Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

NCT03610906 | Enrolling By Invitation DMC

• 1 other identifier: 14-0426.cc
• Study Type: observational
• Target: 250
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Craniopharyngioma, Child

Keywords

Pilot Study; Biological Targets; CTNNB1; Tumor

Outcome Measures

Primary Outcomes (5)

• 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 Outcomes (4)

• Survival

  Beginning of study up to age 21 or death, whichever comes first.

• Progression Free Survival (PFS)

  Beginning of study up to age 21 or death, whichever comes first.

• Visual Deficit Assessment

  At 6 and 12 months after the specimen sample was taken.

• Pituitary Function Assessment

  At 6 and 12 months after the specimen sample was taken.

Study Arms (1)

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

Interventions

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.

Pediatrics with Tumors

Eligibility Criteria

• Age: 30 Days - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Colorado, Denver: lead

Study Sites (1)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Brain tissue and/or cyst fluid, blood

MeSH Terms

Conditions

Craniopharyngioma; Neoplasms

Interventions

Tumor Burden

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Body Weights and Measures; Anthropometry; Investigative Techniques

Study Officials

• Todd Hankinson, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 17, 2018
• First Posted: August 1, 2018
• Study Start: March 12, 2019
• Primary Completion (Estimated): February 2, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: March 23, 2026

Record last verified: 2026-03

Locations

US (1)