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Bending the Curve Trial by Trial
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Integrated analysis of single-agent ONC201 (dordaviprone) in recurrent H3 K27M-mutant diffuse midline glioma

N = 50NCT03416530NCT03295396NCT05392374NCT03134131NCT02525692

Population
Patients with recurrent H3 K27M-mutant diffuse midline glioma: 50 patients (4 paediatric, 46 adult)
Design
Integrated analysis of patients who received oral ONC201 monotherapy in five studies (per the full text: three clinical trials, an expanded access programme and a single-patient compassionate use programme; the abstract says four clinical trials or one expanded access protocol); eligible patients had measurable disease by Response Assessment in Neuro-Oncology high-grade glioma (RANO-HGG) criteria, performance score 60 or higher, and were 90 days or more from radiation; pontine and spinal tumours were ineligible; primary endpoint overall response rate by RANO-HGG criteria, assessed by dual-reader blinded independent central review
Primary endpoint
ORR
Arms
  • ONC201 monotherapy (experimental, n = 50): Oral ONC201 (dordaviprone) monotherapy; dose not given in the abstract
People
Arrillaga-Romany I
Show the numbers
Results reported for Integrated analysis of single-agent ONC201 (dordaviprone) in recurrent H3 K27M-mutant diffuse midline glioma
EndpointAnalysisONC201 monotherapyControlEffect
ORRIntegrated-analysis population (n=50)Overall response rate by RANO high-grade glioma criteria, blinded independent central reviewprimaryArrillaga-Romany 202420% (10.0–33.7%)—Median time to response 8.3 months (range 1.9 to 15.9).
ORRIntegrated-analysis population (n=50)Overall response rate by combined RANO-HGG and RANO low-grade glioma criteria (secondary endpoint)exploratoryArrillaga-Romany 202430% (17.9–44.6%)——
DORIntegrated-analysis population (n=50)Duration of response (secondary endpoint)primaryArrillaga-Romany 2024median 11.2 months—95% CI 3.8 months to not reached; the upper limit cannot be recorded as a number.
Results reported for Integrated analysis of single-agent ONC201 (dordaviprone) in recurrent H3 K27M-mutant diffuse midline glioma
EndpointAnalysisONC201 monotherapyControlEffect
ORRIntegrated-analysis population (n=50)Overall response rate by RANO high-grade glioma criteria, blinded independent central reviewprimaryArrillaga-Romany 202420% (10.0–33.7%)—Median time to response 8.3 months (range 1.9 to 15.9).
ORRIntegrated-analysis population (n=50)Overall response rate by combined RANO-HGG and RANO low-grade glioma criteria (secondary endpoint)exploratoryArrillaga-Romany 202430% (17.9–44.6%)——
DORIntegrated-analysis population (n=50)Duration of response (secondary endpoint)primaryArrillaga-Romany 2024median 11.2 months—95% CI 3.8 months to not reached; the upper limit cannot be recorded as a number.
Illustrative — modelled from reported values, not trial data. DOR, primary.
0%25%50%75%100%0510152025Months (modelled)
  • ONC201 monotherapy (experimental)

Median-only curves assume a constant hazard (exponential) and fit plateaus, as seen with some immunotherapies, poorly.

Data table (modelled values)
MonthsONC201 monotherapy
0100.0%
764.8%
1442.0%
2127.3%
2817.7%
Primary publication: Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, et al. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024;42(13):1542-1552. PubMedDOI

Appears in

Sources

  1. Arrillaga-Romany I, Gardner SL, Odia Y, Aguilera D, Allen JE, Batchelor T, et al. ONC201 (Dordaviprone) in Recurrent H3 K27M-Mutant Diffuse Midline Glioma. J Clin Oncol. 2024;42(13):1542-1552.

    PubMedDOI