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Enrolling by Invitation

NCT Number: NCT03610906

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.

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Key information

Conditions

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

Age range

30 day–21 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

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

Treatment and study plan

Tumor and Blood Specimens

Procedure

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.

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

Secondary outcomes

  1. 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.

  2. 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.

  3. 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.

  4. 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:

    • No dependence on hormone supplementation or evidence of diabetes insipidus
    • Dependence on 1 or 2 hormone supplements without diabetes insipidus
    • The presence of diabetes insipidus with or without the need for 2 or fewer hormone supplements
    • Diabetes Insipidus with panhypopituitarism

Sponsors and collaborators

Lead sponsor

University of Colorado, Denver

Other

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2030
Study completion
2030
First posted
Aug 1, 2018
Registry last updated
Mar 23, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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