Phase 2
Completed N=82
Haploidentical Donor Hematopoietic Progenitor Cell and NK Cell Transplantation for Hematologic Malignancy
Source: ClinicalTrials.gov NCT01807611 ↗Enrolled (actual)
82
Serious AEs
71.8%
Results posted
Oct 2022
Primary outcomePrimary: Number of Transplant Recipients With Successful Engraftment — 70 Participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Transplant Recipients With Successful Engraftment |
70 | — |
| SECONDARY Number of Transplant Recipients With Malignant Relapse |
18 | — |
| SECONDARY Event-free Survival |
49 | — |
| SECONDARY Overall Survival |
59 | — |
| SECONDARY Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD) |
24; 16 | — |
| SECONDARY Number of Transplant Recipients With Transplant-related Mortality (TRM) |
4 | — |
| SECONDARY Severity of Acute Graft Versus Host Disease (aGVHD) |
48; 2; 2; 16; 4 | — |
| SECONDARY Severity of Chronic Graft Versus Host Disease (cGVHD) |
56; 6; 5; 5 | — |
Eligibility 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.
Data sourced from ClinicalTrials.gov (NCT01807611). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.