Trial · NCT03295396
ONC201 in Adults With Recurrent H3 K27M-mutant Glioma
NCT03295396, Phase 2, Terminated, Jazz Pharmaceuticals; results first posted 2025-07-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. ONC201 in Adults With Recurrent H3 K27M-mutant Glioma. Trials, record NCT03295396. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03295396
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- ONC201 in Adults With Recurrent H3 K27M-mutant Glioma
- NCT number
- NCT03295396
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 73 participants, actual
- Lead sponsor
- Jazz Pharmaceuticals
- Conditions
- Glioma
- Interventions
- Dordaviprone (ONC201)
- First posted
- 2017-09-27
- Last updated
- 2025-07-28
- Results first posted
- 2025-07-28
- Limitations and caveats
The Sponsor terminated this study based on the initiation of a randomized Phase 3 study of dordaviprone (ONC201) in an earlier setting; closing this study ensured enrollment would not compete with the Phase 3 study. The decision was not related to any safety concerns with dordaviprone (ONC201). At the time enrollment was halted, some treatment arms had not completed enrollment.~Note: This study did not assess response using RANO 2.0, as these criteria were established after study initiation.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients With Overall Response | From first dose of study treatment through study completion, an average of 1 year | 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. |
| Secondary | Duration of Response (DOR) | From objective response (complete response or partial response) per RANO to disease progression or death, up to 40.6 months. | 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. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients With Overall Response | From first dose of study treatment through study completion, an average of 1 year | 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. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Duration of Response (DOR) | From objective response (complete response or partial response) per RANO to disease progression or death, up to 40.6 months. | This outcome was analyzed in patients who achieved an objective response (complete response or partial response) per RANO criteria. | months | MEDIAN |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients With Overall Response | Arm A | 5 | Participants | ||
| Number of Patients With Overall Response | Arm B | 5 | Participants | ||
| Duration of Response (DOR) | Arm A | 5.6 | 95% Confidence Interval 1.9 to NA | months | |
| Duration of Response (DOR) | Arm B | 15.0 | 95% Confidence Interval 7.5 to NA | months |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm A | 43 | |
| Overall Study | STARTED | Arm B | 30 | |
| Overall Study | COMPLETED | Arm A | 0 | |
| Overall Study | COMPLETED | Arm B | 0 | |
| Overall Study | NOT COMPLETED | Arm A | 43 | |
| Overall Study | NOT COMPLETED | Arm B | 30 |
Source ClinicalTrials.gov
Adverse events200
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Arm A | 4 | 43 | |
| other | Anaemia | Blood and lymphatic system disorders | Arm B | 1 | 30 | |
| other | Sinus tachycardia | Cardiac disorders | Arm A | 1 | 43 | |
| other | Sinus tachycardia | Cardiac disorders | Arm B | 4 | 30 | |
| other | Diplopia | Eye disorders | Arm A | 2 | 43 | |
| other | Diplopia | Eye disorders | Arm B | 6 | 30 | |
| other | Vision blurred | Eye disorders | Arm A | 3 | 43 | |
| other | Vision blurred | Eye disorders | Arm B | 2 | 30 | |
| other | Tinnitus | Ear and labyrinth disorders | Arm A | 3 | 43 | |
| other | Tinnitus | Ear and labyrinth disorders | Arm B | 0 | 30 |
1 to 10 of 200Source ClinicalTrials.gov
See all 200 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm A | 33 | 43 |
| Total, all-cause mortality | Arm B | 25 | 30 |
| Total, other adverse events | Arm A | 42 | 43 |
| Total, other adverse events | Arm B | 27 | 30 |
| Total, serious adverse events | Arm A | 17 | 43 |
| Total, serious adverse events | Arm B | 7 | 30 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38335473 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Arm A vs Arm B: Number of Patients With Overall Response | Trial comparisons |
| Arm A vs Arm B: deaths from any cause | Trial comparisons |
| Arm A vs Arm B: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03295396
GET https://conductscience.org/api/trials/v1/NCT03295396/events?page=2