Haploidentical Donor Hematopoietic Progenitor Cell and NK Cell Transplantation for Hematologic Malignancy

• Status: Completed
• Trial end date: 2021-09-27
• Target enrollment: 0
• Participant gender: All

Summary

In this study, participants with high-risk hematologic malignancies undergoing hematopoietic cell transplantation (HCT), who do not have a suitable human leukocyte antigen (HLA) matched related/sibling donor (MSD) or matched unrelated donor (MUD) identified, will receive a haploidentical donor HCT with additional natural killer (NK) cells.

The investigators anticipate enrollment of 75 donors and 75 recipients.

PRIMARY OBJECTIVE:

• To estimate the rate of successful engraftment at day +42 post-transplant in patients who receive haploidentical donor stem cell plus NK cell transplantation with TLI based conditioning regimen for high risk hematologic malignancy.

SECONDARY OBJECTIVES:

• Estimate the incidence of malignant relapse, event-free survival, and overall survival at one-year post-transplantation.

• Estimate incidence and severity of acute and chronic (GVHD).

• Estimate the rate of transplant related mortality (TRM) in the first 100 days after transplantation.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: St. Jude Children's Research Hospital
• Collaborator: Assisi Foundation
• Treatments: Cyclophosphamide; Fludarabine; Fludarabine phosphate; Lenograstim; Maraviroc; Mechlorethamine; Melphalan; Mesna; Mycophenolate mofetil; Mycophenolic Acid; Sargramostim; Thiotepa

Criteria

Inclusion Criteria - Transplant Recipients:

• Age less than or equal to 21 years.

• Does not have a suitable MSD or volunteer MUD available in the necessary time for stem cell donation.

• Has a suitable single haplotype matched (≥ 3 of 6) and family member donor.

• High risk hematologic malignancy.

• If prior CNS leukemia, it must be treated and in CNS CR

• Does not have any other active malignancy other than the one for which this HCT is indicated.

• No prior allogeneic HCT, and no autologous HCT within the previous 12 months.

• Patient must fulfill pre-transplant evaluation

Inclusion Criteria - Haploidentical Donor:

• At least single haplotype matched (≥ 3 of 6) family member

• At least 18 years of age.

• HIV negative.

• Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).

• Not breast feeding.

• Regarding eligibility, is identified as either: (1) Completed the process of donor eligibility determination as outlined in 21 CFR 1271 and agency guidance; OR (2) Does not meet 21 CFR 1271 eligibility requirements, but has a declaration of urgent medical need completed by the principal investigator or physician sub-investigator per 21 CFR 1271.