Trial Outcomes & Findings for Veliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations (NCT NCT03581292)

Last Updated: 2025-11-13

Results Overview

Compare event free survival to historical data for each stratum. Analysis will be based on a 2-sample, 1 sided logrank test. EFS is measured from time of enrollment to the date of first documented progression, second malignancy, or date of death from any cause, whichever comes first. Subjects who do not have any event will be censored at the last follow-up date.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

38 participants

Primary outcome timeframe

Up to 2 years

Results posted on

2025-11-13

Participant Flow

Participant milestones

Participant milestones
Measure	Stratum 1 Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Overall Study STARTED	24	14
Overall Study COMPLETED	11	9
Overall Study NOT COMPLETED	13	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Stratum 1 Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Overall Study Physician Decision	9	5
Overall Study Withdrawal by Subject	3	0
Overall Study Withdrawal before Therapy Start	1	0

Baseline Characteristics

Veliparib, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Malignant Glioma Without H3 K27M or BRAFV600 Mutations

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stratum 1 n=24 Participants Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 n=14 Participants Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation	Total n=38 Participants Total of all reporting groups
Age, Categorical <=18 years	23 Participants n=10 Participants	6 Participants n=10 Participants	29 Participants n=20 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=10 Participants	8 Participants n=10 Participants	9 Participants n=20 Participants
Age, Categorical >=65 years	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants
Age, Continuous	12.2 years STANDARD_DEVIATION 4 • n=10 Participants	18.4 years STANDARD_DEVIATION 2.9 • n=10 Participants	14.5 years STANDARD_DEVIATION 4.7 • n=20 Participants
Sex: Female, Male Female	4 Participants n=10 Participants	8 Participants n=10 Participants	12 Participants n=20 Participants
Sex: Female, Male Male	20 Participants n=10 Participants	6 Participants n=10 Participants	26 Participants n=20 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=10 Participants	2 Participants n=10 Participants	3 Participants n=20 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=10 Participants	12 Participants n=10 Participants	31 Participants n=20 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=10 Participants	0 Participants n=10 Participants	4 Participants n=20 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Asian	2 Participants n=10 Participants	0 Participants n=10 Participants	2 Participants n=20 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants
Race (NIH/OMB) Black or African American	0 Participants n=10 Participants	0 Participants n=10 Participants	0 Participants n=20 Participants
Race (NIH/OMB) White	16 Participants n=10 Participants	13 Participants n=10 Participants	29 Participants n=20 Participants
Race (NIH/OMB) More than one race	2 Participants n=10 Participants	0 Participants n=10 Participants	2 Participants n=20 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=10 Participants	1 Participants n=10 Participants	5 Participants n=20 Participants
Region of Enrollment United States	24 participants n=10 Participants	14 participants n=10 Participants	38 participants n=20 Participants

PRIMARY outcome

Timeframe: Up to 2 years

Population: Participants who received study therapy will be included in the analysis.

Outcome measures

Outcome measures
Measure	Stratum 1 n=23 Participants Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 n=14 Participants Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Event Free Survival (EFS)	0.9857 probability	0.9571 probability

SECONDARY outcome

Timeframe: Anytime during treatment (up to 1 year from enrollment)

Population: Participants who had measurable disease at baseline and received study therapy will be included in the analysis.

Estimate the objective response rate in patients with measurable disease. The 95% confidence interval will be calculated by Clopper-Pearson method. Objective responses are defined as patients who achieved complete response or partial response anytime during treatment. Tumor response criteria are determined by changes in size using the longest tumor dimension, and its perpendicular. Either T1, T2 weighted/FLAIR images are used - whichever gives the best estimate of tumor size. Complete Response (CR): Disappearance of all target lesions; Partial response (PR): \>=50% decrease in the sum of the products of the two perpendicular diameters of target lesions, compared to baseline measurement. Objective Response = CR + PR.

Outcome measures

Outcome measures
Measure	Stratum 1 n=11 Participants Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 n=7 Participants Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Objective Response	9.1 percentage of participants Interval 0.2 to 41.3	14.3 percentage of participants Interval 0.4 to 57.9

SECONDARY outcome

Timeframe: Up to 2 years

Population: Participants who received study therapy will be included in the analysis.

The Kaplan-Meier method will be used to estimate the 2-year overall survival for each stratum. OS is measured from time of enrollment to the date of death from any cause. Subjects who do not have any event will be censored at the last follow-up date.

Outcome measures

Outcome measures
Measure	Stratum 1 n=23 Participants Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 n=14 Participants Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Overall Survival (OS)	0.27 survival probability of patients Interval 0.11 to 0.46	0.85 survival probability of patients Interval 0.52 to 0.96

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 5.5 years

Will provide a frequency table summarizing the number of patients with each aberration/alteration detected in germline and/or tumor samples. For longitudinal plasma samples used to assess circulating tumor deoxyribonucleic acid, will summarize the percentage of patients with samples as well as display/summarize any changes in molecular markers. When feasible we will explore the association of these aberrations with EFS/OS and objective response rates via Cox models and fisher exact tests, respectively. Will also explore associations between genetic variants and clinical/demographic variables including age, resection status, histology, etc. For analyses exploring associations of a large number of potential markers with clinical outcome, will utilize false discovery rate approaches in order to control family-wise error rate.

Outcome measures

Outcome data not reported

Adverse Events

Stratum 1

Serious events: 22 serious events

Other events: 4 other events

Deaths: 18 deaths

Stratum 2

Serious events: 5 serious events

Other events: 4 other events

Deaths: 3 deaths

Serious adverse events

Other adverse events

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 16264470064

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Stratum 1 n=23 participants at risk Pediatric newly-diagnosed HGG patients who are wild-type with respect to H3 K27M, BRAF, and IDH1/2	Stratum 2 n=14 participants at risk Pediatric newly-diagnosed HGG patients who are positive for either IDH1/2 mutation
Gastrointestinal disorders Diarrhea	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Gastrointestinal disorders Nausea	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Gastrointestinal disorders Vomiting	13.0% 3/23 • Number of events 4 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
General disorders Fatigue	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	7.1% 1/14 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Infections and infestations Covid-19	0.00% 0/23 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	7.1% 1/14 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Injury, poisoning and procedural complications Dermatitis radiation	4.3% 1/23 • Number of events 2 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Investigations Lymphocyte count decreased	8.7% 2/23 • Number of events 3 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Investigations Neutrophil count decreased	8.7% 2/23 • Number of events 2 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Investigations White blood cell decreased	8.7% 2/23 • Number of events 2 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Metabolism and nutrition disorders Hypocalcemia	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Nervous system disorders Central nervous system necrosis	0.00% 0/23 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	7.1% 1/14 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Nervous system disorders Edema cerebral	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Nervous system disorders Headache	4.3% 1/23 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	0.00% 0/14 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Nervous system disorders Presyncope	0.00% 0/23 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	7.1% 1/14 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.
Skin and subcutaneous tissue disorders Alopecia	4.3% 1/23 • Number of events 2 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.	7.1% 1/14 • Number of events 1 • Enrollment up to 3 years after enrollment Adverse event reporting is collected routinely using case report forms. The SAE table reflects NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted by the institution via expedited reporting (NCI AdEERs / CAeRs). All remaining CTCAEs collected by means other than expedited reporting are non-serious and are reported in the "AE Other" table. All-Cause Mortality includes all deaths collected on the study. Ineligible patients are excluded from reporting of adverse events.