Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

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

Detailed Description
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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.

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

Conditions
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Craniopharyngioma, Child

Intervention / Treatment
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* Procedure: Tumor and Blood Specimens


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 0 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pediatrics with Tumors<br>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<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Identification of Potential Therapeutic Targets | 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 | 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 | Investigators will identify potential therapeutic targets immunohistochemistry. | Beginning of study to end of study, up to 1 year. | 
| Identification of Potential Therapeutic Targets | 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 | 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 | 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) | 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 | 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 | 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 | At 6 and 12 months after the specimen sample was taken. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pilot Study, Biological Targets, CTNNB1, Tumor