Trial Outcomes & Findings for A Trial of Surgery and Fractionated Re-Irradiation for Recurrent Ependymoma (NCT NCT02125786)

NCT ID: NCT02125786

Last Updated: 2025-05-08

Results Overview

To prospectively estimate the progression-free distribution for children and young adults with recurrent ependymoma treated with a second course of irradiation.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 68 participants
• Primary outcome timeframe: 2 years follow-up after initiation of radiation therapy for the last patient enrolled
• Results posted on: 2025-05-08

Participant Flow

70 enrollments occurred on 68 participants between from April 21, 2014 and June 30, 2020.

Of 70 enrollments, 3 were found to be ineligible and removed; 3 withdrew before reirradiation to seek other treatment. One participant who was initially ineligible was later found to be eligible and re-enrolled, and a participant who had previously withdrawn to seek other treatment options decided to pursue reirradiation and re-enrolled. The 2 screen failures St. Jude pathology did not result ependymoma diagnosis after consent as expected. Sixty-four (64) completed reirradiation treatment.

Participant milestones

Measure	Reirradiation Children and young adults with Recurrent Ependymoma undergoing reirradiation
Overall Study STARTED	68
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	59

Reasons for withdrawal

Measure	Reirradiation Children and young adults with Recurrent Ependymoma undergoing reirradiation
Overall Study Still in Follow-up	3
Overall Study Screen Failure	2
Overall Study Withdrawal by Family before Start of RT	2
Overall Study Progressive Disease	44
Overall Study Death	8

Baseline Characteristics

A Trial of Surgery and Fractionated Re-Irradiation for Recurrent Ependymoma

Baseline characteristics by cohort

Measure	Reirradiation n=64 Participants Children and young adults with Recurrent Ependymoma undergoing reirradiation.
Age, Continuous	9.95 years STANDARD_DEVIATION 5.11 • n=5 Participants
Sex: Female, Male Female	24 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	55 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	8 Participants n=5 Participants
Race (NIH/OMB) White	51 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Treatment Stratum Stratum 1(Initial pattern of failure is local) Treatment: Focal Irradiation	25 Participants n=5 Participants
Treatment Stratum Stratum 2 (Initial pattern of failure is metastatic) Treatment: CSI	16 Participants n=5 Participants
Treatment Stratum Stratum 3 (Initial pattern of failure is local and metastatic) Treatment: CSI	15 Participants n=5 Participants
Treatment Stratum Stratum 4 (Initial pattern of failure is local) Treatment: CSI (optional-presence of 1q gain & >3yo)	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 years follow-up after initiation of radiation therapy for the last patient enrolled

To prospectively estimate the progression-free distribution for children and young adults with recurrent ependymoma treated with a second course of irradiation.

Outcome measures

Measure	Reirradiation n=64 Participants Children and young adults with Recurrent Ependymoma undergoing reirradiation
3-year Progression-free Survival Rate	21.88 percentage of participants Interval 12.73 to 32.61

PRIMARY outcome

Timeframe: 2 years follow-up after initiation of radiation therapy for the last patient enrolled

OS was calculated from the date of initial radiation therapy to date of death or date of last contact for the patients who were treated with RT. The event of interest was death.

Outcome measures

Measure	Reirradiation n=64 Participants Children and young adults with Recurrent Ependymoma undergoing reirradiation
3-year Overall Survival Rate	60.33 percentage of participants Interval 47.14 to 71.2

SECONDARY outcome

Timeframe: Through 5 years after initiation of second course of irradiation

Evaluation will be in children and young adults with ependymoma treated with a second course of irradiation.

Outcomes will be reported by p-values.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 5 years after initiation of second course of irradiation

Evaluation will be in children and young adults with ependymoma treated with a second course of irradiation.

Outcome will be reported with p values.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 5 years after initiation of second course of irradiation

Evaluation will be in children and young adults with ependymoma treated with a second course of irradiation.

Outcome will be reported with p-values.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 5 years after initiation of second course of irradiation

Evaluation will be in children and young adults with ependymoma treated with a second course of irradiation.

Outcome will be reported with p-values.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through 5 years after initiation of second course of irradiation

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Measured task sets: BOT2 (4-21 years old) and PPT (≥22 years old).

• BOT2 outcome: total motor composite, a standardized score range from 20 to 80, with a mean of 50 and a standard deviation of 10.
• PPT (7-item questionnaire) outcome: a score range from 0 to 28. The normative data is available from the human performance lab.

The above scores will be standardized into z-scores/T-scores for data analysis based on normative data.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Self-reported instruments (PROMIS): Three questionnaires.

• Pediatric Physical Function - Mobility - SF1: 8-item questionnaire with the score range from 0 to 32.
• Pediatric Physical Function - Upper Extremity - SF1: same as above.
• Physical Function - SF1: 10-item questionnaire with the score range from 0 to 50.

The above scores will be standardized into z-scores/T-scores for data analysis based on normative data

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Ankle dorsiflexion active and passive range of motion: measured by goniometer, and recorded as an angle (degree).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Overall flexibility: measured by sit and reach test, and recorded as a length (cm).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Balance measured by Measured by sensory organization test (SOT), and the result from the test is the equilibrium score, a percentage range from 0% to 100%, with the higher percentage, the better balance. The outcome is a binary variable with a cutoff score of < 70% indicates future risk for a fall.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Fine motor coordination (finger and hand coordination). Measured by Composite Cerebellar Functional Severity Score (CCFS), and the outcome is an age-adjusted z-score and log transformed.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Fine motor coordination (finger and hand coordination).

Baseline is defined as day 1 of radiation therapy.

Overall coordination measured by brief ataxia rating scale (five-item questionnaire). The outcome is a total scale range from 0 to 22.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Lower extremity strength measured by BiodexIII. The outcomes are peak torque value/body weight ratios at different speeds of motion.

The scores will be standardized into z-scores for data analysis based on normative data.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Hand grip strength is measured by a Jamar hand held dynamometer and recorded in kilograms (kg).

The scores will be standardized into z-scores for data analysis based on normative data.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Resting energy expenditure is measured with indirect calorimetry after an overnight fast and recorded as REE (kcal/day).

The scores will be standardized into z-scores for data analysis based on normative data.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as day 1 of radiation therapy.

Cardiopulmonary exercise test (CPET) will be completed on a treadmill using the Balke protocol or cycle ergometer using an incremental ramping protocol. The outcome is recorded as VO2max (ml/kg/min).

The scores will be standardized into z-scores for data analysis based on normative data.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as the last MRI prior to initiation of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline through 5 years after initiation of radiation therapy

Baseline is defined as the last MRI prior to initiation of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to recovery of gray and white matter tract injury, up to a maximum of 5 years

This outcome will be evaluated by neuroimaging. Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to recovery of gray and white matter tract injury, up to a maximum of 5 years

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to recovery of gray and white matter tract injury, up to a maximum of 5 years

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline

Baseline is the last FDG-PET and MET-PET prior to initiation of radiation therapy.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 12, 24 and 36 months after second course of irradiation

Correlation of avidity of ependymoma to 18F-fluorodeoxyglucose and 11C-methionine PET prior to radiation therapy with progression-free survival will be reported

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline, and at 12, 24 and 36 month

Association between necrosis measured with MET/FDG vs. MRI Baseline is defined as day 1 of radiation therapy (RT).

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline through 5 years after radiation therapy

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline

Evaluate will be of genetic variations in germline associated with treatment response and side effects. P-values will be reported.

Baseline is defined as day 1 of radiation therapy.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 2 years after initiation of irradiation for the last patient enrolled

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 2 years after initiation of irradiation for the last patient enrolled

Outcome measures

Outcome data not reported

Adverse Events

Reirradiation

Serious events: 2 serious events

Other events: 30 other events

Deaths: 8 deaths

Serious adverse events

Measure	Reirradiation n=64 participants at risk Children and young adults with Recurrent Ependymoma undergoing reirradiation.
Gastrointestinal disorders Gastric Hemorrhage	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
General disorders Death NOS	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.

Other adverse events

Measure	Reirradiation n=64 participants at risk Children and young adults with Recurrent Ependymoma undergoing reirradiation.
Blood and lymphatic system disorders Febrile neutropenia	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Cardiac disorders Sinus bradycardia	3.1% 2/64 • Number of events 2 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Cardiac disorders Sinus tachycardia	4.7% 3/64 • Number of events 3 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
General disorders Other-Pseudomeningocele	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Infections and infestations Peripheral nerve infection	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Injury, poisoning and procedural complications Wound complication	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Investigations Lymphocyte count decreased	3.1% 2/64 • Number of events 2 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Nervous system disorders Central nervous system necrosis	23.4% 15/64 • Number of events 15 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Nervous system disorders Cerebrospinal fluid leakage	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Nervous system disorders Headache	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Nervous system disorders Seizure	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Respiratory, thoracic and mediastinal disorders Other - Bradypnea	10.9% 7/64 • Number of events 9 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Respiratory, thoracic and mediastinal disorders Other - Tachypnea	7.8% 5/64 • Number of events 5 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Vascular disorders Hypertension	3.1% 2/64 • Number of events 2 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.
Vascular disorders Hypotension	1.6% 1/64 • Number of events 1 • 11C-Methionine-Related Adverse Events: From time of injection through one hour post injection of 11C-methionine. Radiation-Related Adverse Events: From initiation of radiation therapy through study completion, an average of 2 years. All-cause mortality was assessed in eligible participants who received radiation therapy. Adverse events were assessed in eligible participants with adverse event data who received radiation therapy.

Additional Information

Thomas E. Merchant, DO, PhD

St. Jude Children's Research Hospital

Phone: 8662785833

Email: referralinfo@stjude.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place