NCT04323046

Brief Summary

This phase I trial studies the side effects of nivolumab before and after surgery in treating children and young adults with high grade glioma that has come back (recurrent) or is increasing in scope or severity (progressive). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at P25-P50 for phase_1

Timeline
34mo left

Started Oct 2020

Longer than P75 for phase_1

Geographic Reach
2 countries

20 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress67%
Oct 2020Mar 2029

First Submitted

Initial submission to the registry

March 24, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 26, 2020

Completed
6 months until next milestone

Study Start

First participant enrolled

October 2, 2020

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2026

Completed
3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2029

Expected
Last Updated

May 4, 2025

Status Verified

May 1, 2025

Enrollment Period

5.4 years

First QC Date

March 24, 2020

Last Update Submit

May 2, 2025

Conditions

Outcome Measures

Primary Outcomes (2)

  • Percentage change in cell cycle-related genetic signature

    Will assess the percentage change in cell cycle-related genetic signature post administration of neoadjuvant treatments when compared to archived recurrent pediatric HGG group. The number of participants with high cell cycle gene signature (positive median gene set variation analysis (GSVA) score) will be tabulated. Variables involved in the primary analyses will be examined graphically and summarized by descriptive statistics.

    From screening to surgery visit (neoadjuvant treatment groups); at time of recurrent high grade glioma (HGG) tissue collection (for archived non-treated samples)

  • Proportion of participants with treatment-related adverse events

    Adverse events will be monitored throughout the trial and graded in severity according to the guidelines outlined in the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 from initiation of study treatment until 2 years after treatment discontinuation or until study treatment related adverse events resolve or return to baseline, Adverse experiences (specific terms as well as system organ class terms) and predefined limits of change in laboratory, and vital sign parameters that are not pre-specified as events of interest will be summarized with descriptive statistics (counts, percentage, mean, standard deviation, etc.).

    Up to 2 years

Secondary Outcomes (2)

  • Overall survival

    Up to 12 months

  • Progression-free survival (PFS)

    Up to 12 months

Study Arms (1)

Neoadjuvant nivolumab and adjuvant nivolumab

EXPERIMENTAL

NEOADJUVANT: Patients receive nivolumab IV over 30 minutes 14 days before undergoing standard of care surgical resection. ADJUVANT MAINTENANCE: After completion of neoadjuvant infusion, patients receive nivolumab IV over 30 minutes on days 1 and 15. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Biological: NivolumabOther: Quality-of-Life AssessmentOther: Questionnaire Administration

Interventions

NivolumabBIOLOGICAL

Given IV

Also known as: BMS-936558, MDX-1106, NIVO, ONO-4538, Opdivo
Neoadjuvant nivolumab and adjuvant nivolumab

Ancillary studies, given in person or online

Also known as: Quality of Life Assessment
Neoadjuvant nivolumab and adjuvant nivolumab

Ancillary studies, given in person or online

Neoadjuvant nivolumab and adjuvant nivolumab

Eligibility Criteria

Age6 Months - 25 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Participants with recurrent or progressive high-grade gliomas (HGG) (World Health Organization (WHO) grade III or grade IV) who are candidates for surgical tumor debulking will be enrolled in this trial
  • All assessments are to occur within 14 days of registration except where otherwise noted. The participant and their legal parent/guardian must be thoroughly informed about all aspects of the study, including the study visit schedule and required evaluations and all regulatory requirements for informed consent. The written informed consent must be obtained from the participant and legal parent/guardian prior to enrollment
  • Have a history of previously treated histologically confirmed World Health Organization grade III or IV HGG. Previous first line therapy with radiation and/or chemotherapy
  • Have evidence of recurrence or progression of disease by MRI scan
  • Participants must be adequate medical candidates for surgical resection. The intent of surgical resection is to allow both cytoreduction and tumor debulking as part of standard of care, and also collect a minimum of 100 mg of tumor tissue for the study tissue endpoints
  • A primary goal of surgery must be cytoreduction, and not solely on diagnostic biopsy
  • Age: Participants must be \> 6 months and \< 25 years of age at time of enrollment
  • Karnofsky \>= 50 for participants \> 16 years of age and Lansky \>= 50 for participants =\< 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
  • Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to enrollment (on stable or tapering dosing of steroids). A baseline detailed neurological exam should clearly document the neurologic status of the patient at the time of enrollment on the study.
  • Prior Therapy: Participants must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the defined eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately
  • Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive. At least 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
  • An interval of at least 12 weeks from the completion of radiation therapy to registration unless there is unequivocal histologic confirmation of tumor progression
  • Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events (AEs) are known to occur. The duration of this interval must be discussed with the study chair.
  • Had their last dose of biologic (anti-neoplastic agent) ≥7 days prior to study registration, or beyond the time during which AEs are known to occur.
  • Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count \[ANC\] counts): At least 7 days after the last dose of agent
  • +22 more criteria

You may not qualify if:

  • Current or planned participation in a study of an investigational agent or using an investigational device.
  • Has a diagnosis of immunodeficiency.
  • Has tumor primarily localized to the brainstem or spinal cord.
  • Has presence of diffuse leptomeningeal disease or or disseminated/multi-focal disease, or extracranial disease.
  • Has received systemic immunosuppressive treatments (such as methotrexate, chloroquine, azathioprine, etc.), aside from anti-neoplastic chemotherapy or systemic corticosteroids within six months of registration.
  • Participants with a concurrent condition requiring systemic treatment with either corticosteroids (\> 0.25 mg/kg daily prednisone equivalent) or other immunosuppressive medications within 14 days of start of study treatment. Inhaled or topical steroids, and adrenal replacement steroid doses \> 0.25 mg/kg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.
  • Unable to taper steroids due to ongoing mass effect; a maximum dexamethasone dose of 0.1 mg/kg/day is allowed (4mg maximum), but preferably have been discontinued (inhaled or topical use of steroids is allowed).
  • Has a known history of active TB (Bacillus tuberculosis).
  • Has a known additional malignancy that is progressing or requires active treatment within 3 years of registration. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy.
  • Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (e.g. thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
  • Has known history of, or any evidence of active non-infectious pneumonitis.
  • Has an active infection requiring systemic therapy.
  • Has a known hypersensitivity to any of the study therapy products.
  • Has a known history of positive test for human immunodeficiency virus (HIV) or known acquired immunodeficiency syndrome (AIDS).
  • NOTE: Testing for HIV must be performed at sites where mandated locally
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

University of Alabama at Birmingham, Children's of Alabama

Birmingham, Alabama, 35233, United States

RECRUITING

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Rady Children's Hospital

San Diego, California, 92123, United States

RECRUITING

University of California, San Francisco

San Francisco, California, 94115, United States

RECRUITING

Children's National Hospital

Washington D.C., District of Columbia, 20310, United States

RECRUITING

University of Florida

Gainesville, Florida, 32611, United States

RECRUITING

Riley Children's Hospital

Indianapolis, Indiana, 46202, United States

RECRUITING

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Washington University St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Hackensack Meridian Children's Health at Joseph M. Sanzari Children's Hospital

Hackensack, New Jersey, 07601, United States

RECRUITING

Duke Children's Hospital & Health Center

Durham, North Carolina, 27705, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84113, United States

RECRUITING

Sydney Children's Hospital

Sydney, New South Wales, 1291, Australia

RECRUITING

The Children's Hospital at Westmead

Westmead, New South Wales, 2152, Australia

RECRUITING

Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia

RECRUITING

Women's and Children's Hospital

North Adelaide, South Australia, 5006, Australia

RECRUITING

Royal Children's Hospital

Parkville, Victoria, 3052, Australia

RECRUITING

Perth Children's' Hospital

Perth, Western Australia, 6009, Australia

RECRUITING

MeSH Terms

Conditions

GlioblastomaGlioma

Interventions

Nivolumab

Condition Hierarchy (Ancestors)

AstrocytomaNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Tom Davidson (tdavidson@chla.usc.edu), MD

    Children's Hospital Los Angeles

    STUDY CHAIR

Central Study Contacts

Sabine Mueller, MD, PhD

CONTACT

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

March 24, 2020

First Posted

March 26, 2020

Study Start

October 2, 2020

Primary Completion

March 1, 2026

Study Completion (Estimated)

March 1, 2029

Last Updated

May 4, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations