Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

March 18, 2026 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

Status

Indicates the current recruitment status or the expanded access status.
Enrolling by invitation

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of Colorado, Denver

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 12, 2019

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 2, 2030

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2030

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 17, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 25, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 1, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 18, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

product manufactured in and exported from the U.S.

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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