Craniopharyngioma, Child
Conditions
Keywords
Pilot Study, Biological Targets, CTNNB1, Tumor
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.
Interventions
PROCEDURETumor 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.
Sponsors
University of Colorado, Denver
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
30 Days to 21 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Identification of Potential Therapeutic Targets | Beginning of study to end of study, up to 1 year. | Investigators will identify potential therapeutic targets through mRNA expression. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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 |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORTodd Hankinson, MD
University of Colorado, Denver
Outcome results
None listed