Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

February 26, 2024 updated by: University of Colorado, Denver

Advancing Treatment for Pediatric Craniopharyngioma: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

New data suggests that the current treatment for pediatric adamantinomatous craniopharyngioma (CPA) may not be as effective as it could be.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed Description

Current treatment regimens for pediatric CPA are limited to surgery and radiation therapy. This pilot study seeks to identify biologically rational therapeutics for the medical treatment of adamantinomatous CPA by confirming the overexpression of specific molecules.

Study Type

Observational

Enrollment (Estimated)

250

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The population to be enrolled in this study will be children ages 0 through 21 years of age with a high preoperative suspicion of craniopharyngioma. The study population will be selected from the clinics at the 10 POETIC member institutions, and selected additional institutions by the treating clinicians.

Description

Inclusion Criteria:

  1. Patients between 0-21 years of age.
  2. Patients with the diagnosis or clinical suspicion of craniopharyngioma in whom planned clinical management will include tissue sampling.

Exclusion Criteria:

  1. Patients in whom final pathology does not demonstrate adamantinomatous craniopharyngioma
  2. Patients in whom tissue specimen is not obtained/available
  3. Patients over 21 years of age.
  4. Patients who choose not to participate

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Pediatrics with Tumors
Pediatrics who have a tumor specimen that is suspected to be craniopharyngioma, but is deemed superfluous to the clinical care of the patient (e.g. pathological diagnosis).
Procedure: Tumor and Blood Specimens
If available, specimen will be sampled from within the tumor, and not include portions of the tumor capsule. If the tumor includes a cyst, fluid from the cyst will be sampled if available. For each tumor specimen, a companion sample of blood that would otherwise be disposed of through usual clinical practice in the operating room will also be collected.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Identification of Potential Therapeutic Targets
Time Frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through mRNA expression.
Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Time Frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through microarray analysis.
Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Time Frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets immunohistochemistry.
Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Time Frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through quantitative PCR.
Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Time Frame: Beginning of study to end of study, up to 1 year.
Investigators will also perform immunostaining for beta-catenin and use DNA SNaPshot analysis to determine what parts of the tumor are responsible for observed gene signatures.
Beginning of study to end of study, up to 1 year.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Survival
Time Frame: Beginning of study up to age 21 or death, whichever comes first.
The amount of time the patient survives with or without the disease.
Beginning of study up to age 21 or death, whichever comes first.
Progression Free Survival (PFS)
Time Frame: Beginning of study up to age 21 or death, whichever comes first.
The amount of time the patient survives without advancement of disease.
Beginning of study up to age 21 or death, whichever comes first.
Visual Deficit Assessment
Time Frame: At 6 and 12 months after the specimen sample was taken.
With regard to visual deficits, investigators will compare the rates of functional blindness, unilateral blindness, and visual field deficit.
At 6 and 12 months after the specimen sample was taken.
Pituitary Function Assessment
Time Frame: At 6 and 12 months after the specimen sample was taken.

With regard to pituitary function, investigators will assess patients based on the quality of life impairment that is associated with their dysfunction. Assessment will be divided among 4 groups:

  1. No dependence on hormone supplementation or evidence of diabetes insipidus
  2. Dependence on 1 or 2 hormone supplements without diabetes insipidus
  3. The presence of diabetes insipidus with or without the need for 2 or fewer hormone supplements
  4. Diabetes Insipidus with panhypopituitarism
At 6 and 12 months after the specimen sample was taken.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Colorado, Denver

Investigators

  • Principal Investigator: Todd Hankinson, MD, University of Colorado, Denver

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 12, 2019

Primary Completion (Estimated)

February 2, 2030

Study Completion (Estimated)

December 1, 2030

Study Registration Dates

First Submitted

July 17, 2018

First Submitted That Met QC Criteria

July 25, 2018

First Posted (Actual)

August 1, 2018

Study Record Updates

Last Update Posted (Actual)

February 28, 2024

Last Update Submitted That Met QC Criteria

February 26, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 14-0426.cc

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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