Clinical trial NCT01419067: A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma or Observation After Radical Resection
Official title: A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Craniopharyngioma
Keywords: Proton Therapy
Radical resection
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
First posted: 2011-08-17
Enrollment: 112
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this study is to determine the feasibility and safety of treating patients with a brain tumor known as craniopharyngioma with limited surgery and a 5mm clinical target volume margin in combination with proton therapy. Proton therapy will be indicated for patients with diagnosed craniopharyngioma who are not treated with radical surgery (gross-total resection). Irradiated patients will undergo a series of evaluations designed to evaluate the effects of proton therapy. Similar evaluations will be performed on patients treated with radical surgery. Proton therapy will include 30 treatment fractions administered 5 days per week. Weekly imaging will be a requirement to monitor for cyst expansion and target volume deformation.

Detailed description: The primary objectives of this study :

To estimate the progression-free and overall survival distributions for children and young adults with craniopharyngioma treated with limited surgery and proton therapy using a 5mm clinical target volume margin while monitoring for excessive central nervous system necrosis.

The Secondary Objectives of this study:

* To estimate the cumulative incidence of cystic intervention and the event-free survival distribution for children and young adults with craniopharyngioma treated with limited surgery and proton therapy using a 5mm clinical target volume margin; and compare the distributions of progression-free, event-free and overall survival with the distributions for the St. Jude Children's Research Hospital cohort of 93 patients on which the design of this trial is based.
* To estimate the distributions of progression-free survival and overall survival for children and young adults with craniopharyngioma treated only with primary surgical resection and to compare these distributions with the distributions observed for patients treated with limited surgery and proton therapy.

Eligibility criteria: Inclusion Criteria:

* Craniopharyngioma diagnosed by histology, cytology or neuroimaging.
* Patients ages 0-21 years at the time of diagnosis.

Exclusion Criteria

* Prior history of fractionated radiation therapy.
* Prior treatment with intracystic P-32, intracystic bleomycin or radiosurgery.
* Pregnant females are excluded. Radiation has teratogenic or abortifacient effects.

Primary outcomes:
measure: Estimate the progression-free and overall survival distributions for children and young adults with craniopharyngioma treated with limited surgery and proton therapy
timeFrame: 5 years
Secondary outcomes:
measure: Estimate the cumulative incidence of cystic intervention and the event-free survival distribution for children and young adults with craniopharyngioma treated with limited surgery and proton therapy
timeFrame: 5 years
measure: Estimate the distributions of progression-free survival and overall survival for children and young adults with craniopharyngioma treated only with primary surgical resection
timeFrame: 5 years
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: University of Florida Health Proton Therapy Institute
state: Florida
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Thomas E Merchant, DO, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wilson LJ, Pirlepesov F, Moskvin V, Li Z, Guo Y, Li Y, Merchant TE, Faught AM. Proton therapy delivery method affects dose-averaged linear energy transfer in patients. Phys Med Biol. 2021 Apr 1;66(7). doi: 10.1088/1361-6560/abe835.