Trial Outcomes & Findings for Haploidentical Natural Killer (NK) Cells in Patients With Relapsed or Refractory Neuroblastoma (NCT NCT00698009)

Results Overview

Neuroblastoma International Response Criteria: Complete Response (CR): No evidence of disease (primary and metastasis) clinically \& radiographic studies, (homovanillic acid (HVA)/vanillylmandelic acid (VMA) normal). Very Good Partial Response (VGPR): \>90% reduction in primary tumor, resolution all metastatic tumor except bone. No new bone lesions and improvement on scan of all pre-existing lesions; HVA/VMA decreased \>90%. Partial Response (PR): 50-90% reduction primary and all measurable metastatic lesions, 0-1 bone marrow samples with tumor; scans of bone lesions same as VGPR. HVA/VMA decreased 50-90%. Mixed Response (MR): \> 50% reduction any measurable disease (primary or metastases); no new lesions; \<25% increase in any existing lesion (exclude bone marrow evaluation). No Response (NR): No new lesions; \< 25% increase in existing lesion. Progressive Disease (PD): Any new lesions. Increase \<25% in measurable lesion, previous negative bone marrow positive for tumor.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

1 Year for overall patient response, or until disease progression

Results posted on

2020-09-24

Participant Flow

Recruitment period June 05, 2008 to December 03, 2010. All recruitment done at UT MD Anderson Cancer Center.

Participant milestones

Participant milestones
Measure	Fludarabine + Cyclophosphamide + NK Cell Infusion Fludarabine 25 mg/m\^2 intravenous (IV) Daily Over 30 minutes Starting 6 days before the NK cell infusion (considered Day -6) and once a day through Day -2. Cyclophosphamide 60 mg/kg IV Daily Over 2 Hours On Days -5 and -4. Natural Killer Cell Infusion on Day 0. Mesna 12 mg/kg By Vein, Over about 15 minutes, 5 Times Per Day on Days -5 and -4. Interleukin-2 subcutaneously three times weekly for 9 total doses following NK Cell Infusion.
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Fludarabine + Cyclophosphamide + NK Cell Infusion Fludarabine 25 mg/m\^2 intravenous (IV) Daily Over 30 minutes Starting 6 days before the NK cell infusion (considered Day -6) and once a day through Day -2. Cyclophosphamide 60 mg/kg IV Daily Over 2 Hours On Days -5 and -4. Natural Killer Cell Infusion on Day 0. Mesna 12 mg/kg By Vein, Over about 15 minutes, 5 Times Per Day on Days -5 and -4. Interleukin-2 subcutaneously three times weekly for 9 total doses following NK Cell Infusion.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Haploidentical Natural Killer (NK) Cells in Patients With Relapsed or Refractory Neuroblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fludarabine + Cyclophosphamide + NK Cell Infusion n=1 Participants Fludarabine 25 mg/m\^2 intravenous (IV) Daily Over 30 minutes Starting 6 days before the NK cell infusion (considered Day -6) and once a day through Day -2. Cyclophosphamide 60 mg/kg IV Daily Over 2 Hours On Days -5 and -4. Natural Killer Cell Infusion on Day 0. Mesna 12 mg/kg By Vein, Over about 15 minutes, 5 Times Per Day on Days -5 and -4. Interleukin-2 subcutaneously three times weekly for 9 total doses following NK Cell Infusion.
Age, Continuous	10 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Region of Enrollment United States	1 participants n=5 Participants

PRIMARY outcome

Timeframe: 1 Year for overall patient response, or until disease progression

Neuroblastoma International Response Criteria: Complete Response (CR): No evidence of disease (primary and metastasis) clinically \& radiographic studies, (homovanillic acid (HVA)/vanillylmandelic acid (VMA) normal). Very Good Partial Response (VGPR): \>90% reduction in primary tumor, resolution all metastatic tumor except bone. No new bone lesions and improvement on scan of all pre-existing lesions; HVA/VMA decreased \>90%. Partial Response (PR): 50-90% reduction primary and all measurable metastatic lesions, 0-1 bone marrow samples with tumor; scans of bone lesions same as VGPR. HVA/VMA decreased 50-90%. Mixed Response (MR): \> 50% reduction any measurable disease (primary or metastases); no new lesions; \<25% increase in any existing lesion (exclude bone marrow evaluation). No Response (NR): No new lesions; \< 25% increase in existing lesion. Progressive Disease (PD): Any new lesions. Increase \<25% in measurable lesion, previous negative bone marrow positive for tumor.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 21 days, up to 1 year

Feasibility of an infused allogeneic donor NK cell product and IL-2 following a cyclophosphamide and fludarabine preparative regimen to treat relapsed neuroblastoma after autologous peripheral blood stem cell (PBSC) transplant where feasibility is defined as being able to infuse NK cells on day 0.

Outcome measures

Outcome data not reported

Adverse Events

Fludarabine + Cyclophosphamide + NK Cell Infusion

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Fludarabine + Cyclophosphamide + NK Cell Infusion n=1 participants at risk Fludarabine 25 mg/m\^2 intravenous (IV) Daily Over 30 minutes Starting 6 days before the NK cell infusion (considered Day -6) and once a day through Day -2. Cyclophosphamide 60 mg/kg IV Daily Over 2 Hours On Days -5 and -4. Natural Killer Cell Infusion on Day 0. Mesna 12 mg/kg By Vein, Over about 15 minutes, 5 Times Per Day on Days -5 and -4. Interleukin-2 subcutaneously three times weekly for 9 total doses following NK Cell Infusion.
Immune system disorders Fever - Neutropenia	100.0% 1/1 • Number of events 1 • 4 months

Additional Information

Eugenie S Kleinerman,MD/Professor, Pediatrics - Research

UT MD Anderson Cancer Center

Phone: (713) 497-6226

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place