Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

Craniopharyngioma, Child Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03610906
• Other study ID #: 14-0426.cc
• Status: Enrolling by invitation
• Start date: March 12, 2019
• Est. completion date: December 2030

Study information

• Verified date: February 2024
• Source: University of Colorado, Denver
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 250
• Est. completion date: December 2030
• Est. primary completion date: February 2, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Days to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Craniopharyngioma
• Craniopharyngioma, Child

Intervention

Procedure:
• Tumor and Blood Specimens
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.

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado

Sponsors (1)

Lead Sponsor	Collaborator
University of Colorado, Denver

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.
Primary	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.
Primary	Identification of Potential Therapeutic Targets	Investigators will identify potential therapeutic targets immunohistochemistry.	Beginning of study to end of study, up to 1 year.
Primary	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.
Primary	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	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.
Secondary	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.
Secondary	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.
Secondary	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.