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

Investigation of Biological Targets for Pediatric Craniopharyngioma Treatment

J
Jignesh Tailor

Primary Investigator

Enrolling By Invitation
30 days - 21 years
All
Phase N/A
5 participants needed
1 Location

Brief description of study

New data suggests that the current treatment for pediatric adamantinomatous craniopharyngioma (CPA) may not be as effective as it could be. 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.
 
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.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY - Consistent with most oncology trials, patients are not actively “recruited,” but are screened by their physician for appropriate clinical trial(s) at the time of their routine clinic visit. Occasionally, a patient may be a self-referral or physician referral, but are still screened for appropriate clinical trials at the time of their routine clinic visit. PI and staff may send copies of relevant consent forms to these patients to look over prior to actually consenting or enrolling them. This may take place at the patient's visit at which the consent is presented or the patient's next visit to the outpatient hematology/oncology clinic. 
 
Interested in participating? For more information about this research study or other cancer-related clinical trials at IU Simon Comprehensive Cancer Center, please contact:
IU Clinical Trials Office 
Phone: (317) 278-5632

Detailed description of study

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

PRIMARY OUTCOMES:

  • Investigators will identify potential therapeutic targets through mRNA expression.
  • Investigators will identify potential therapeutic targets through microarray analysis.
  • Investigators will identify potential therapeutic targets immunohistochemistry.
  • Investigators will identify potential therapeutic targets through quantitative PCR.
  • 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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Craniopharyngioma, Cancer, Riley
  • Age: 30 days - 21 years
  • Gender: All

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

This study investigates pediatric adamantinomatous craniopharyngioma, a type of brain tumor. Craniopharyngioma is a condition where a non-cancerous tumor forms near the pituitary gland, which is located at the base of the brain. This study aims to identify biological targets that could lead to new medical therapies, as current treatments are limited to surgery and radiation.

Participants in the study will undergo procedures such as mRNA expression analysis, microarray analysis, immunohistochemistry, quantitative PCR, and immunostaining. These procedures help researchers understand which parts of the tumor are responsible for certain gene signatures, potentially leading to better treatment options in the future.

  • Who can participate: Children and young adults aged 0 to 21 years with a diagnosis or suspicion of craniopharyngioma, who are scheduled for tissue sampling, can participate.
  • Study details: Participants will provide tissue samples for analysis. Researchers will perform various investigational tests to identify potential therapeutic targets. This study does not involve any investigational medication or placebo.
Updated on 23 Sep 2025. Study ID: SNEU-UC-ATPC, 26544
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only