Phase 2
N=54
Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive · Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome · Acute Myeloid Leukemia With Gene Mutations · Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive · de Novo Myelodysplastic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01904136 ↗Enrolled (actual)
54
Serious AEs
46.2%
Results posted
Jan 2024
Primary outcome: Primary: Number of Participants With Dose Limiting Toxicities (DLTs) — 0; 0; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Allogeneic Hematopoietic Stem Cell Transplantation (Procedure); Bone Marrow Transplantation (Procedure); Cyclophosphamide (Drug); Fludarabine (Drug); Laboratory Biomarker Analysis (Other); Melphalan (Drug); Mycophenolate Mofetil (Drug); Natural Killer Cell Therapy (Biological); Peripheral Blood Stem Cell Transplantation (Procedure); Tacrolimus (Drug); Total-Body Irradiation (Radiation)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- M.D. Anderson Cancer Center
- Primary completion
- Feb 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Dose Limiting Toxicities (DLTs) |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY 100-day Treatment Related Mortality |
1; 0; 0; 0; 0; 0 | — |
| SECONDARY Overall Survival |
1; 1; 1; 0; 0; 0 | — |
Summary
This phase I/II studies the side effects and best dose of natural killer cells before and after donor stem cell transplant and to see how well they work in treating patients with acute myeloid leukemia, myelodysplastic syndrome, or chronic myelogenous leukemia. Giving chemotherapy with or without total body irradiation before a donor peripheral blood stem cell or bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells and natural killer cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets.
Eligibility Criteria
Inclusion Criteria
- Eligibility for pediatric patients will be determined in conjunction with an MD Anderson Cancer Center (MDACC) pediatrician; patients age 2-17 years old may be enrolled after at least 4 adults (ages 18-65 years old) have been treated without toxicity
- Patient with no matched related donor who has a related haploidentical donor identified (= 3 abnormalities); (vi) treatment-related AML, or b. second or greater remission; patients beyond second remission have to be in complete remission (CR) at transplant to be eligible, or c. primary induction failure with partial response to therapy who achieve adequate cytoreduction
- Patients with myelodysplastic syndromes (MDS): a. de novo MDS with intermediate or high-risk International Prognostic Scoring System (IPSS) scores; patients with intermediate-1 features should have failed to respond to hypomethylating agent therapy, or b. patients with treatment-related MDS
- Chronic myeloid leukemia (CML): a. failed to achieve cytogenetic remission or have cytogenetic relapse after treatment with at least 2 tyrosine kinase inhibitors, or b. accelerated phase or blast phase at any time
- Performance score of at least 70% by Karnofsky or 0 to 1 by Eastern Cooperative Oncology Group (ECOG) (age >= 12 years), or Lansky Play-performance scale of at least 70% or greater (age = 92% on room air by pulse oximetry
- Patient or patient's legal representative, parent(s) or guardian should provide written informed consent; assent of a minor if participant's age is at least seven and less than eighteen years
Exclusion Criteria
- Human immunodeficiency virus (HIV) positive; active hepatitis B or C
- Uncontrolled infections; principal investigator (PI) is the final arbiter of this criterion
- Liver cirrhosis
- Central nervous system (CNS) involvement within 3 months
- Positive pregnancy test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization
- Inability to comply with medical therapy or follow-up
Data sourced from ClinicalTrials.gov (NCT01904136). 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.