CraniopharyngiomaPhase 2NCT01419067
A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma or Observation After Radical Resection
A Phase 2 study testing Radical Surgery or Limited Surgery, Proton Therapy and ^1^8F-fluorodeoxyglucose in people with Craniopharyngioma, run by St. Jude Children's Research Hospital at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Craniopharyngioma
- What is being tested?
- Radical Surgery or Limited Surgery, Proton Therapy and ^1^8F-fluorodeoxyglucose
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 112 participants
- Where
- 2 sitesUnited States
- Study dates
- 22 Aug 2011to Jun 2031
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Craniopharyngioma diagnosed by histology, cytology or neuroimaging.
- Patients ages 0-21 years at the time of diagnosis.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Florida Health Proton Therapy Institute
Jacksonville, Florida, 32206
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
Official title: A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection
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.
Read the 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.
- Conditions
- Craniopharyngioma
- Being tested
- Radical Surgery or Limited Surgery, Proton Therapy, ^1^8F-fluorodeoxyglucose, ^1^1C-methionine
- Keywords
- Proton Therapy, Radical resection
- Medical subject headings
- Craniopharyngioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Radical Surgery or Limited SurgeryProcedure or surgery
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.
Proton TherapyDevice
Also called Radiation
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.
^1^8F-fluorodeoxyglucoseDrug
Also called ^1^8F-FDG, Contrast Media
This is a contrast media that will be given intravenously to aid in tumor visualization.
^1^1C-methionineDrug
Also called Contrast Media
This is a contrast media that will be given intravenously to aid in tumor visualization.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Procedure: Radical Surgery or Limited Surgery; Device: Proton Therapy; Drug: ^1^8F-fluorodeoxyglucose; Drug: ^1^1C-methionine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
This will be improved by models based on clinical and treatment factors and longitudinal measures of CNS effects.
Measured: 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
This will be improved by models based on clinical and treatment factors and longitudinal measures of CNS effects
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2011 (estimated)
- Study start22 Aug 2011
- Primary completionJun 2031 (estimated)
- Study completionJun 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for CraniopharyngiomaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 112 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Thomas E Merchant, DO, PhD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01419067
- Sponsor's protocol ID
- RT2CR
- Other IDs
- NCI-2011-03707
- Status last verified
- Aug 2026
- First submitted
- 16 Aug 2011
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Publications
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.
PubMed 37084748Wilson 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.
PubMed 33607632