Expanded Natural Killer Cell Infusion in Treating Younger Patients With Recurrent/Refractory Brain Tumors

NCT02271711 | Completed Phase 1 FDA Drug

• 3 other identifiers: 2013-0765NCI-2014-02677P30CA016672
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This phase I trial studies the side effects and best dose of expanded natural killer cells in treating younger patients with brain tumors that have come back or do not respond to treatment. Infusing a particular type of a patient's own white blood cells called natural killer cells that have been through a procedure to expand (increase) their numbers may work in treating patients with recurrent/refractory brain tumors.

Conditions

Recurrent Childhood Medulloblastoma; Recurrent Ependymoma; Recurrent Medulloblastoma

Outcome Measures

Primary Outcomes (1)

• Maximum tolerated dose (MTD) of natural killer (NK) cells

  Defined as the highest dose studied in which 6 patients have been treated at most 1 patient with dose limiting toxicities are observed. Toxicities will be summarized by tabulation in terms of type, grade and attribution for each dose level of each group of patients studied at the end of the trial.

  4 weeks

Secondary Outcomes (6)

• Activation status of NK cells

  Up to 30 days after the last infusion in course 3

• Persistence of NK cells

  Up to 30 days after the last infusion in course 3

• Function of NK cells

  Up to 30 days after the last infusion in course 3

• Response of medulloblastoma to NK cells

  Up to 30 days after the last infusion in course 3

• Feasibility of NK cell manufacturing

  Up to 12 weeks

Study Arms (1)

Treatment (autologous ex vivo-expanded NK cells)

EXPERIMENTAL

Patients receive autologous expanded NK cells IV into the ventricle over 3 minutes once weekly on weeks 1-3. Treatment repeats every 4 weeks for up to 3 courses in the absence of disease progression or unacceptable toxicity. Patients experiencing disease progression may continue treatment at the discretion of the treating physician if pseudo-progression or benefit of slowed progression is suspected.

Other: Laboratory Biomarker Analysis; Biological: Natural Killer Cell Therapy

Interventions

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (autologous ex vivo-expanded NK cells)

Natural Killer Cell Therapy BIOLOGICAL

Given autologous ex-vivo expanded NK cells IV

Treatment (autologous ex vivo-expanded NK cells)

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis: patients with recurrent/refractory medulloblastoma (MB), atypical teratoid (AT)/rhabdoid tumors (RT) or ependymoma involving the brain and/or spine at original diagnosis or relapse; they must have histological verification at diagnosis and/or relapse; patient must have presented with these tumors in the posterior fossa (PF) or relapsed in the PF
• Patient must have either measurable or evaluable tumor
• Presence of or determined by neurosurgery to be a candidate for an implanted catheter in the ventricles to receive NK cell infusion
• Life expectancy of at least 12 weeks in opinion of principal investigator (PI) and/or designee
• Lansky score of 50 or greater if =\<16 years of age or a Karnofsky score of 50 or greater if \> 16 years of age (NOTE: patients who are unable to walk because of paralysis, but who are in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score)
• Neurologic deficits must have been relatively stable for a minimum of 1 week prior to study enrollment
• Patients must have recovered from the acute toxic effects of all prior anticancer chemotherapy
• Patient must be 4 weeks off any palliative radiation or craniospinal radiation
• Absolute neutrophil count (ANC) of \>= 1000/uL
• Platelet count of \>= 30,000
• Hemoglobin of \>= 9.0 g/dl
• Patients with a seizure disorder may be enrolled if well-controlled and on non-enzyme inducing anticonvulsants
• Patient or patient's legal representative, parent(s), or guardian able to provide written informed consent

You may not qualify if:

• Enrolled in another treatment protocol
• Evidence of untreated infection
• Extra-cranial metastasis
• Chronic corticosteroid dependence (except replacement therapy)
• Extensive disease, disease location, and/or co-morbid condition that the PI or designee considers unsafe for surgical intervention of NK cell infusion
• Pregnant or lactating women

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Medulloblastoma; Ependymoma

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neuroectodermal Tumors, Primitive; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Soumen Khatua

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2014
• First Posted: October 22, 2014
• Study Start: March 17, 2015
• Primary Completion: August 28, 2020
• Study Completion: August 28, 2020
• Last Updated: September 2, 2020

Record last verified: 2020-08

Locations

US (1)