Clinical Trial: A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After

Brief Summary:

Craniopharyngioma is a rare brain tumor that affects both children and adults. It arises in a region of the brain near the pituitary gland, visual pathways, and central blood vessels. Patients often present with headache, loss of vision or delayed growth. In some instances they may present with imbalance of water and salts in the body.

The treatment for craniopharyngioma may be radical surgery or a combination of surgery and radiation therapy. In some instances surgery is not required. If the tumor cannot be completely removed, radiation therapy may be required. In this study we will use the most advanced form of proton therapy which is called intensity-modulated proton therapy. This is a newer form of radiation therapy which has a number of advantages over older forms of proton therapy and conventional radiation therapy using x-rays.

The main goal of this study is to learn if proton therapy will effectively treat patients with craniopharyngioma brain tumors and reduce side effects compared to more traditional forms of radiation therapy.

Detailed Summary:

This study will include participants who are diagnosed or presumed to have craniopharyngioma based on neuroimaging, intra-operative assessment, or the evaluation of cyst fluid or tissue after limited or radical surgery. When possible, participants will be enrolled on protocol prior to surgery.

PRIMARY OBJECTIVE:

• To estimate the distributions of progression-free and overall survival for children and young adults with craniopharyngioma treated with intensity-modulated proton therapy while monitoring for excessive central nervous system necrosis, clinically significant vasculopathy, and permanent neurological conditions or deficits.

SECONDARY OBJECTIVES:

• To estimate the cumulative incidence of cystic intervention and the event-free survival distribution for children and young adults with craniopharyngioma treated with intensity-modulated proton therapy; and to compare the distributions of progression-free, event-free and overall survival with the distributions for the St. Jude Children's Research Hospital (SJCRH) cohort of patients treated with photon therapy on which the design of this trial is based.
• To estimate the cumulative incidence of cystic intervention and the distributions of progression-free survival, overall survival and the event-free 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 proton therapy.

OTHER PRE-SPECIFIED OBJECTIVES:

• Progression-free survival (PFS) [ Time Frame: 3 years after initiation of proton therapy ]
  PFS is defined as the interval of time from the initiation of radiation therapy (RT) until the earliest of the events: death from any cause; and disease progression.
• Overall survival (OS) [ Time Frame: 3 years after initiation of proton therapy ]
  OS is defined as the interval of time from the initiation of RT until the death from any cause.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Compare incidence of first cystic intervention with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital [ Time Frame: 3 years after initiation of proton therapy ]
  The cumulative incidence estimates of first cystic intervention for cystic fluctuations after proton therapy. Competing events are death, disease progression and development of a new or secondary tumor.
• Compare event-free survival (EFS) with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital [ Time Frame: 3 years after initiation of proton therapy ]
  Kaplan-Meier estimates will be provided of EFS, which is measured from initiation of proton therapy until the earliest of PFS failure or secondary malignancy or new tumor, or surgical intervention for cystic fluctuation. Patients who have not experienced an event will be censored at their last date of contact.
• Compare survival distributions with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital [ Time Frame: 3 years after initiation of proton therapy ]
  Final analyses will also compare survival distributions using the log-rank test for patients treated with limited surgery and proton therapy on this protocol with the updated SJCRH cohort of patients treated with limited surgery and photon irradiation. The definition of survival will be defined in same way for both cohorts. This secondary analysis will be stratified by whether or not patients have a cerebrospinal fluid (CSF) shunt in place prior to irradiation and race (black versus white).
• Compare survival distributions between groups [ Time Frame: 3 years after initiation of proton therapy ]
  To estimate the survival distributions 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. A stratified log-rank test will be used to compare the distributions for children and young adults with craniopharyngioma treated only with primary surgical resection and the distributions observed for patients treated with limited surgery and proton therapy.

Original Secondary Outcome: Same as current

Information By: St. Jude Children's Research Hospital

Dates:
Date Received: June 1, 2016
Date Started: June 22, 2016
Date Completion: June 30, 2027
Last Updated: April 24, 2017
Last Verified: March 2017