Trial Outcomes & Findings for A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors (NCT NCT03257631)

Last Updated: 2024-04-16

Results Overview

The percentage of participants who achieved either an objective response, defined as a complete response (CR) or partial response (PR) in the first 6 cycles of treatment (or within 3 cycles for DIPG), or long-term stable disease (SD) defined as SD maintained for ≥ 6 cycles (≥ 3 cycles for DIPG), measured from first dose date. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions, and/or persistence of non-target lesions with no progression or decrease in size. SD: A decrease of \< 50% or an increase of \< 25% in the size of measurable lesions and no evidence of new lesions, response does not meet the criteria for CR, PR, or progressive disease, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

53 participants

Primary outcome timeframe

6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group

Results posted on

2024-04-16

Participant Flow

The study consisted of 4 groups, for each of the following primary brain tumor types: diffuse intrinsic pontine glioma (DIPG), ependymoma, high-grade glioma, and medulloblastoma.

In stage 1 approximately 9 participants were to be enrolled in parallel to each group. If two or more participants in a group achieved an objective response or long-term stable disease within the first 6 cycles of treatment (within the first 3 cycles for DIPG) an additional 11 participants were to be enrolled in that group.

Participant milestones

Participant milestones
Measure	Diffuse Intrinsic Pontine Glioma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Overall Study STARTED	11	9	23	10
Overall Study Received Study Drug	11	9	22	10
Overall Study COMPLETED	0	0	1	0
Overall Study NOT COMPLETED	11	9	22	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Diffuse Intrinsic Pontine Glioma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Overall Study Death	1	0	1	1
Overall Study Progressive Disease	10	9	17	8
Overall Study Withdrawal by Parent/Guardian	0	0	2	1
Overall Study Adverse Event	0	0	2	0

Baseline Characteristics

A Study of Pomalidomide Monotherapy for Children and Young Adults With Recurrent or Progressive Primary Brain Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Diffuse Intrinsic Pontine Glioma n=11 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=23 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=10 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Total n=53 Participants Total of all reporting groups
Age, Continuous	7.0 years n=5 Participants	12.0 years n=7 Participants	14.0 years n=5 Participants	10.0 years n=4 Participants	12.0 years n=21 Participants
Age, Customized ≥ 1 to < 6 years	1 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	1 Participants n=4 Participants	5 Participants n=21 Participants
Age, Customized ≥ 6 to < 12 years	9 Participants n=5 Participants	1 Participants n=7 Participants	5 Participants n=5 Participants	6 Participants n=4 Participants	21 Participants n=21 Participants
Age, Customized ≥ 12 years	1 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants	3 Participants n=4 Participants	27 Participants n=21 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants	3 Participants n=4 Participants	19 Participants n=21 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	5 Participants n=7 Participants	15 Participants n=5 Participants	7 Participants n=4 Participants	34 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants	0 Participants n=7 Participants	6 Participants n=5 Participants	1 Participants n=4 Participants	11 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants	9 Participants n=7 Participants	13 Participants n=5 Participants	8 Participants n=4 Participants	37 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	4 Participants n=5 Participants	1 Participants n=4 Participants	5 Participants n=21 Participants
Race/Ethnicity, Customized American Indian or Alaskan Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	3 Participants n=21 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	2 Participants n=21 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Race/Ethnicity, Customized White	10 Participants n=5 Participants	9 Participants n=7 Participants	11 Participants n=5 Participants	8 Participants n=4 Participants	38 Participants n=21 Participants
Race/Ethnicity, Customized Not Collected or Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	5 Participants n=5 Participants	2 Participants n=4 Participants	7 Participants n=21 Participants
Race/Ethnicity, Customized Other	0 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	0 Participants n=4 Participants	3 Participants n=21 Participants

PRIMARY outcome

Timeframe: 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group

Population: The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD)

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=19 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Percentage of Participants With an Objective Response and Long-term Stable Disease	0 percentage of participants Interval 0.0 to 33.6	11.1 percentage of participants Interval 0.3 to 48.2	10.5 percentage of participants Interval 1.3 to 33.1	0 percentage of participants Interval 0.0 to 33.6

SECONDARY outcome

Timeframe: 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group

Population: The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD)

Objective response rate was defined as the percentage of participants who achieved a complete response (CR) or partial response (PR) within the first 6 cycles of treatment (or within 3 cycles for participants in the DIPG group). Disease assessments were based on MRI and assessed by an independent central review. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=19 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Percentage of Participants Who Achieved an Objective Response (ORR)	0 percentage of participants Interval 0.0 to 33.6	0 percentage of participants Interval 0.0 to 33.6	5.3 percentage of participants Interval 0.1 to 26.0	0 percentage of participants Interval 0.0 to 33.6

SECONDARY outcome

Timeframe: 6 months (first 6 cycles) or 3 months (first 3 cycles) for participants in the DIPG group

Population: The response population consisted of all enrolled participants receiving at least one cycle of pomalidomide if therapy was not discontinued earlier due to progressive disease (PD)

Long-term stable disease (SD) rate was defined as the percentage of participants who achieved SD maintained for ≥ 6 cycles (or \> 3 cycles for DIPG), measured from the date of first dose of treatment. Disease assessments were based on MRI and assessed by an independent central review. SD: A decrease of \< 50% or an increase of \< 25% in the size of measurable lesions and no evidence of new lesions, response does not meet the criteria for CR, PR, or progressive disease, and/or the persistence of non-target lesions with no progression or decrease in size. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions, or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=19 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Percentage of Participants With Long-term Stable Disease	0 percentage of participants Interval 0.0 to 33.6	11.1 percentage of participants Interval 0.3 to 48.2	5.3 percentage of participants Interval 0.1 to 26.0	0 percentage of participants Interval 0.0 to 33.6

SECONDARY outcome

Timeframe: From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months)

Population: All enrolled participants with response (PR or CR), regardless of whether they received any treatment or not

DoR is defined as the time from the date of the first objective response (complete response \[CR\] or partial response \[PR\]) to disease progression. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at the time of start of new anticancer therapy, whichever occurred first. Progressive disease (PD): ≥ 25% increase in the size of the measurable lesions taking as a reference the smallest disease measurement recorded since the start of protocol therapy (nadir), or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions, or if spine MRI and/or lumbar cerebrospinal fluid (CSF) cytology were previously negative and became positive. CR: Disappearance of all lesions and no new lesions. PR: A reduction of ≥ 50% in the size of measurable lesions compared to baseline, and/or the persistence of non-target lesions with no progression or decrease in size.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=1 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=1 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Duration of Response (DoR)	12.29 weeks 95% CI was not estimable due to insufficient number of events	—	NA weeks Median and 95% CI were not estimable due to insufficient number of events	—

SECONDARY outcome

Timeframe: From the first dose of pomalidomide to the date of the first documented tumor progression or death due to any cause, whichever occurs first (Up to 71 months)

Population: The intent-to-treat population includes all enrolled participants, regardless of whether they received any treatment or not

Progression-free survival was defined as the time from the date of first dose of pomalidomide until the date progressive disease (PD) was first observed or until the date of death due to any cause, whichever occurred first. Participants who did not have PD or had not died at the time of analysis were censored at the time of their last disease assessment or at the start of new anticancer therapy, whichever occurred first. Progressive Disease (PD): ≥ 25% increase in the size of the measurable lesions taking as a reference the smallest disease measurement recorded since the start of protocol therapy (nadir), or the appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions, or if spine MRI and/or lumbar CSF cytology were previously negative and became positive.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=11 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=23 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=10 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Kaplan-Meier Estimate of Progression-Free Survival (PFS)	11.43 weeks Interval 4.43 to 12.57	8.43 weeks Interval 5.57 to 16.14	7.86 weeks Interval 5.43 to 8.29	8.29 weeks Interval 7.29 to 18.0

SECONDARY outcome

Timeframe: From the first dose of pomalidomide to the date of death due to any cause (Up to 71 months)

Population: The intent-to-treat population includes all enrolled participants, regardless of whether they received any treatment or not

Overall survival was defined as the time from the date of the first dose to the date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=11 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=23 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=10 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Kaplan-Meier Estimate of Overall Survival (OS)	4.86 months Interval 1.02 to 10.91	12.02 months Interval 2.86 to 20.9	5.06 months Interval 2.04 to 16.66	11.60 months Interval 1.74 to 35.32

SECONDARY outcome

Timeframe: From the first dose of pomalidomide until 28 days after the last dose (Up to approximately 72 months)

Population: The safety population included all participants who received at least 1 dose of study drug

Treatment-emergent adverse events were defined as any adverse events (AE) occurring from the first dose of pomalidomide until 28 days after the last dose. The severity of each AE was graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.03 and according to the following scale: Grade 1: Mild (transient or mild discomfort; no limitation in activity or medical intervention required); Grade 2: Moderate (mild to moderate limitation in activity, assistance may be needed; minimal medical intervention required); Grade 3: Severe (marked limitation in activity, assistance and medical intervention required, hospitalization possible); Grade 4: Life-threatening (extreme limitation in activity, significant assistance or medical intervention required, hospitalization or hospice care probable); Grade 5: Death. Drug-related AEs are those suspected by the Investigator as being related to administration of study drug.

Outcome measures

Outcome measures
Measure	Diffuse Intrinsic Pontine Glioma n=11 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Ependymoma n=9 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	High-grade Glioma n=22 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.	Medulloblastoma n=10 Participants Participants received 2.6 mg/m²/day oral pomalidomide on days 1 to 21 of each 28-day treatment cycle for up to 24 cycles or until disease progression, withdrawal of consent/assent, treatment became intolerable, or death, whichever occurred first.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Serious TEAE related to study drug	1 Participants	0 Participants	6 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Grade 3/4 TEAE	8 Participants	6 Participants	14 Participants	6 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Grade 3/4 TEAE related to study drug	3 Participants	2 Participants	10 Participants	4 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE leading to study drug discontinuation	2 Participants	1 Participants	2 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Any treatment-emergent adverse event (TEAE)	11 Participants	8 Participants	21 Participants	9 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE related to study drug	5 Participants	7 Participants	14 Participants	8 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Serious TEAE	9 Participants	4 Participants	14 Participants	4 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE leading to death	5 Participants	1 Participants	3 Participants	1 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE leading to dose reduction	1 Participants	0 Participants	3 Participants	0 Participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE leading to dose interruption	4 Participants	3 Participants	5 Participants	2 Participants

Adverse Events

Diffuse Intrinsic Pontine Glioma

Serious events: 9 serious events

Other events: 11 other events

Deaths: 11 deaths

Ependymoma

Serious events: 4 serious events

Other events: 8 other events

Deaths: 6 deaths

High-grade Glioma

Serious events: 14 serious events

Other events: 20 other events

Deaths: 15 deaths

Medulloblastoma

Serious events: 4 serious events

Other events: 9 other events

Deaths: 9 deaths

Serious adverse events

Other adverse events

Additional Information

Bristol-Myers Squibb Study Director

Bristol-Myers Squibb

Phone: Please Email

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
Publication restrictions are in place

Restriction type: OTHER