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Trial Outcomes & Findings for ONC201 in Adults With Recurrent H3 K27M-mutant Glioma (NCT NCT03295396)

NCT ID: NCT03295396

Last Updated: 2025-07-28

Results Overview

Quantitative thresholds for objective response by Response Assessment in Neuro-Oncology (RANO) criteria for target lesions on radiographic imaging are Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) requires a ≥50% decrease in the sum of products of perpendicular diameters of target lesions from baseline. Other considerations for RANO response include assessments of non-target lesions, corticosteroids, and performance status. The Overall Response Rate is the proportion of patients who achieve either CR or PR. Tumor assessments were conducted at 8 weeks (±1 week) following the initiation of therapy and every 8 weeks (±1 week) thereafter. All patients who received at least one dose of dordaviprone (ONC201) were included in the analysis, including patients with diffuse intrinsic pontine glioma (DIPG) or primary spinal tumors, which are historically not assessable for response using RANO-HGG criteria due to anatomical or imaging limitations.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

73 participants

Primary outcome timeframe

From first dose of study treatment through study completion, an average of 1 year

Results posted on

2025-07-28

Participant Flow

Participant milestones

Participant milestones
Measure
Arm A
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies.
Arm B
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Overall Study
STARTED
43
30
Overall Study
COMPLETED
0
0
Overall Study
NOT COMPLETED
43
30

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

ONC201 in Adults With Recurrent H3 K27M-mutant Glioma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Arm A
n=43 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies.
Arm B
n=30 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Total
n=73 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Age, Categorical
Between 18 and 65 years
40 Participants
n=99 Participants
29 Participants
n=107 Participants
69 Participants
n=206 Participants
Age, Categorical
>=65 years
3 Participants
n=99 Participants
1 Participants
n=107 Participants
4 Participants
n=206 Participants
Age, Continuous
38.6 years
n=99 Participants
35.0 years
n=107 Participants
37.1 years
n=206 Participants
Sex: Female, Male
Female
21 Participants
n=99 Participants
12 Participants
n=107 Participants
33 Participants
n=206 Participants
Sex: Female, Male
Male
22 Participants
n=99 Participants
18 Participants
n=107 Participants
40 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
2 Participants
n=99 Participants
6 Participants
n=107 Participants
8 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
40 Participants
n=99 Participants
23 Participants
n=107 Participants
63 Participants
n=206 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
1 Participants
n=99 Participants
1 Participants
n=107 Participants
2 Participants
n=206 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Asian
4 Participants
n=99 Participants
0 Participants
n=107 Participants
4 Participants
n=206 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Black or African American
6 Participants
n=99 Participants
4 Participants
n=107 Participants
10 Participants
n=206 Participants
Race (NIH/OMB)
White
30 Participants
n=99 Participants
21 Participants
n=107 Participants
51 Participants
n=206 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
0 Participants
n=107 Participants
0 Participants
n=206 Participants
Race (NIH/OMB)
Unknown or Not Reported
3 Participants
n=99 Participants
5 Participants
n=107 Participants
8 Participants
n=206 Participants
Primary Tumor Location/Diagnosis
Brainstem (excluding DIPG)
7 Participants
n=99 Participants
3 Participants
n=107 Participants
10 Participants
n=206 Participants
Primary Tumor Location/Diagnosis
Midline (excluding brainstem, DIPG, and spinal)
26 Participants
n=99 Participants
20 Participants
n=107 Participants
46 Participants
n=206 Participants
Primary Tumor Location/Diagnosis
Non-midline
1 Participants
n=99 Participants
7 Participants
n=107 Participants
8 Participants
n=206 Participants
Primary Tumor Location/Diagnosis
DIPG
2 Participants
n=99 Participants
0 Participants
n=107 Participants
2 Participants
n=206 Participants
Primary Tumor Location/Diagnosis
Spinal
7 Participants
n=99 Participants
0 Participants
n=107 Participants
7 Participants
n=206 Participants

PRIMARY outcome

Timeframe: From first dose of study treatment through study completion, an average of 1 year

Population: Note: A total of 9 (21%) patients in Arm A, including 7 with primary spinal tumors and 2 with DIPG, were included in the overall analysis (i.e. Overall Response Rate) despite not being assessable for response per RANO-HGG.

Quantitative thresholds for objective response by Response Assessment in Neuro-Oncology (RANO) criteria for target lesions on radiographic imaging are Complete Response (CR) is the disappearance of all target lesions; Partial Response (PR) requires a ≥50% decrease in the sum of products of perpendicular diameters of target lesions from baseline. Other considerations for RANO response include assessments of non-target lesions, corticosteroids, and performance status. The Overall Response Rate is the proportion of patients who achieve either CR or PR. Tumor assessments were conducted at 8 weeks (±1 week) following the initiation of therapy and every 8 weeks (±1 week) thereafter. All patients who received at least one dose of dordaviprone (ONC201) were included in the analysis, including patients with diffuse intrinsic pontine glioma (DIPG) or primary spinal tumors, which are historically not assessable for response using RANO-HGG criteria due to anatomical or imaging limitations.

Outcome measures

Outcome measures
Measure
Arm A
n=43 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies. Notably, patients with DIPG and primary spinal tumors are historically not evaluable for response using RANO-HGG criteria.
Arm B
n=30 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Number of Patients With Overall Response
5 Participants
5 Participants

SECONDARY outcome

Timeframe: From objective response (complete response or partial response) per RANO to disease progression or death, up to 40.6 months.

Population: This outcome was analyzed in patients who achieved an objective response (complete response or partial response) per RANO criteria.

DOR is defined as the time from the first documented response to the earliest date of disease progression or death, whichever occurred first. Patients who had not progressed or died at the time of analysis were censored at their last adequate tumor assessment.

Outcome measures

Outcome measures
Measure
Arm A
n=5 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies. Notably, patients with DIPG and primary spinal tumors are historically not evaluable for response using RANO-HGG criteria.
Arm B
n=5 Participants
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Duration of Response (DOR)
5.6 months
Interval 1.9 to
Not calculable. The statistical procedure did not have sufficient information (insufficient number of patients with events) in order to calculate the upper bound of the 95% confidence interval (CI).
15.0 months
Interval 7.5 to
Not calculable. The statistical procedure did not have sufficient information (insufficient number of patients with events) in order to calculate the upper bound of the 95% confidence interval (CI).

Adverse Events

Arm A

Serious events: 17 serious events
Other events: 42 other events
Deaths: 33 deaths

Arm B

Serious events: 7 serious events
Other events: 27 other events
Deaths: 25 deaths

Serious adverse events

Serious adverse events
Measure
Arm A
n=43 participants at risk
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies.
Arm B
n=30 participants at risk
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Nervous system disorders
Seizure
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Depressed level of consciousness
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Dizziness
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Cardiac disorders
Sinus tachycardia
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Eye disorders
Eye pain
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Vomiting
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Abdominal pain
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Dysphagia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Nausea
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Death
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Gait disturbance
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Pyrexia
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
Pneumonia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
Sepsis
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
Urinary tract infection
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Injury, poisoning and procedural complications
Fall
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Injury, poisoning and procedural complications
Rib fracture
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hyperkalaemia
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hypokalaemia
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hypophosphataemia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Musculoskeletal and connective tissue disorders
Muscular weakness
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Encephalopathy
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Hydrocephalus
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Brain oedema
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Headache
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Dysarthria
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Haemorrhage intracranial
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Peripheral sensory neuropathy
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Tremor
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Psychiatric disorders
Agitation
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Renal and urinary disorders
Urinary retention
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Dyspnoea
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Respiratory distress
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Hypoxia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Pneumothorax
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Vascular disorders
Embolism
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Vascular disorders
Hypertension
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months

Other adverse events

Other adverse events
Measure
Arm A
n=43 participants at risk
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm A included patients with recurrent H3 K27M-mutant glioma including those with diffuse intrinsic pontine glioma (DIPG), primary spinal tumors, and some atypical histologies.
Arm B
n=30 participants at risk
Patients received 625 mg dordaviprone (ONC201) once weekly. Arm B included patients with recurrent H3 K27M-mutant glioma, but excluded patients with the following: * Primary malignant lesion located in the pons or spinal cord. * Atypical non-astrocytic histologies such as ependymoma, ganglioma and pleomorphic xanthoastrocytoma, or pilocytic astrocytoma and subependymal giant cell astrocytoma (SEGA). * Prior bevacizumab treatment of \>4 doses of \>7.5 mg/kg
Blood and lymphatic system disorders
Anaemia
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Cardiac disorders
Sinus tachycardia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Eye disorders
Diplopia
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
20.0%
6/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Eye disorders
Vision blurred
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Ear and labyrinth disorders
Tinnitus
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Nausea
30.2%
13/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
16.7%
5/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Vomiting
18.6%
8/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Dysphagia
11.6%
5/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Diarrhoea
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Abdominal pain
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Gastrointestinal disorders
Constipation
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Fatigue
32.6%
14/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
43.3%
13/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Gait disturbance
23.3%
10/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
20.0%
6/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Oedema peripheral
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
16.7%
5/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Pyrexia
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Asthenia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
General disorders
Non-cardiac chest pain
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
Urinary tract infection
14.0%
6/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
COVID-19
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Infections and infestations
Pneumonia
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Injury, poisoning and procedural complications
Fall
20.9%
9/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
20.0%
6/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Alanine aminotransferase increased
14.0%
6/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Aspartate aminotransferase increased
14.0%
6/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Lymphocyte count decreased
18.6%
8/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Platelet count decreased
11.6%
5/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Weight increased
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
White blood cell count decreased
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Blood bilirubin increased
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Investigations
Blood glucose increased
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hypokalaemia
14.0%
6/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hyponatraemia
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Decreased appetite
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hyperglycaemia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hypocalcaemia
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hyperkalaemia
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Musculoskeletal and connective tissue disorders
Muscular weakness
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
20.0%
6/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Musculoskeletal and connective tissue disorders
Back pain
2.3%
1/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Headache
23.3%
10/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
23.3%
7/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Dizziness
14.0%
6/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Hemiparesis
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Dysarthria
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Encephalopathy
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Memory impairment
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Paraesthesia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Tremor
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
13.3%
4/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Aphasia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
3.3%
1/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Hydrocephalus
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Depressed level of consciousness
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Nervous system disorders
Seizure
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Psychiatric disorders
Confusional state
11.6%
5/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Psychiatric disorders
Insomnia
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Renal and urinary disorders
Urinary incontinence
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Dyspnoea
9.3%
4/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Cough
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Respiratory, thoracic and mediastinal disorders
Hypoxia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Skin and subcutaneous tissue disorders
Rash maculo-papular
11.6%
5/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Skin and subcutaneous tissue disorders
Dry skin
4.7%
2/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Vascular disorders
Hypertension
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
10.0%
3/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Endocrine disorders
Cushingoid
0.00%
0/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
6.7%
2/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
Metabolism and nutrition disorders
Hypoalbuminaemia
7.0%
3/43 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months
0.00%
0/30 • From the time/date of initiation of study treatment through 30 days following cessation of study treatment (regardless of treatment duration), or for duration of study treatment until initiation of other anticancer therapy, whichever occurred first, up to 51 months

Additional Information

Chief Medical Officer

Chimerix, Inc.

Phone: 919-806-1074

Results disclosure agreements

  • Principal investigator is a sponsor employee Within 12 months of the completion of the Study at all sites, if no publication of the overall multi-center results has been made, institutions are entitled to publish their locally obtained results, provided the Sponsor is given the opportunity to review and comment. Institution publications may be delayed up to an additional 90 days to allow the Sponsor to seek patent protection.
  • Publication restrictions are in place

Restriction type: OTHER