CraniopharyngiomaPhase 2NCT02792582
A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection
A Phase 2 study testing Proton Therapy and Surgery in people with Craniopharyngioma, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Craniopharyngioma
- What is being tested?
- Proton Therapy and Surgery
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Jun 2016to Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 or intra-operative assessment
- 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 or intracystic bleomycin
- Pregnant females are excluded. Radiation has teratogenic or abortifacient effects
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
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.
Read the detailed description
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:
- To explore potential associations of clinical and treatment factors with the incidence and severity of neurological, endocrine and cognitive deficits in children and young adults with craniopharyngioma treated with radical surgery or proton therapy. Descriptively compare findings for patients treated with proton therapy with the reported findings for the updated SJCRH cohort treated with photon therapy.
- Using specific measures of sleep quality, excessive daytime sleepiness, daytime activity, circadian rhythm, fatigue, symptom distresses, and quality of life, explore associations of sleep, fatigue and quality of life with other measures of central nervous system (CNS) effects, clinical and treatment factors in children and young adults with craniopharyngioma treated with radical surgery or proton therapy.
- To evaluate and explore differences in physical performance and movement in children and young adults with craniopharyngioma treated with radical surgery or proton therapy, using specific measures of overall physical performance, flexibility, balance, coordination, muscle strength and power, and cardiopulmonary fitness.
- Estimate and compare the response of residual tumor and the incidence and severity of structural, functional and vascular effects of normal brain in children and young adults with craniopharyngioma after treatment with radical surgery or proton therapy using specific methods of diffusion, contrast-enhancement, vascular and functional neuroimaging, and explore the association between these and other measures of CNS effects and clinical and treatment factors.
- Investigate the feasibility of using positron emission tomography (PET) as an in vivo dose and distal edge verification system for craniopharyngioma patients treated with proton therapy.
- Measure growth factor and cytokine responses in children and young adults with craniopharyngioma after treatment with radical surgery or proton therapy, and explore associations between these and other measures of CNS effects and clinical and treatment factors. Descriptively compare findings for patients treated with proton therapy with the reported findings for the updated SJCRH cohort treated with photon therapy.
- Conditions
- Craniopharyngioma
- Being tested
- Proton Therapy, Surgery
- Keywords
- Brain tumor, Pediatrics, Radiation therapy, Proton therapy, Surgery
- Medical subject headings
- Craniopharyngioma, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Proton TherapyRadiation
Also called Radiation therapy
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.
SurgeryProcedure or surgery
Also called Tumor resection
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Radiation: Proton Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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
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.
Measured: 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
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.
Measured: 3 years after initiation of proton therapy
Compare survival distributions with participants treated with conventional radiation therapy at St. Jude Children's Research Hospital
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).
Measured: 3 years after initiation of proton therapy
Compare survival distributions between groups
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.
Measured: 3 years after initiation of proton therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jun 2016 (estimated)
- Study start22 Jun 2016
- Primary completionOct 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 160 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
- NCT02792582
- Sponsor's protocol ID
- RT3CR
- Other IDs
- NCI-2016-00813
- Status last verified
- Sep 2026
- First submitted
- 1 Jun 2016
- Last updated
- 22 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Publications
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.
PubMed 33607632
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection
- ClinicalTrials.gov ID
- NCT02792582
- Acronym / sponsor ID
- RT3CR
- Conditions
- Craniopharyngioma
- Keywords
- Brain tumor, Pediatrics, Radiation therapy, Proton therapy, Surgery
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 160 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 22 Jun 2016 (actual)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 7 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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:
- To explore potential associations of clinical and treatment factors with the incidence and severity of neurological, endocrine and cognitive deficits in children and young adults with craniopharyngioma treated with radical surgery or proton therapy. Descriptively compare findings for patients treated with proton therapy with the reported findings for the updated SJCRH cohort treated with photon therapy.
- Using specific measures of sleep quality, excessive daytime sleepiness, daytime activity, circadian rhythm, fatigue, symptom distresses, and quality of life, explore associations of sleep, fatigue and quality of life with other measures of central nervous system (CNS) effects, clinical and treatment factors in children and young adults with craniopharyngioma treated with radical surgery or proton therapy.
- To evaluate and explore differences in physical performance and movement in children and young adults with craniopharyngioma treated with radical surgery or proton therapy, using specific measures of overall physical performance, flexibility, balance, coordination, muscle strength and power, and cardiopulmonary fitness.
- Estimate and compare the response of residual tumor and the incidence and severity of structural, functional and vascular effects of normal brain in children and young adults with craniopharyngioma after treatment with radical surgery or proton therapy using specific methods of diffusion, contrast-enhancement, vascular and functional neuroimaging, and explore the association between these and other measures of CNS effects and clinical and treatment factors.
- Investigate the feasibility of using positron emission tomography (PET) as an in vivo dose and distal edge verification system for craniopharyngioma patients treated with proton therapy.
- Measure growth factor and cytokine responses in children and young adults with craniopharyngioma after treatment with radical surgery or proton therapy, and explore associations between these and other measures of CNS effects and clinical and treatment factors. Descriptively compare findings for patients treated with proton therapy with the reported findings for the updated SJCRH cohort treated with photon therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tumor-Surgery | Active comparator | Radiation: Proton Therapy, Procedure: SurgeryParticipants 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. |
| Tumor-No Surgery | Active comparator | Radiation: Proton TherapyParticipants 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. |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Craniopharyngioma diagnosed by histology, cytology or neuroimaging or intra-operative assessment
- Patients ages 0-21 years at the time of diagnosis
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Proton TherapyAlso: Radiation therapyThe 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. | Radiation | Tumor-No Surgery, Tumor-Surgery |
| SurgeryAlso: Tumor resectionWhen 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. | Procedure or surgery | Tumor-Surgery |
Primary outcome
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.
4 secondary outcomes
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
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
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
Compare survival distributions between groups
Time frame: 3 years after initiation of proton therapy
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2026.
- Derived from this study: PubMed 33607632Wilson 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.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas E. Merchant, DO, PhD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Proton Therapy and Surgery in people with Craniopharyngioma, run by St. Jude Children's Research Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 22 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression-free survival (PFS) (time frame: 3 years after initiation of proton therapy). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02792582, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2026.