Status:
ENROLLING_BY_INVITATION
Donor-Derived Ex-Vivo Expanded Natural Killer Cell Infusions in Children and Young Adults With High Risk Acute Myeloid Leukemia Receiving Myeloablative HLA-Haploidentical Hematopoietic Cell Transplant
Lead Sponsor:
Michael Pulsipher
Collaborating Sponsors:
Nationwide Children's Hospital
Seattle Children's Hospital
Conditions:
Acute Myeloid Leukemia
Eligibility:
All Genders
Up to 25 years
Phase:
PHASE2
Brief Summary
This is a Phase II pilot study to determine the efficacy of three fixed dose (1 x 108/kg) infusions of ex-vivo expanded human leukocyte antigen (HLA)-haploidentical donor natural killer (NK) cells (ha...
Eligibility Criteria
Inclusion
- Age ≤ 25 years at time of enrollment
- High-risk AML, as defined by one of the following:
- AML in CR1 (defined as \<5% blasts in BM by morphology and flow cytometry) having at least one of these high-risk features:
- Mutations associated with high risk disease (Appendix A). Other high-risk features not explicitly stated in Appendix A can be considered after discussion/approval with the protocol chair/team
- MRD-positive at the end of Induction I chemotherapy (defined as flow cytometry ≥ 0.1% blasts)
- AML in ≥CR2 (defined by \<5% blasts in BM by morphology and flow cytometry)
- Recovery from prior cycle of chemotherapy as defined by an absolute neutrophil count ≥ 500/mm3
- AML secondary to select germline marrow failure disorders (with exception of Fanconi Anemia) may be eligible but require approval from Protocol Chairs prior to enrollment.
- Performance status ≥70% (Lansky for \<16 years; Karnofsky for ≥16 years)
- Adequate major organ system function as demonstrated by:
- Renal: Creatinine clearance (CrCl) ≥60 mL/min/1.73m2 by Cockcroft-Gault formula, Schwartz formula, or nuclear GFR study (Table 3)
- Hepatic: Total bilirubin \<2 mg/dL (unless due to Gilbert syndrome) and ALT and AST \< 5x ULN
- Cardiac: LVEF at rest ≥50% or SF ≥27% (by MUGA or ECHO)
- Pulmonary: DLCO, FEV1, and FVC ≥ 50% of predicted corrected for hemoglobin. For patients \<7 years of age or those unable to perform PFTs: O2 Sat \>92% on room air by pulse oximetry and on no supplemental O2 at rest
- The patient, patient's parent, guardian, or legal representative can provide written informed consent
Exclusion
- Active extramedullary disease
- Unresolved/ongoing and serious viral, bacterial, or fungal infection despite appropriate treatment
- Positive pregnancy test in a female of child-bearing potential (FCBP)
- Inability to comply with medical therapy or follow-up
- Prior allogeneic transplant
- Patients with Fanconi Anemia and Down syndrome
Key Trial Info
Start Date :
August 24 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2028
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT04836390
Start Date
August 24 2021
End Date
May 1 2028
Last Update
May 4 2025
Active Locations (13)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
AdventHealth Orlando
Orlando, Florida, United States, 32803