Results from a Phase 3 trial evaluating tofacitinib for recurrent glioblastoma were posted on 2026-06-29. The study reported a median progression-free survival of 4 months for both patient cohorts and no radiographic response among participants.
Background
The trial investigated tofacitinib as a potential treatment for patients with recurrent glioblastoma.
Trial design
This was a Phase 3 trial (NCT05326464) titled "Tofacitinib in Recurrent GBM Patients". The study enrolled 18 participants with glioblastoma. The trial examined the effects of tofacitinib 10mg, alongside interventions like temozolomide and lomustine, in patients with recurrent glioblastoma.
Key results
The trial reported key measurements for two cohorts: Newly Diagnosed or Recurrent Off Temozolomide and Recurrent Disease on Temozolomide.
- For both the Newly Diagnosed or Recurrent Off Temozolomide group and the Recurrent Disease on Temozolomide group, the median Progression-free Survival (PFS) was 4 months.
- For both groups, the mean Overall Survival (OS) was 13.1 months.
- The percentage of treatment related adverse events was 100% for both groups.
- The radiographic response rate by RANO criteria showed 0 participants with a response for both groups.
What this means
The posted results indicate that tofacitinib, when evaluated in patients with recurrent glioblastoma, did not show a radiographic response, with 0 participants achieving a response by RANO criteria. The median progression-free survival was 4 months, and the mean overall survival was 13.1 months across both study cohorts. A high incidence of treatment-related adverse events was observed, affecting 100% of participants in both groups.
Source
The trial results were posted on 2026-06-29 on ClinicalTrials.gov, an official database of clinical studies. The full details are available on clinicaltrials.gov under the identifier NCT05326464.