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NCT01729091

Umbilical Cord Blood-Derived Natural Killer Cells, Elotuzumab, Lenalidomide, and High Dose Melphalan, Followed by Stem Cell Transplant in Treating Patients With Multiple Myeloma

Completed Phase 2 Results posted Last updated 9 May 2025
What this trial tests

Phase 2 trial testing Autologous Hematopoietic Stem Cell Transplantation in Plasma Cell Leukemia in 72 participants. Completed in 25 June 2024.

Timeline
10 June 2013
Primary endpoint
25 June 2024
25 June 2024

Quick facts

Lead sponsorM.D. Anderson Cancer Center
PhasePhase 2
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment72
Start date10 June 2013
Primary completion25 June 2024
Estimated completion25 June 2024
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

M.D. Anderson Cancer Center — full company profile →

Who can join

Adults 18 to 75, any sex, with Plasma Cell Leukemia or Plasma Cell Myeloma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Dose Limiting Toxicities Primary · Within 30 days post-transplant

Dose limiting toxicity is defined as number of participants experienced: * grade 4 NK infusion related toxicity, * failure to engraft by D+28 or delayed engraftment, * grades 3-5 allergic reactions related to study cell infusion, * grade 3-5 organ toxicity (cardiac, dermatologic, gastrointestinal, hepatic, pulmonary, renal/genitourinary, or neurologic) not pre-existing or due to the underlying malignancy or due to preparative chemotherapy and occurring within 30 days (+3 days if necessary) post-transplant, * grades 3-4 acute GVHD occurring within 45 days post-transplant, * treatment-related d

grade 4 NK infusion related toxicity
GroupValue95% CI
P1_C1_5x10^60
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^80
P2_1x10^8+Elotuzumab0
failure to engraft by D+28 or delayed engraftment
GroupValue95% CI
P1_C1_5x10^60
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^81
P2_1x10^8+Elotuzumab0
· grades 3-5 allergic reactions related to study cell infusion
GroupValue95% CI
P1_C1_5x10^60
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^80
P2_1x10^8+Elotuzumab0
grade 3-5 organ toxicity
GroupValue95% CI
P1_C1_5x10^61
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^813
P2_1x10^8+Elotuzumab11
grades 3-4 acute GVHD occurring within 45 days post-transplant
GroupValue95% CI
P1_C1_5x10^60
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^80
P2_1x10^8+Elotuzumab0
treatment-related death within 8 weeks of the study cell infusion
GroupValue95% CI
P1_C1_5x10^60
P1_C2_1x10^70
P1_C3_5x10^70
P1_C4_1X10^80
P2_1x10^8+Elotuzumab0
Number of Participants That Achieved Very Good Response (VGPR) + Complete Response (CR) Primary · At 3 months post-transplant

Complete response (CR) (all of the following): 1. Negative immunofixation in serum and urine. 2. \< 5% plasma cells in the bone marrow. 3. Disappearance of any soft tissue plasmacytomas. Very good partial response (VGPR) (one of the following): 1. Serum and urine M protein detectable by immunofixation but not by electrophoresis. 2. 90% or greater reduction in serum M protein plus urine M protein level \<100 mg per 4h.

VGPR
GroupValue95% CI
P1_C1_5x10^61
P1_C2_1x10^70
P1_C3_5x10^71
P1_C4_1X10^84
P2_1x10^8+Elotuzumab5
CR
GroupValue95% CI
P1_C1_5x10^61
P1_C2_1x10^73
P1_C3_5x10^72
P1_C4_1X10^817
P2_1x10^8+Elotuzumab22
Progression-free Survival (PFS) Secondary · Up to 12 months

Number of participants that are alive and disease free one year post transplant

GroupValue95% CI
P1_C1_5x10^62
P1_C2_1x10^72
P1_C3_5x10^74
P1_C4_1X10^828
P2_1x10^8+Elotuzumab28

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 12 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

P1_C1_5x10^6
Serious: 0/3 (0%)
Deaths: 2/3
P1_C2_1x10^7
Serious: 0/3 (0%)
Deaths: 2/3
P1_C3_5x10^7
Serious: 0/4 (0%)
Deaths: 2/4
P1_C4_1X10^8
Serious: 1/32 (3%)
Deaths: 11/32
P2_1x10^8+Elotuzumab
Serious: 1/30 (3%)
Deaths: 3/30

Serious adverse events (3 terms)

ReactionSystemP1_C1_5x10^6P1_C2_1x10^7P1_C3_5x10^7P1_C4_1X10^8P2_1x10^8+Elotuzumab
Low plateletInvestigations
Primary graft failureBlood and lymphatic system disorders
ViralInfections and infestations
Other adverse events (38 terms — click to expand)

ReactionSystemP1_C1_5x10^6P1_C2_1x10^7P1_C3_5x10^7P1_C4_1X10^8P2_1x10^8+Elotuzumab
NauseaGastrointestinal disorders
DiarrheaGastrointestinal disorders
Oral mucositisGastrointestinal disorders
Febrile neutropeniaBlood and lymphatic system disorders
Fluid overloadGeneral disorders
BacterialInfections and infestations
FeverGeneral disorders
ALT increasedInvestigations
AST increasedInvestigations
RashSkin and subcutaneous tissue disorders
ALK increasedInvestigations
Creatinine increasedInvestigations
HemorrhoidsGastrointestinal disorders
T bilirubin increasedInvestigations
ViralInfections and infestations
Engraftment syndromeImmune system disorders
HeadacheNervous system disorders
HypotensionVascular disorders
PneumonitisRespiratory, thoracic and mediastinal disorders
CoughRespiratory, thoracic and mediastinal disorders
DyspneaRespiratory, thoracic and mediastinal disorders
DysrhythmiaCardiac disorders
Gastrointestinal bleedingGastrointestinal disorders
HypertensionVascular disorders
Peripheral neuropathyNervous system disorders
Abdominal painGastrointestinal disorders
AnorexiaMetabolism and nutrition disorders
ConfusionPsychiatric disorders
Cystitis noninfectiveRenal and urinary disorders
Dry eyeEye disorders
Dry skinSkin and subcutaneous tissue disorders
DysgeusiaGastrointestinal disorders
Ejection fraction decreasedCardiac disorders
Generalized muscle weaknessMusculoskeletal and connective tissue disorders
HiccupsRespiratory, thoracic and mediastinal disorders
Pulmonary edemaRespiratory, thoracic and mediastinal disorders
TremorNervous system disorders
Thromboembolic eventVascular disorders

Most-reported serious reactions: Low platelet, Primary graft failure, Viral.

Data from ClinicalTrials.gov NCT01729091 adverse events section.

Sponsor's own description

This phase II trial studies the side effects and best dose of umbilical cord blood-derived natural killer cells when given together with elotuzumab, lenalidomide, and high dose melphalan before autologous stem cell transplant and to see how well they work in treating patients with multiple myeloma. Before transplant, stem cells are taken from patients and stored. Immunotherapy with monoclonal antibodies, such as elotuzumab, may induce changes in the body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as lenalidomide and melphalan, may work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving natural killer cells from donor umbilical cord blood before transplant may also kill myeloma cells that remain in the body after the last chemotherapy treatment. After treatment, stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. The Application of Natural Killer Cell Immunotherapy for the Treatment of Cancer.
    Rezvani K, Rouce RH. · · 2015 · cited 202× · PMID 26635792 · DOI 10.3389/fimmu.2015.00578
  2. Killers 2.0: NK cell therapies at the forefront of cancer control.
    Hodgins JJ, Khan ST, Park MM, Auer RC, et al · · 2019 · cited 178× · PMID 31478911 · DOI 10.1172/jci129338
  3. Phase I study of cord blood-derived natural killer cells combined with autologous stem cell transplantation in multiple myeloma.
    Shah N, Li L, McCarty J, Kaur I, et al · · 2017 · cited 171× · PMID 28295190 · DOI 10.1111/bjh.14570
  4. Natural Killer Cells as Allogeneic Effectors in Adoptive Cancer Immunotherapy.
    Lupo KB, Matosevic S. · · 2019 · cited 146× · PMID 31163679 · DOI 10.3390/cancers11060769
  5. The Rise of Allogeneic Natural Killer Cells As a Platform for Cancer Immunotherapy: Recent Innovations and Future Developments.
    Veluchamy JP, Kok N, van der Vliet HJ, Verheul HMW, et al · · 2017 · cited 146× · PMID 28620386 · DOI 10.3389/fimmu.2017.00631
  6. Natural killer cells: a promising immunotherapy for cancer.
    Chu J, Gao F, Yan M, Zhao S, et al · · 2022 · cited 140× · PMID 35606854 · DOI 10.1186/s12967-022-03437-0
  7. Utilizing chimeric antigen receptors to direct natural killer cell activity.
    Hermanson DL, Kaufman DS. · · 2015 · cited 130× · PMID 25972867 · DOI 10.3389/fimmu.2015.00195
  8. Natural killer cells in clinical development as non-engineered, engineered, and combination therapies.
    Lamers-Kok N, Panella D, Georgoudaki AM, Liu H, et al · · 2022 · cited 125× · PMID 36348457 · DOI 10.1186/s13045-022-01382-5

Verify or expand the search:

Other trials of Autologous Hematopoietic Stem Cell Transplantation

Trials testing the same drug.

Other recruiting trials for Plasma Cell Leukemia

Currently open trials in the same condition.

Other M.D. Anderson Cancer Center trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01729091.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing