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

NCT02792582 | Active Not Recruiting Phase 2 FDA Device

• 2 other identifiers: RT3CRNCI-2016-00813
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

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.

Conditions

Craniopharyngioma

Keywords

Brain tumor; Pediatrics; Radiation therapy; Proton therapy; Surgery

Outcome Measures

Primary Outcomes (2)

• Progression-free survival (PFS)

  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.

  3 years after initiation of proton therapy

• Overall survival (OS)

  OS is defined as the interval of time from the initiation of RT until the death from any cause.

  3 years after initiation of proton therapy

Secondary Outcomes (4)

• Compare incidence of first cystic intervention with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital

  3 years after initiation of proton therapy

• Compare event-free survival (EFS) with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital

  3 years after initiation of proton therapy

• Compare survival distributions with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital

  3 years after initiation of proton therapy

• Compare survival distributions between groups

  3 years after initiation of proton therapy

Other Outcomes (13)

• Change in vision status compared between groups

  From baseline to 5 years following start of proton therapy

• Change in hearing status compared between groups

  From baseline to 5 years following start of proton therapy

• Compare number of neurological deficits to participants treated using conventional radiation therapy at St. Jude Children's Research Hospital

  From baseline to 5 years following start of proton therapy

Study Arms (2)

Tumor-Surgery

ACTIVE COMPARATOR

Participants who are eligible to undergo surgery to remove the tumor will proceed to surgery. If all tumor is removed, they will be followed over 5 years for outcome comparison to the other participant groups. If the entire tumor is not removed by surgery, participants will receive 6 weeks of proton therapy. They will then be followed for 5 years to collect outcome data for comparison to the other participant groups.

Radiation: Proton Therapy; Procedure: Surgery

Tumor-No Surgery

ACTIVE COMPARATOR

Participants whose tumor cannot be resected through surgery will receive 6 weeks of proton therapy. They will then be followed over 5 years for outcome comparison to the other participant groups.

Radiation: Proton Therapy

Interventions

Proton Therapy RADIATION

The protocol-specific radiation dose will be prescribed to a target defined by the use of computerized tomography (CT) and magnetic resonance imaging (MRI). The target will encompass the residual tumor and/or tumor bed and will include a margin mean to treat subclinical microscopic disease. There is no time limit for the interval from surgery or study enrollment to when proton therapy commences. The time course of administration of proton therapy will be 1 treatment/day, 5 days/week, for a period 6 weeks.

Also known as: Radiation therapy

Tumor-No Surgery; Tumor-Surgery

Surgery PROCEDURE

When possible, tumors will be removed surgically prior to proton therapy. If no tumor remains following surgery, no proton therapy will be given, and participants will be followed for 5 years to collect outcome data for comparison between groups.

Also known as: Tumor resection

Tumor-Surgery

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 or intra-operative assessment
• 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 or intracystic bleomycin
• Pregnant females are excluded. Radiation has teratogenic or abortifacient effects

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Publications (1)

• 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; Brain Neoplasms

Interventions

Proton Therapy; Radiotherapy; Surgical Procedures, Operative; Transurethral Resection of Bladder

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Heavy Ion Radiotherapy; Therapeutics; Urologic Surgical Procedures; Urogenital Surgical Procedures

Study Officials

• Thomas E. Merchant, DO, PhD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 1, 2016
• First Posted: June 7, 2016
• Study Start: June 22, 2016
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): December 1, 2028
• Last Updated: April 23, 2026

Record last verified: 2026-04

Locations

US (1)