Craniopharyngioma, ChildNCT03610906
Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
An observational study of Craniopharyngioma, Child, run by University of Colorado, Denver at 1 site in United States.
Trial summary
New data suggests that the current treatment for pediatric adamantinomatous craniopharyngioma (CPA) may not be as effective as it could be. Read more about this study
- Who is it for?
- Ages 30 days to 21 years
- Conditions the study is for
- Craniopharyngioma, Child
- What is being tested?
- Tumor and Blood Specimens
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- How many people
- 250 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Mar 2019to Dec 2030
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 days to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Children's Hospital Colorado
Aurora, Colorado, 80045
About this study
Official title: Advancing Treatment for Pediatric Craniopharyngioma: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
New data suggests that the current treatment for pediatric adamantinomatous craniopharyngioma (CPA) may not be as effective as it could be.
Read the 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.
- Conditions
- Craniopharyngioma, Child
- Being tested
- Tumor and Blood Specimens
- Keywords
- Pilot Study, Biological Targets, CTNNB1, Tumor
- Medical subject headings
- Craniopharyngioma, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tumor and Blood SpecimensProcedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: Procedure: Tumor and Blood Specimens |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets through mRNA expression.
Measured: 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.
Measured: Beginning of study to end of study, up to 1 year.
Identification of Potential Therapeutic Targets
Investigators will identify potential therapeutic targets immunohistochemistry.
Measured: 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.
Measured: 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.
Measured: Beginning of study to end of study, up to 1 year.
Secondary outcomes (4)
Survival
The amount of time the patient survives with or without the disease.
Measured: Beginning of study up to age 21 or death, whichever comes first.
Progression Free Survival (PFS)
The amount of time the patient survives without advancement of disease.
Measured: Beginning of study up to age 21 or death, whichever comes first.
Visual Deficit Assessment
With regard to visual deficits, investigators will compare the rates of functional blindness, unilateral blindness, and visual field deficit.
Measured: At 6 and 12 months after the specimen sample was taken.
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
Measured: At 6 and 12 months after the specimen sample was taken.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2018
- Study start12 Mar 2019
- Primary completion2 Feb 2030 (estimated)
- Study completionDec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Craniopharyngioma, ChildStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Todd Hankinson, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03610906
- Sponsor's protocol ID
- 14-0426.cc
- Status last verified
- Mar 2026
- First submitted
- 17 Jul 2018
- Last updated
- 23 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Advancing Treatment for Pediatric Craniopharyngioma: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
- ClinicalTrials.gov ID
- NCT03610906
- Acronym / sponsor ID
- 14-0426.cc
- Conditions
- Craniopharyngioma, Child
- Keywords
- Pilot Study, Biological Targets, CTNNB1, Tumor
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- 12 Mar 2019 (actual)
- Primary completion
- 2 Feb 2030 (estimated)
- Study completion
- Dec 2030 (estimated)
- First posted
- 1 Aug 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pediatrics with Tumors | – | Procedure: Tumor and Blood SpecimensPediatrics 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). |
Who may qualify
- Ages
- 30 days to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Tumor and Blood SpecimensIf 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. | Procedure or surgery | Pediatrics with Tumors |
Primary outcome
Identification of Potential Therapeutic Targets
Time frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through mRNA expression.
Identification of Potential Therapeutic Targets
Time frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through microarray analysis.
Identification of Potential Therapeutic Targets
Time frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets immunohistochemistry.
Identification of Potential Therapeutic Targets
Time frame: Beginning of study to end of study, up to 1 year.
Investigators will identify potential therapeutic targets through quantitative PCR.
Identification of Potential Therapeutic Targets
Time frame: 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.
4 secondary outcomes
Survival
Time frame: Beginning of study up to age 21 or death, whichever comes first.
Progression Free Survival (PFS)
Time frame: Beginning of study up to age 21 or death, whichever comes first.
Visual Deficit Assessment
Time frame: At 6 and 12 months after the specimen sample was taken.
Pituitary Function Assessment
Time frame: At 6 and 12 months after the specimen sample was taken.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Todd Hankinson, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Craniopharyngioma, Child, run by University of Colorado, Denver at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identification of Potential Therapeutic Targets (time frame: Beginning of study to end of study, up to 1 year.). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2026.