Clinical trial NCT03610906: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
Official title: Advancing Treatment for Pediatric Craniopharyngioma: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Craniopharyngioma, Child
Keywords: Pilot Study
Biological Targets
CTNNB1
Tumor
Lead sponsor: University of Colorado, Denver
Sponsor class: OTHER
First posted: 2018-08-01
Enrollment: 250
Sex: ALL
Minimum age (years): 30
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Identification of Potential Therapeutic Targets
timeFrame: Beginning of study to end of study, up to 1 year.
measure: Identification of Potential Therapeutic Targets
timeFrame: Beginning of study to end of study, up to 1 year.
measure: Identification of Potential Therapeutic Targets
timeFrame: Beginning of study to end of study, up to 1 year.
measure: Identification of Potential Therapeutic Targets
timeFrame: Beginning of study to end of study, up to 1 year.
measure: Identification of Potential Therapeutic Targets
timeFrame: Beginning of study to end of study, up to 1 year.
Secondary outcomes:
measure: Survival
timeFrame: Beginning of study up to age 21 or death, whichever comes first.
measure: Progression Free Survival (PFS)
timeFrame: Beginning of study up to age 21 or death, whichever comes first.
measure: Visual Deficit Assessment
timeFrame: At 6 and 12 months after the specimen sample was taken.
measure: Pituitary Function Assessment
timeFrame: At 6 and 12 months after the specimen sample was taken.
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
Study officials:
affiliation: University of Colorado, Denver
name: Todd Hankinson, MD
role: PRINCIPAL_INVESTIGATOR
References: