Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 12 of 101 for:    "Craniopharyngioma"

Tocilizumab in Children With ACP

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03970226
Recruitment Status : Recruiting
First Posted : May 31, 2019
Last Update Posted : July 4, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
University of Colorado, Denver

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, we will submit this study to COMIRB to review and approve opening the second phase of the study (feasibility phase). During this phase patients will be administered tocilizumab every two weeks for up to 26 cycles (approximately 2 years). Patients will be followed for up to 5 years in the feasibility phase.

Condition or disease Intervention/treatment Phase
Adamantinomatous Craniopharyngioma Drug: Tocilizumab Early Phase 1

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 27 participants
Intervention Model: Sequential Assignment
Intervention Model Description: Phase 0 followed by Feasibility Phase
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 0/Feasibility Trial of Tocilizumab in Children and Adolescents With Newly‐ Diagnosed or Recurrent/Progressive Adamantinomatous Craniopharyngioma
Actual Study Start Date : June 27, 2019
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Tocilizumab

Arm Intervention/treatment
Experimental: Tocilizumab Administration: Phase 0 and Feasibility Phase

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

During the Feasibility Phase, patients will receive tocilizumab every 2 weeks for up to 26 cycles (approximately 2 years). Patients will be followed for up to 5 years.

Drug: Tocilizumab

Phase 0: One dose of tocilizumab prior to surgery

Feasibility phase: Tocilizumab administered every 2 weeks for up to 26 cycles (approximately 2 years).





Primary Outcome Measures :
  1. Phase 0: Presence of Tocilizumab and Metabolites [ Time Frame: Within 4 to 8 hours of administration of tocilizumab ]
    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.

  2. Feasibility Phase: Overall Response Rate (ORR) [ Time Frame: Start of study to end of study, or up to 5 years ]
    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.

  3. Feasibility Phase: Partial Response Rate (PR) [ Time Frame: Start of study to end of study, or up to 5 years ]
    Utilize radiography to estimate the partial response rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.

  4. Feasibility Phase: Complete Response Rate (CR) [ Time Frame: Start of study to end of study, or up to 5 years ]
    Utilize radiography to estimate the complete response rate of subjects with newly diagnosed, unresectable or recurrent/progressive ACP (with or without prior radiation therapy) treated with systemic tocilizumab.

  5. Feasibility Phase: 1-Year Disease Stabilization [ Time Frame: Start of study to 1 year post treatment ]
    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.


Secondary Outcome Measures :
  1. Phase 0: IL6 and Inflammatory Cytokines [ Time Frame: Within 4 to 8 hours of administration of tocilizumab ]
    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

  2. Feasibility Phase: Progression Free Survival (PFS) [ Time Frame: 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 ]
    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.

  3. Feasibility Phase: Toxicity Profile [ Time Frame: Start of study to end of study, up to 5 years ]
    To define toxicities of tocilizumab therapy using CTCAE version 5.

  4. Feasibility Phase: Pathway Activation [ Time Frame: Start of study to end of study, up to 5 years ]
    To demonstrate evidence of WNT (Wingless-related integration site) in tumor tissue using immunohistochemistry and transcription array

  5. Feasibility Phase: Pathway Activation [ Time Frame: Start of study to end of study, up to 5 years ]
    To demonstrate evidence of MAPK (mitogen activated protein kinases) in tumor tissue using immunohistochemistry and transcription arr

  6. Feasibility Phase: Immunity [ Time Frame: Start of study to end of study, up to 5 years ]
    To demonstrate immune cell infiltration in tumor tissue using immunohistochemistry and flow cytometry

  7. Feasibility Phase: Cytokines [ Time Frame: Start of study to end of study, up to 5 years ]
    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)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

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:

    1. Presumed craniopharyngioma based on imaging features and best judgement of treating medical team (if newly diagnosed)
    2. Previous histologically confirmed ACP that has progressed or recurred at the time of enrollment

Feasibility Eligibility:

  • Must meet one of the following criteria:

    1. Recurrent or progressive* ACP treated with surgery alone without radiation
    2. 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 reaccumulation of the cyst following one or more cyst aspirations.
  • Newly diagnosed, histologically confirmed ACP with unresectable residual 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

    a. Adequate bone marrow function defined as:

  • Platelet count ≥100,000/μl (transfusion independent)
  • Absolute neutrophil count (ANC) ≥2,000/μl b. 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 c. Adequate liver function defined as:
  • SGOT (AST) and SGPT (ALT) <1.5x ULN for age
  • Subjects must meet one of the following performance scores:

    1. ECOG performance status scores of 0, 1, or 2;
    2. Karnofsky score of ≥60 for patients > 16 years of age; or
    3. 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.

Exclusion Criteria

  • Pregnant or breastfeeding
  • Uncontrolled intercurrent illness including, but not limited to:

    1. ongoing or active infection (including active tuberculosis)
    2. symptomatic congestive heart failure
    3. unstable angina pectoris
    4. cardiac arrhythmia
    5. 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03970226


Contacts
Layout table for location contacts
Contact: Jessica Channell 720-777-8847 jessica.channell@childrenscolorado.org
Contact: Elizabeth Chick 720-777-3214 elizabeth.chick@childrenscolorado.org

Locations
Layout table for location information
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Kathleen Dorris, MD       kathleen.dorris@childrenscolorado.org   
Sponsors and Collaborators
University of Colorado, Denver
National Cancer Institute (NCI)
Investigators
Layout table for investigator information
Principal Investigator: Lindsey Hoffman, DO University of Colorado, Denver

Layout table for additonal information
Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT03970226     History of Changes
Other Study ID Numbers: 18-2143.cc
1R03CA235200-01 ( U.S. NIH Grant/Contract )
P30CA046934 ( U.S. NIH Grant/Contract )
First Posted: May 31, 2019    Key Record Dates
Last Update Posted: July 4, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Colorado, Denver:
Phase 0
Feasibility
Tocilizumab
Additional relevant MeSH terms:
Layout table for MeSH terms
Craniopharyngioma
Adamantinoma
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Nerve Tissue
Bone Neoplasms
Neoplasms by Site
Bone Diseases
Musculoskeletal Diseases