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Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

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ClinicalTrials.gov Identifier: NCT03610906
Recruitment Status : Recruiting
First Posted : August 1, 2018
Last Update Posted : August 1, 2018
Sponsor:
Information provided by (Responsible Party):
University of Colorado, Denver

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

Condition or disease Intervention/treatment
Craniopharyngioma, Child Procedure: Tumor and Blood Specimens

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.

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Advancing Treatment for Pediatric Craniopharyngioma: Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
Actual Study Start Date : April 7, 2014
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Group/Cohort Intervention/treatment
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
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.




Primary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.


Secondary Outcome Measures :
  1. Survival [ Time Frame: Beginning of study up to age 21 or death, whichever comes first. ]
    The amount of time the patient survives with or without the disease.

  2. Progression Free Survival (PFS) [ Time Frame: Beginning of study up to age 21 or death, whichever comes first. ]
    The amount of time the patient survives without advancement of disease.

  3. Visual Deficit Assessment [ Time Frame: 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.

  4. Pituitary Function Assessment [ Time Frame: 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



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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03610906


Contacts
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Contact: Susan Staulcup susan.staulcup@ucdenver.edu
Contact: Todd Hankinson, MD todd.hankinson@ucdenver.edu

Locations
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United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Susan Staulcup       susan.staulcup@ucdenver.edu   
Principal Investigator: Todd Hankinson, MD         
Sponsors and Collaborators
University of Colorado, Denver
Investigators
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Principal Investigator: Todd Hankinson, MD University of Colorado, Denver

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Responsible Party: University of Colorado, Denver
ClinicalTrials.gov Identifier: NCT03610906     History of Changes
Other Study ID Numbers: 14-0426.cc
First Posted: August 1, 2018    Key Record Dates
Last Update Posted: August 1, 2018
Last Verified: July 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Colorado, Denver:
Pilot Study
Biological Targets
CTNNB1
Tumor
Additional relevant MeSH terms:
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Craniopharyngioma
Adamantinoma
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Nerve Tissue
Bone Neoplasms
Neoplasms by Site
Bone Diseases
Musculoskeletal Diseases