Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

• Conditions: Craniopharyngioma, Child
• Interventions: Procedure: Tumor and Blood Specimens

• Registration Number: NCT03610906
• Lead Sponsor: University of Colorado, Denver

Brief Summary

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

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 Timeline

• Study First Submitted: 2018-07-17
• Study First Submitted QC: 2018-07-25
• Study First Posted: 2018-08-01
• Study Start: 2019-03-12
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-04
• Last Update Posted: 2025-03-07
• Primary Completion: 2030-02-02
• Study Completion: 2030-12

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 250

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Pediatrics with Tumors	Tumor and Blood Specimens	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).

Primary Outcome Measures

Name	Time	Method
Identification of Potential Therapeutic Targets	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 Outcome Measures

Name	Time	Method
Progression Free Survival (PFS)	Beginning of study up to age 21 or death, whichever comes first.	The amount of time the patient survives without advancement of disease.
Visual Deficit Assessment	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.
Pituitary Function Assessment	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
Survival	Beginning of study up to age 21 or death, whichever comes first.	The amount of time the patient survives with or without the disease.

Trial Locations

Locations (1)

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States