Tocilizumab in Children With ACP

NCT03970226 | Active Not Recruiting Early Phase 1 DMC FDA Drug

• 2 other identifiers: 18-2143.ccR03CA235200
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

This study will be conducted in two phases. The first phase (phase 0) will be looking at patients with new or recurrent/ progressed craniopharyngioma tumors. These patients will be given one dose of tocilizumab before they have SOC surgery of their tumor. The objective of this phase is to see if drug reaches the tumor. If phase 0 is favorable and shows that drug is penetrating the tumor, the second phase of the study (feasibility phase) will open. Both phases will remain open concurrently and patients will be able to enroll on the Phase 0 then "roll over" and enroll on the feasibility phase. During the feasibility phase patients will be administered tocilizumab every two weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years in the feasibility phase.

Conditions

Adamantinomatous Craniopharyngioma

Keywords

Phase 0; Feasibility; Tocilizumab

Outcome Measures

Primary Outcomes (2)

• Phase 0: Presence of Tocilizumab and Metabolites

  Utilize biopsy and/or drainage to identify the presence of tocilizumab and its metabolites in adamantinomatous craniopharyngioma (ACP) tumor tissue and/or cyst fluid and/or CSF following one dose of systemically administered tocilizumab.

  Within 4 to 8 hours of administration of tocilizumab

• Feasibility Phase: Toxicity Profile

  To define toxicities of tocilizumab therapy using CTCAE version 5.

  Start of study to end of study, up to 5 years

Secondary Outcomes (8)

• Phase 0: IL6 and Inflammatory Cytokines

  Within 4 to 8 hours of administration of tocilizumab

• Feasibility Phase: Progression Free Survival (PFS)

  From date of enrollment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60 months

• Feasibility Phase: Pathway Activation

  Start of study to end of study, up to 5 years

• Feasibility Phase: Pathway Activation

  Start of study to end of study, up to 5 years

• Feasibility Phase: Immunity

  Start of study to end of study, up to 5 years

Study Arms (2)

Tocilizumab Administration: Phase 0

EXPERIMENTAL

In Phase 0, patients will receive one dose of tocilizumab prior to surgery.

Drug: Tocilizumab

Tocilizumab Administration: Feasibility Phase

EXPERIMENTAL

During the Feasibility Phase, patients will receive tocilizumab every 2 weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years.

Drug: Tocilizumab

Interventions

Tocilizumab DRUG

Phase 0: One dose of tocilizumab prior to surgery Feasibility phase: Tocilizumab administered every 2 weeks for up to 13 cycles (approximately 1 year).

Also known as: Actemra

Tocilizumab Administration: Feasibility Phase; Tocilizumab Administration: Phase 0

Eligibility Criteria

• Age: 2 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Phase 0 Eligibility:
• Tumor biopsy/resection and/or cyst aspiration planned for the clinical care of the patient independent of study participation by the treating pediatric neurosurgeon and neuro-oncologist
• Must meet one of the following criteria:
• Presumed craniopharyngioma based on imaging features and best judgement of treating medical team (if newly diagnosed)
• Previous histologically confirmed ACP that has progressed or recurred at the time of enrollment
• Feasibility Eligibility:
• Must meet one of the following criteria:
• Recurrent or progressive\* ACP treated with surgery alone without radiation
• Recurrent or progressive\* ACP treated with surgery and radiation
• \* Progressive disease for eligibility purposes will be defined as follows: Solid disease: any growth deemed progression based on discretion of the investigator regardless of timing from RT Cystic disease: must be at least 6 months from last day of RT. Patients demonstrating isolated cyst growth \>6 months after RT must show a continued increase in the cystic component on two serial MRI scans performed at least 4 weeks apart OR at least partial reaccumulation of the cyst following one or more cyst aspirations.
• Newly diagnosed, by histology or imaging ACP with unresectable residual cystic and/or solid disease that is measurable in 2 dimensions
• Subjects who participated in the Phase 0 portion and meet eligibility, may enroll in the Feasibility Phase of the study once open.
• Age: ≥ 2 years and \< 21 years
• Subjects may have received prior tocilizumab or other IL6 or IL6R inhibitor
• Organ Function Requirements

You may not qualify if:

• Pregnant or breastfeeding
• Uncontrolled intercurrent illness including, but not limited to:
• ongoing or active infection (including active tuberculosis)
• symptomatic congestive heart failure
• unstable angina pectoris
• cardiac arrhythmia
• psychiatric illness/social situations that would limit compliance with study requirements are not eligible.
• Known hypersensitivity or history of anaphylaxis to tocilizumab
• Received any live vaccinations within 3 months prior to start of therapy
• Evidence of metastatic disease or other cancer
• Inability to return for follow up visits or obtain required follow-up studies to assess toxicity of therapy

Sponsors & Collaborators

• University of Colorado, Denver: lead
• National Cancer Institute (NCI): collaborator
• National Institutes of Health (NIH): collaborator

Study Sites (1)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Craniopharyngioma

Interventions

tocilizumab

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Study Officials

• Margaret Macy, MD

  Children's Hospital Colorado

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Phase 0 to open first and if outcomes are favorable showing drug penetration then the Feasibility Phase will open. Both arms will be open concurrently.

Study Record Dates

• First Submitted: April 11, 2019
• First Posted: May 31, 2019
• Study Start: September 16, 2019
• Primary Completion: May 26, 2024
• Study Completion (Estimated): May 1, 2027
• Last Updated: February 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)