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Adamantinomatous CraniopharyngiomaEarly Phase 1NCT03970226

Tocilizumab in Children With ACP

A Early Phase 1 study testing Tocilizumab in people with Adamantinomatous Craniopharyngioma, run by University of Colorado, Denver at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 21 years
Conditions the study is for
Adamantinomatous Craniopharyngioma
What is being tested?
Tocilizumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
1 siteUnited States
Study dates
16 Sep 2019to May 2027
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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:
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  • 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.
  • Overall Study Inclusion Criteria:
  • Age: ≥ 2 years and < 21 years
  • Subjects may have received prior tocilizumab or other IL6 or IL6R inhibitor
  • Organ Function Requirements
  • Adequate bone marrow function defined as:
  • Platelet count ≥100,000/μl (transfusion independent)
  • Absolute neutrophil count (ANC) ≥1500/μl
  • Adequate renal function defined as:
  • Creatinine clearance or radioisotope GFR ≥70 ml/min/1.73 m2 or
  • A serum creatinine based on age/gender as follows: (Age, Male, Female) 3 to < 6 years, 0.8, 0.8; 6 to < 10 years, 1, 1; 10 to < 13 years, 1.2, 1.2; 13 to < 16 years, 1.5, 1.4; 16 years to < 18 years, 1.7, 1.4
  • Adequate liver function defined as:
  • SGOT (AST) and SGPT (ALT) <1.5x ULN for age
  • Subjects must meet one of the following performance scores:
  • ECOG performance status scores of 0, 1, or 2;
  • Karnofsky score of ≥60 for patients > 16 years of age; or
  • Lansky score of ≥60 for patients ≤16 years of age
  • Subjects of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study.
  • Informed consent and assent obtained as appropriate.

Who cannot join

  • 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
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  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

Other trials in United States

About this study

Official title: A Phase 0/Feasibility Trial of Tocilizumab in Children and Adolescents With Newly- Diagnosed or Recurrent/Progressive Adamantinomatous Craniopharyngioma

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.

Being tested
Tocilizumab
Keywords
Phase 0, Feasibility, Tocilizumab
Medical subject headings
Craniopharyngioma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TocilizumabDrug

    Also called Actemra

    Phase 0: One dose of tocilizumab prior to surgery

    Feasibility phase: Tocilizumab administered every 2 weeks for up to 13 cycles (approximately 1 year).

Study groups in NCT03970226
Study groupRoleWhat they receive
Tocilizumab Administration: Phase 0ExperimentalIn Phase 0, patients will receive one dose of tocilizumab prior to surgery.
Receives: Drug: Tocilizumab
Tocilizumab Administration: Feasibility PhaseExperimentalDuring 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.
Receives: Drug: Tocilizumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Within 4 to 8 hours of administration of tocilizumab

  2. Feasibility Phase: Toxicity Profile

    To define toxicities of tocilizumab therapy using CTCAE version 5.

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

Secondary outcomes (8)

  1. Phase 0: IL6 and Inflammatory Cytokines

    To define levels of IL6 and other inflammatory cytokines in biopsied tissue and/or cyst fluid as measured by enzyme-linked immunosorbent assay (ELISA) following 1 dose of systemically administered tocilizumab

    Measured: Within 4 to 8 hours of administration of tocilizumab

  2. Feasibility Phase: Progression Free Survival (PFS)

    Utilize radiography to estimate PFS of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.

    Measured: 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

  3. Feasibility Phase: Pathway Activation

    To demonstrate evidence of WNT (Wingless-related integration site) in tumor tissue using immunohistochemistry and transcription array

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

  4. Feasibility Phase: Pathway Activation

    To demonstrate evidence of MAPK (mitogen activated protein kinases) in tumor tissue using immunohistochemistry and transcription arr

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

  5. Feasibility Phase: Immunity

    To demonstrate immune cell infiltration in tumor tissue using immunohistochemistry and flow cytometry

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

  6. Feasibility Phase: Cytokines

    To characterize cytokine signaling in tumor tissue and/or cyst fluid using enzyme-linked immunosorbent assay (ELISA)To characterize cytokine signaling in tumor tissue and/or cyst fluid using enzyme-linked immunosorbent assay (ELISA)

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

  7. Feasibility Phase: Overall Response Rate (ORR)

    Utilize radiography to estimate the overall response rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.

    Measured: Start of study to end of study, or up to 5 years

  8. Feasibility Phase: 1-Year Disease Stabilization

    Utilize radiography to estimate the 1-year disease stabilization rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.

    Measured: Start of study to 1 year post treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 May 2019
  2. Study start16 Sep 2019
  3. Primary completion26 May 2024
  4. Study completionMay 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Model description
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.
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03970226
Sponsor's protocol ID
18-2143.cc
Other IDs
R03CA235200
Status last verified
Feb 2026
First submitted
11 Apr 2019
Last updated
17 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Tocilizumab in people with Adamantinomatous Craniopharyngioma, run by University of Colorado, Denver at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 30 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Phase 0: Presence of Tocilizumab and Metabolites (time frame: Within 4 to 8 hours of administration of tocilizumab). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03970226.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2026.

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