Anaplastic EpendymomaPhase 1NCT03750513
LET Optimized IMPT in Treating Pediatric Patients With Ependymoma
A Phase 1 study testing Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy, Quality-of-Life Assessment and Questionnaire Administration in people with Anaplastic Ependymoma and Ependymoma, run by M.D. Anderson Cancer Center at 2 sites in United States.
Trial summary
This phase I trial studies the side effects of linear energy transfer (LET) optimized image modulated proton therapy (IMPT) in treating pediatric patients with ependymoma. Radiation therapy such as LET optimized IMPT, uses proton beams to kill tumor cells and shrink tumors without damaging surrounding normal tissues. Read more about this study
- Who is it for?
- Ages Up to 22 years
- Conditions the study is for
- Anaplastic Ependymoma, Ependymoma
- What is being tested?
- Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy, Quality-of-Life Assessment and Questionnaire Administration
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Apr 2019to 31 Dec 2027
- Phase
- Phase 1
- 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 22 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Previous pathologic confirmation of ependymoma, World Health Organization (WHO) grade II or III
- Disease must be confined to the brain (no evidence of spread on MR imaging of the spine or on staging lumbar puncture)
- Patient may not receive chemotherapy concurrent with radiation
- Signed informed consent by patient and/or parents or legal guardian
- Lansky performance status score of 50 -100
Who cannot join
- Patients with previous radiation therapy to the brain
- Ependymoma of the spine
- Disseminated ependymoma requiring craniospinal radiation therapy
- Pregnancy
- Inability to undergo MR imaging
- Inability to receive gadolinium-based contrast agent
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Not yet recruiting
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Kaden L Mitra Litzinger, klitzinger@mgh.harvard.edu
Torunn Yock
- Recruiting
M D Anderson Cancer Center
Houston, Texas, 77030
David R. Grosshans, 713-563-2300
David R. Grosshans
About this study
Official title: Pilot Trial of LET Optimized IMPT for Pediatric Patients With Ependymoma
This phase I trial studies the side effects of linear energy transfer (LET) optimized image modulated proton therapy (IMPT) in treating pediatric patients with ependymoma. Radiation therapy such as LET optimized IMPT, uses proton beams to kill tumor cells and shrink tumors without damaging surrounding normal tissues.
Read the detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety of linear energy transfer (LET) optimized image modulated proton therapy (IMPT) (bio-IMPT) for pediatric patients with ependymoma.
SECONDARY OBJECTIVES:
- To utilize advanced multiparametric magnetic resonance (MR) imaging to identify imaging biomarkers of structural and biological changes after proton therapy in pediatric ependymoma patients.
II. To compare quantitative image biomarkers in patients treated with bio-IMPT and standard proton therapy using a voxel level analysis.
III. To test and evaluate the validity of relative biological effectiveness (RBE) models and enhance their precision based on prospectively collected clinical image biomarkers.
IV. To evaluate acute and late toxicities, including pseudoprogression and symptom burden, in patients treated with bio-IMPT.
- To estimate progression-free survival (PFS) and overall survival (OS). VI. To evaluate disease outcomes following the use of a simultaneous integrated boost (SIB) for pediatric ependymoma patients with gross residual disease following surgery.
OUTLINE:
Patients receive LET optimized IMPT for up to 6 weeks.
After completion of study treatment, patients are followed up at 1 month, then every 3 months for up to 24 months.
- Conditions
- Anaplastic Ependymoma, Ependymoma
- Being tested
- Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy, Quality-of-Life Assessment, Questionnaire Administration
- Medical subject headings
- Ependymoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Linear Energy Transfer-Optimized Intensity Modulated Proton TherapyRadiation
Also called LET-Optimized IMPT, LET-Optimized Intensity Modulated Proton Therapy, Linear Energy Transfer (LET)-Optimized Intensity Modulated Proton Therapy (IMPT)
Given LET optimized IMPT
Quality-of-Life AssessmentOther
Also called Quality of Life Assessment
Ancillary studies
Questionnaire AdministrationOther
Ancillary studies
| Study group | Role | What they receive |
|---|---|---|
| Treatment (LET optimized IMPT) | Experimental | Patients receive LET optimized IMPT for up to 6 weeks. Receives: Radiation: Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy; Other: Quality-of-Life Assessment; Other: Questionnaire Administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
The safety of linear energy transfer-optimized intensity modulated proton therapy will be defined by treatment failures which include imaging changes consistent with necrosis in the presence of Common Terminology Criteria for Adverse Events version 4.03 symptoms of grade 3 brain necrosis and/or local recurrence within the first 6 months from the end of proton therapy.
Measured: Up to 6 months post treatment
Secondary outcomes (7)
Identify imaging biomarkers of structural and biological changes after proton therapy
Descriptive statistics will be used to summarize the study data.
Measured: Up to 24 months post-treatment
Quantitative image biomarkers
Descriptive statistics will be used to summarize the study data.
Measured: Up to 24 months post-treatment
Validity of relative biological effectiveness models
Descriptive statistics will be used to summarize the study data.
Measured: Up to 24 months post-treatment
Incidence of late and acute toxicities
Descriptive statistics will be used to summarize the study data. For each study cohort, we will tabulate treatment- failures and other relevant clinically-related features. Interval estimates for proportions will be provided using exact 95% confidence intervals.
Measured: Up to 24 months post treatment
Progression-free survival
The method of Kaplan and Meier will be used to provide estimates.
Measured: Up to 24 months post treatment
Overall survival
The method of Kaplan and Meier will be used to provide estimates.
Measured: Up to 24 months post treatment
Disease outcomes following the use of a simultaneous integrated boost
Descriptive statistics will be used to summarize the study data.
Measured: Up to 24 months post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2018
- Study start1 Apr 2019
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anaplastic EpendymomaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- David R Grosshans, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03750513
- Sponsor's protocol ID
- 2018-0344
- Other IDs
- NCI-2018-02519, 2018-0344, P30CA016672, U19CA021239
- Status last verified
- May 2026
- First submitted
- 15 Nov 2018
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Trial of LET Optimized IMPT for Pediatric Patients With Ependymoma
- ClinicalTrials.gov ID
- NCT03750513
- Acronym / sponsor ID
- 2018-0344
- Conditions
- Anaplastic Ependymoma, Ependymoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 1 Apr 2019 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 23 Nov 2018 (actual)
- 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.
This phase I trial studies the side effects of linear energy transfer (LET) optimized image modulated proton therapy (IMPT) in treating pediatric patients with ependymoma. Radiation therapy such as LET optimized IMPT, uses proton beams to kill tumor cells and shrink tumors without damaging surrounding normal tissues.
Detailed description
PRIMARY OBJECTIVES:
- To evaluate the safety of linear energy transfer (LET) optimized image modulated proton therapy (IMPT) (bio-IMPT) for pediatric patients with ependymoma.
SECONDARY OBJECTIVES:
- To utilize advanced multiparametric magnetic resonance (MR) imaging to identify imaging biomarkers of structural and biological changes after proton therapy in pediatric ependymoma patients.
II. To compare quantitative image biomarkers in patients treated with bio-IMPT and standard proton therapy using a voxel level analysis.
III. To test and evaluate the validity of relative biological effectiveness (RBE) models and enhance their precision based on prospectively collected clinical image biomarkers.
IV. To evaluate acute and late toxicities, including pseudoprogression and symptom burden, in patients treated with bio-IMPT.
- To estimate progression-free survival (PFS) and overall survival (OS). VI. To evaluate disease outcomes following the use of a simultaneous integrated boost (SIB) for pediatric ependymoma patients with gross residual disease following surgery.
OUTLINE:
Patients receive LET optimized IMPT for up to 6 weeks.
After completion of study treatment, patients are followed up at 1 month, then every 3 months for up to 24 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (LET optimized IMPT) | Experimental | Radiation: Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy, Other: Quality-of-Life Assessment, Other: Questionnaire AdministrationPatients receive LET optimized IMPT for up to 6 weeks. |
Who may qualify
- Ages
- Up to 22 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Previous pathologic confirmation of ependymoma, World Health Organization (WHO) grade II or III
- Disease must be confined to the brain (no evidence of spread on MR imaging of the spine or on staging lumbar puncture)
- Patient may not receive chemotherapy concurrent with radiation
- Signed informed consent by patient and/or parents or legal guardian
- Lansky performance status score of 50 -100
Key exclusion criteria (6 listed)
- Patients with previous radiation therapy to the brain
- Ependymoma of the spine
- Disseminated ependymoma requiring craniospinal radiation therapy
- Pregnancy
- Inability to undergo MR imaging
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 |
|---|---|---|
| Linear Energy Transfer-Optimized Intensity Modulated Proton TherapyAlso: LET-Optimized IMPT, LET-Optimized Intensity Modulated Proton Therapy, Linear Energy Transfer (LET)-Optimized Intensity Modulated Proton Therapy (IMPT)Given LET optimized IMPT | Radiation | Treatment (LET optimized IMPT) |
| Quality-of-Life AssessmentAlso: Quality of Life AssessmentAncillary studies | Other | Treatment (LET optimized IMPT) |
| Questionnaire AdministrationAncillary studies | Other | Treatment (LET optimized IMPT) |
Primary outcome
Incidence of adverse events
Time frame: Up to 6 months post treatment
The safety of linear energy transfer-optimized intensity modulated proton therapy will be defined by treatment failures which include imaging changes consistent with necrosis in the presence of Common Terminology Criteria for Adverse Events version 4.03 symptoms of grade 3 brain necrosis and/or local recurrence within the first 6 months from the end of proton therapy.
7 secondary outcomes
Identify imaging biomarkers of structural and biological changes after proton therapy
Time frame: Up to 24 months post-treatment
Quantitative image biomarkers
Time frame: Up to 24 months post-treatment
Validity of relative biological effectiveness models
Time frame: Up to 24 months post-treatment
Incidence of late and acute toxicities
Time frame: Up to 24 months post treatment
Progression-free survival
Time frame: Up to 24 months post treatment
Overall survival
Time frame: Up to 24 months post treatment
Disease outcomes following the use of a simultaneous integrated boost
Time frame: Up to 24 months post-treatment
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 20 May 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Anaplastic Ependymoma
RecruitingPhase 1
Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
Central Nervous System Neoplasms, Glioblastoma
RecruitingPhase 1
HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors
Neoplasms, Brain, Glioblastoma Multiforme
Recruiting
Individualized Treatment Plan in Children and Young Adults With Relapsed Medulloblastoma and Ependymoma
Medulloblastoma, Medulloblastoma, Childhood
RecruitingPhase 1/2
Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors
High Grade Glioma, Meningioma
Active, not recruitingPhase 1
A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Medulloblastoma, Glioblastoma Multiforme
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.
David R Grosshans principal investigator
M.D. Anderson Cancer Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Linear Energy Transfer-Optimized Intensity Modulated Proton Therapy, Quality-of-Life Assessment and Questionnaire Administration in people with Anaplastic Ependymoma and Ependymoma, run by M.D. Anderson Cancer Center at 2 sites 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 5 inclusion and 6 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: looking for participants now, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Contact David Grosshans (713-563-2300, dgrossha@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Up to 6 months post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 May 2026.