Trial Outcomes & Findings for Everolimus for Children With Recurrent or Progressive Ependymoma (NCT NCT02155920)

Last Updated: 2024-05-08

Results Overview

Complete Response: Disappearance of all enhancing measurable and non-measurable disease Partial Response: ≥50% decrease in sum of products of perpendicular diameters of all measurable enhancing lesions compared with baseline

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

11 participants

Primary outcome timeframe

2 years

Results posted on

2024-05-08

Participant Flow

Eleven patients met the eligibility criteria and were enrolled on the study.

Participant milestones

Participant milestones
Measure	Everolimus The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Overall Study STARTED	11
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Everolimus for Children With Recurrent or Progressive Ependymoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Age, Continuous	1.5 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Race/Ethnicity, Customized White Non-Hispanic	9 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic	1 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants
Region of Enrollment United States	11 Participants n=5 Participants
Tumor Location at Diagnosis: Posterior Fossa Posterior Fossa	10 Participants n=5 Participants
Tumor Location at Diagnosis: Posterior Fossa Spinal Cord	1 Participants n=5 Participants
Molecular Subgroup PF-A	8 Participants n=5 Participants
Molecular Subgroup PF-B	1 Participants n=5 Participants
Molecular Subgroup No subgrouping available	2 Participants n=5 Participants
Tumor Histology WHO grade II (classic)	2 Participants n=5 Participants
Tumor Histology WHO grade III (anaplastic)	9 Participants n=5 Participants
Age at study enrollment	8 years n=5 Participants
Lansky Performance Scale	90 units on a scale n=5 Participants
Number of tumor recurrences	2 tumor recurrences n=5 Participants

PRIMARY outcome

Timeframe: 2 years

Outcome measures

Outcome measures
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Objective Response Rate (Complete Response Rate and Partial Response Rate) Following Treatment With Everolimus for Children With Recurrent or Progressive Ependymomas.	0 Participants

SECONDARY outcome

Timeframe: 2 years

Population: No participants had an Objective Response, therefore duration of response was not assessed in any participant

The duration of overall response is measured from the time measurement criteria are met for CR or PR (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented (taking as reference for progressive disease the smallest measurements recorded since the treatment started). The duration of overall CR is measured from the time measurement criteria are first met for CR until the first date that recurrent disease is objectively documented.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 2 years

Progression-free survival (PFS) is defined as the duration of time from start of treatment to time of progression.

Outcome measures

Outcome measures
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Progression Free Survival (PRS)	10.5 months Interval 3.5 to Subjects experienced tumor progression and discontinued therapy after a median of two cycles of therapy and hence range not reached.

SECONDARY outcome

Timeframe: 2 years

Event-Free Survival (EFS) is defined as the duration of time from start of treatment to: (1) disease progression; (2) second malignant neoplasm; (3) death regardless of cause; or (4) date of last contact, whichever comes first.

Outcome measures

Outcome measures
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Event Free Survival (EFS)	56 days Interval 28.0 to 224.0

SECONDARY outcome

Timeframe: 2 years

Adverse events will be graded by a numerical score according to the defined NCI Common Terminology Criteria for Adverse Events (NCI CTCAE) and Version 5.0. Adverse events not specifically defined in the NCI CTCAE will be scored on the Adverse Event log according to the general guidelines provided by the NCI CTCAE and as outlined below. * Grade 1: Mild * Grade 2: Moderate * Grade 3: Severe or medically significant but not immediately life threatening * Grade 4: Life threatening consequences * Grade 5: Death related to the adverse event

Outcome measures

Outcome measures
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability.	1 Participants

SECONDARY outcome

Timeframe: 2 years

Biomarkers of mTOR Pathway Activation of Ependymomas. Immunostaining of proteins associated with mTOR pathway activation would be performed and scored as 1+: weak, focal/multifocal, 2+: weak/diffuse, 3+: strong, focal/multifocal; 4+: strong/diffuse.

Outcome measures

Outcome measures
Measure	Everolimus n=11 Participants The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs. Everolimus: The recommended dose of Everolimus is 4.5 mg/m2/dose, once daily for up to 2 years, until disease progression or unacceptable toxicity occurs.
Number of Participants With Upregulated Biomarkers of mTOR Activation	9 participants

Adverse Events

Reported Adverse Events Among Patients Enrolled on Trial

Serious events: 1 serious events

Other events: 0 other events

Deaths: 10 deaths

Serious adverse events

Serious adverse events
Measure	Reported Adverse Events Among Patients Enrolled on Trial n=11 participants at risk One patient developed a grade 3 pneumonia requiring hospitalization and brief suspension of study drug. The remainder of reported adverse events were grades 1-2, spontaneously resolved, and were consistent with previously established adverse events associated with children taking everolimus.
Infections and infestations Pneumonia	9.1% 1/11 • Number of events 1 • 3 years NCI Common Toxicity Criteria for Adverse Events, version 4.03.30

Other adverse events

Adverse event data not reported

Additional Information

Daniel C. Bowers, MD

University of Texas Southwestern Medical School

Phone: 214-648-3896

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place