TriangleNCT03387020 · STUDY IN CONTEXT
Ribociclib and Everolimus in Treating Children With Recurrent or Refractory Malignant Brain Tumors
Draft scope assessment: unclear. Attached because the study's own record describes recurrent disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
Read the full study record and all posted results →
Linked outcomes
Ribociclib plasma concentration-time data obtained from patients who received Phase I defined treatment will be modeled using compartmental approaches to estimate the AUC (hr\*μM) for each dose level on days 1 and 17 of course 1. Patients who were enrolled in the Surgical stratum but subsequently received Phase I defined treatment with PK data available will also be included in the analysis population. 5 patients from surgical stratum received dose level 1 treatment.
Everolimus plasma concentration-time data obtained from patients who received Phase I defined treatment will be modeled using compartmental approaches to estimate the AUC (hr\*nM) for each dose level on day 17 of course 1 and day 1 of course 2. Patients who were enrolled in the Surgical stratum but subsequently received Phase I defined treatment with PK data available will also be included in the analysis population. 5 patients from surgical stratum received dose level 1 treatment.
Defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose limiting toxicity (DLT) and the next higher dose level has been determined to be too toxic. Will be estimated using a Rolling-6 phase I design. Only patients who are evaluable for DLT will be consider for estimating of Maximum Tolerated Dose. Patient who were enrolled under Surgical cohort but received sufficient study defined treatment would also be evaluable for DLT.
Average tumor Ribociclib concentrations will be compared to plasma concentrations. The mean tumor-to-plasma ratio of Ribociclib at the time of surgery will be described.
Number of patients with sustained objective responses will be reported by stratum and dose. Objective response refers to at least 50% reduction in 2 dimensional measurements of the tumor. In order to count towards this objective, any response needs to be sustained for at least 8 weeks.
For the surgical study, Ki67 will be evaluated in paired pre-treatment tumor (diagnostic) and post-treatment tumor (recurrent) tissue in consenting patients to assess percent change. The percent of Ki67 in tumor tissues will be measured and the result is presented as the percent of Ki67 in post-treatment tumor minus the percent of Ki67 measured in pre-treatment tumor.
Reported safety
Quality of life and patient-reported measures
No linked quality-of-life measure is recorded for this setting and study.
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- trial label
- Ribociclib and Everolimus in Treating Children With Recurrent or Refractory Malignant Brain Tumors
- applicability
- unclear
- applicability note
- Attached because the study's own record describes recurrent disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- registry-condition-search
- discovery note
- Found by searching ClinicalTrials.gov for this cancer type, restricted to studies that have posted results to the registry. No FDA notice, FDA label or NCI summary held by this site cites this study. Because the search asked for posted results, studies without posted results were never looked for, so their absence from this page means nothing.
- outcome Count
- 6
- harms Count
- 3
- qol Count
- 0
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0004
- source section id
- NCT03387020-om-0004
- source outcome index
- 4
- endpoint
- Ribociclib AUC (Area Under the Plasma Concentration Versus Time Curve) as Obtained From the Patients Who Received Phase I Defined Treatment
- endpoint definition
- Ribociclib plasma concentration-time data obtained from patients who received Phase I defined treatment will be modeled using compartmental approaches to estimate the AUC (hr\*μM) for each dose level on days 1 and 17 of course 1. Patients who were enrolled in the Surgical stratum but subsequently received Phase I defined treatment with PK data available will also be included in the analysis population. 5 patients from surgical stratum received dose level 1 treatment.
- time origin
- Pre-dose, 1, 2, 4, 8, 24, 32 (day 1 course 1 only), & 48 (day 1 course 1 only) hours after dose on days 1 and 17 of course 1.
- analysis role
- exploratory
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Patients with blood samples collected for pharmacokinetic studies on days 1 and 17 of course 1 were included. Surgical patients who received Phase I treatment with samples collected were also included in Dose Level 1. There are total 17 patients received dose level 1 treatment and 3 patients received dose level 2 treatment.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0005
- source section id
- NCT03387020-om-0005
- source outcome index
- 5
- endpoint
- Everolimus AUC (Area Under the Plasma Concentration Versus Time Curve) as Obtained From the Phase I Study
- endpoint definition
- Everolimus plasma concentration-time data obtained from patients who received Phase I defined treatment will be modeled using compartmental approaches to estimate the AUC (hr\*nM) for each dose level on day 17 of course 1 and day 1 of course 2. Patients who were enrolled in the Surgical stratum but subsequently received Phase I defined treatment with PK data available will also be included in the analysis population. 5 patients from surgical stratum received dose level 1 treatment.
- time origin
- Pre-dose, 0.5 (course 2 only), 1, 1.5 (course 2 only), 2, 4, 6 (course 2 only), 8, 24 hours after dose on day 17 of course 1, and day 1 of course 2.
- analysis role
- exploratory
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Patients with blood samples collected for pharmacokinetic studies on day 17 of course 1 and day 1 of course 2 were included. Surgical patients who received Phase I treatment with samples collected were also included in Dose Level 1. There are total 17 patients received dose level 1 treatment and 3 patients received dose level 2 treatment. Analysis population only includes treated patients with corresponding PK data available.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0000
- source section id
- NCT03387020-om-0000
- source outcome index
- 0
- endpoint
- Maximum Tolerated Dose of Ribociclib and Everolimus
- endpoint definition
- Defined as the highest dose level at which six patients have been treated with at most one patient experiencing a dose limiting toxicity (DLT) and the next higher dose level has been determined to be too toxic. Will be estimated using a Rolling-6 phase I design. Only patients who are evaluable for DLT will be consider for estimating of Maximum Tolerated Dose. Patient who were enrolled under Surgical cohort but received sufficient study defined treatment would also be evaluable for DLT.
- time origin
- 4 weeks
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Patients who were evaluable for dose finding assessment were used to determine the MTD. Evaluable patients are those who were eligible, started treatment and either experienced a dose limiting toxicity during course 1 or received adequate amount of treatment and were followed adequately to be assessed for safety during course 1. Evaluable patients could be from either Phase I stratum or from Surgical stratum as long as they received sufficient protocol defined Phase I treatment.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0001
- source section id
- NCT03387020-om-0001
- source outcome index
- 1
- endpoint
- Average Tumor and Plasma Concentrations of Ribociclib for the Surgical Study
- endpoint definition
- Average tumor Ribociclib concentrations will be compared to plasma concentrations. The mean tumor-to-plasma ratio of Ribociclib at the time of surgery will be described.
- time origin
- Surgical study: Prior to starting ribociclib (day -7 or -10), prior to ribociclib dose on days -5 & -2, day 0, and at the time of surgery
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Patients who were enrolled on surgical study and with tumor and matching plasma samples available.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0002
- source section id
- NCT03387020-om-0002
- source outcome index
- 2
- endpoint
- Objective Responses (Complete Response + Partial Response)
- endpoint definition
- Number of patients with sustained objective responses will be reported by stratum and dose. Objective response refers to at least 50% reduction in 2 dimensional measurements of the tumor. In order to count towards this objective, any response needs to be sustained for at least 8 weeks.
- time origin
- Up to 2 years
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Patients who received Phase I treatment were included in the analysis. One patient in Phase I study withdrew prior to beginning protocol therapy and one patient in Surgical study was not eligible to start Phase I treatment.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0003
- source section id
- NCT03387020-om-0003
- source outcome index
- 3
- endpoint
- Percent Change in ki67 Between Archival and Post-treatment Tissue
- endpoint definition
- For the surgical study, Ki67 will be evaluated in paired pre-treatment tumor (diagnostic) and post-treatment tumor (recurrent) tissue in consenting patients to assess percent change. The percent of Ki67 in tumor tissues will be measured and the result is presented as the percent of Ki67 in post-treatment tumor minus the percent of Ki67 measured in pre-treatment tumor.
- time origin
- Up to 2 years
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Consented patients in surgical study with pre-treatment tumor and post-treatment tumor tissue available
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0000
- arm key
- OG000
- arm label
- DLT Evaluable Set
- regimen
- Patients either from Phase I stratum and received treatment from beginning, or from Surgical stratum who subsequently received Phase I defined treatment would be evaluable for MTD as long as they received sufficient Phase I defined treatment. Of the patients enrolled in Phase I stratum, 11 of them were evaluable for dose finding assessment. Of the patients enrolled in Surgical stratum, 5 of them were evaluable for dose finding assessment. Therefore, we have total 16 patients evaluable for dose finding assessment, 11 from Phase I stratum and 5 from Surgical stratum.
- analyzed count
- 16
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0001
- arm key
- OG000
- arm label
- Surgical
- regimen
- Patients who are undergoing surgery also receive ribociclib PO QD on days 7-10 at 120 mg/m2/day before surgery
- analyzed count
- 5
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0002
- arm key
- OG000
- arm label
- Phase I, Dose Level 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 120 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day
- analyzed count
- 12
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0002
- arm key
- OG001
- arm label
- Phase I, Dose Level 2
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 170 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day
- analyzed count
- 3
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0002
- arm key
- OG002
- arm label
- Surgical
- regimen
- Patients who are undergoing surgery also received ribociclib PO QD on days 7-10 at 120 mg/m2/day before surgery. Eligible patients were subsequently treated on Phase 1, Dose Level 1.
- analyzed count
- 5
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0002#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0003
- arm key
- OG000
- arm label
- Surgical
- regimen
- Patients who are undergoing surgery also receive ribociclib PO QD on days 7-10 at 120 mg/m2/day before surgery
- analyzed count
- 2
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0003#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0004
- arm key
- OG000
- arm label
- Dose Level 1, Day 1 of Course 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 120 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 1 of Course 1.
- analyzed count
- 16
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0004#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0004
- arm key
- OG001
- arm label
- Dose Level 1, Day 17 of Course 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 120 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 17 of Course 1.
- analyzed count
- 15
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0004#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0004
- arm key
- OG002
- arm label
- Dose Level 2, Day 1 of Course 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 170 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 1 of Course 1.
- analyzed count
- 3
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0004#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0004
- arm key
- OG003
- arm label
- Dose Level 2, Day 17 of Course 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 170 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 17 of Course 1
- analyzed count
- 2
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0004#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0005
- arm key
- OG000
- arm label
- Dose Level 1, Day 17 of Course 1
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 120 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 17 of Course 1.
- analyzed count
- 14
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0005#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- outcome key
- om-0005
- arm key
- OG001
- arm label
- Dose Level 1, Day 1 of Course 2
- regimen
- Patients receive ribociclib PO QD on days 1-21 of course 1 and subsequent courses and days 2-21 of course 2 at 120 mg/m2/day, and everolimus PO QD on days 3-28 of course 1 and days 1-28 of subsequent courses at 1.2 mg/m2/day. Samples are collected on Day 1 of Course 2.
- analyzed count
- 10
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03387020-om-0005#groups/OG001
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- harm key
- EG000
- source section id
- NCT03387020-ae
- event group key
- EG000
- event group label
- Phase I, Dose Level 1
- serious affected
- 6
- serious at risk
- 12
- other affected
- 12
- other at risk
- 12
- frequency threshold
- 5
- collection period
- Up to 2 years after starting treatment
- event definition
- Adverse events (AEs) were graded using CTCAE v5.0. AEs collected per protocol included: a) grade 1 and 2 AEs if attribution was at least possibly related to Ribociclib or Everolimus and b) all grade 3+ AEs on treatment and within 30 days off treatment. AEs were summarized in SAE and Other AE tables below. All mortality on this study were due to disease. Two patients did not started phase I protocol defined therapy and were not counted as at risk.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- harm key
- EG001
- source section id
- NCT03387020-ae
- event group key
- EG001
- event group label
- Phase I, Dose Level 2
- serious affected
- 1
- serious at risk
- 3
- other affected
- 3
- other at risk
- 3
- frequency threshold
- 5
- collection period
- Up to 2 years after starting treatment
- event definition
- Adverse events (AEs) were graded using CTCAE v5.0. AEs collected per protocol included: a) grade 1 and 2 AEs if attribution was at least possibly related to Ribociclib or Everolimus and b) all grade 3+ AEs on treatment and within 30 days off treatment. AEs were summarized in SAE and Other AE tables below. All mortality on this study were due to disease. Two patients did not started phase I protocol defined therapy and were not counted as at risk.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-ependymoma-pediatric-recurrent-72f73bfa
- nct id
- NCT03387020
- harm key
- EG002
- source section id
- NCT03387020-ae
- event group key
- EG002
- event group label
- Surgical
- serious affected
- 1
- serious at risk
- 5
- other affected
- 5
- other at risk
- 5
- frequency threshold
- 5
- collection period
- Up to 2 years after starting treatment
- event definition
- Adverse events (AEs) were graded using CTCAE v5.0. AEs collected per protocol included: a) grade 1 and 2 AEs if attribution was at least possibly related to Ribociclib or Everolimus and b) all grade 3+ AEs on treatment and within 30 days off treatment. AEs were summarized in SAE and Other AE tables below. All mortality on this study were due to disease. Two patients did not started phase I protocol defined therapy and were not counted as at risk.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- setting source links
Preserved source evidence · Not medical advice