase (AST) > 4 x upper limit of normal unresolved veno-occlusive disease

• Pulmonary disease with forced vital capacity (FVC), forced expiratory volume (FEV1) or diffusing capacity for carbon monoxide (DLCO) parameters <45% predicted (corrected for hemoglobin) or requiring supplemental oxygen. Children who are developmentally unable to perform pulmonary function testing will be assessed solely on their need for supplemental oxygen.

• Karnofsky performance score or Lansky Play-Performance Scale score <80

• Human leukocyte antigen (HLA) antibody screen positive for HLA antibodies specific against all available MSC products.