A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma or Observation After Radical Resection

NCT01419067 | Active Not Recruiting Phase 2 FDA Drug FDA Device

• 2 other identifiers: RT2CRNCI-2011-03707
• Study Type: interventional
• Target: 112
• Locations: 1 country
• Sites: 2

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.

Conditions

Craniopharyngioma

Keywords

Proton Therapy; Radical resection

Outcome Measures

Primary Outcomes (1)

• Estimate the progression-free and overall survival distributions for children and young adults with craniopharyngioma treated with limited surgery and proton therapy

  Reducing the clinical target volume margin to 5mm and using proton therapy, with the goal of reducing side effects from irradiation, will not increase the rate of tumor progression compared to photon therapy with a similar or larger clinical target volume margin.

  5 years

Secondary Outcomes (2)

• 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

  5 years

• Estimate the distributions of progression-free survival and overall survival for children and young adults with craniopharyngioma treated only with primary surgical resection

  5 years

Study Arms (1)

Craniopharyngioma Patients

OTHER

Craniopharyngioma patients will have 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). Patients who have had radical surgery or limited surgery prior to enrollment on this study and have no evidence of tumor will be observed for 5 years. Participants receive \^1\^8F-fluorodeoxyglucose and \^1\^1C-methionine will be given to aid in tumor visualization.

Procedure: Radical Surgery or Limited Surgery; Device: Proton Therapy; Drug: ^1^8F-fluorodeoxyglucose; Drug: ^1^1C-methionine

Interventions

Radical Surgery or Limited Surgery PROCEDURE

Patients will be selected for radical surgery based on the neurosurgeon's assessment that a Gross-Total Resection may be achieved with acceptable post-operative morbidity. The goal of surgical intervention in this study should be to facilitate tumor control, keeping surgical morbidity to a minimum. Common indications for surgical intervention directed at the tumor will include establishing a tissue diagnosis, tumor control by radical resection, relieving tumor mass effect to reduce symptoms and decreasing the target volume for proton therapy by planned volume reduction surgery. These indications can be achieved through radical or limited surgery.

Craniopharyngioma Patients

Proton Therapy DEVICE

Proton therapy will be started once a final treatment plan has been developed and approved. There is no time limit for the interval from enrollment to when proton therapy commences. The total prescribed dose will be 54CGE administered at 1.8CGE per fraction. The time course of administration will be one fraction per day, 5 days per week, for a period of 6 weeks. Radiation will be prescribed to the planning target volume which will include the tumor bed encompassed by an anatomically defined margin meant to include subclinical microscopic disease, and an additional geometric margin that is meant to account for the technical limitations associated with planning and administering daily fractionated treatment. Making radiation therapy safer through the use of proton therapy.

Also known as: Radiation

Craniopharyngioma Patients

^1^8F-fluorodeoxyglucose DRUG

This is a contrast media that will be given intravenously to aid in tumor visualization.

Also known as: ^1^8F-FDG, Contrast Media

Craniopharyngioma Patients

^1^1C-methionine DRUG

This is a contrast media that will be given intravenously to aid in tumor visualization.

Also known as: Contrast Media

Craniopharyngioma Patients

Eligibility Criteria

• Age: Up to 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (2)

University of Florida Health Proton Therapy Institute

Jacksonville, Florida, 32206, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Publications (2)

• Merchant TE, Hoehn ME, Khan RB, Sabin ND, Klimo P, Boop FA, Wu S, Li Y, Burghen EA, Jurbergs N, Sandler ES, Aldana PR, Indelicato DJ, Conklin HM. Proton therapy and limited surgery for paediatric and adolescent patients with craniopharyngioma (RT2CR): a single-arm, phase 2 study. Lancet Oncol. 2023 May;24(5):523-534. doi: 10.1016/S1470-2045(23)00146-8. Epub 2023 Apr 18.

  PMID: 37084748 DERIVED

• 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.

  PMID: 33607632 DERIVED

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

MeSH Terms

Conditions

Craniopharyngioma

Interventions

Proton Therapy; Radiotherapy; Contrast Media

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Heavy Ion Radiotherapy; Therapeutics; Diagnostic Uses of Chemicals; Pharmacologic Actions; Chemical Actions and Uses; Specialty Uses of Chemicals

Study Officials

• Thomas E Merchant, DO, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 16, 2011
• First Posted: August 17, 2011
• Study Start: August 22, 2011
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: April 29, 2026

Record last verified: 2026-04

Locations

US (2)