Trial results for the Phase 1/2 study (NCT04187404) investigating the combination of Nivolumab with the novel therapeutic vaccine EO2401 in metastatic adrenocortical carcinoma, pheochromocytoma, and paraganglioma were posted on ClinicalTrials.gov on 2026-01-30. The study, which was terminated, reported safety data, including counts of treatment-emergent serious and non-serious adverse events across various cohorts.
Background
The study, titled "A Novel Therapeutic Vaccine (EO2401) in Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/Paraganglioma," was designed as a multicenter, First-In-Human study. It aimed to assess the safety, tolerability, immunogenicity, and preliminary efficacy of EO2401 in these specific metastatic neuroendocrine tumors. Nivolumab is an established immunotherapy, often used in various cancer types.
Trial design
The Phase 1/2 study (NCT04187404) was a multicenter, First-In-Human trial with an enrollment of 70 participants. The trial investigated the combination of EO2401 and Nivolumab in patients with Metastatic Adrenocortical Carcinoma, Malignant Pheochromocytoma, or Paraganglioma. The overall status of the study is TERMINATED. The trial's primary objectives included assessing safety, tolerability, immunogenicity, and preliminary efficacy.
Key results
The trial results focused on the assessment of treatment-emergent adverse events. The reported data includes the count of participants experiencing serious and non-serious adverse events across different study cohorts:
- For "Treatment-Emergent Serious Adverse Events Assessment":
- Cohort 2A (Non-randomized): 5 participants
- Randomized Extension of Cohort 2A (3 Arms): C2A-I: 4 participants
- Randomized Extension of Cohort 2A (3 Arms): C2A-II: 1 participant
- Randomized Extension of Cohort 2A (3 Arms): C2A-III: 0 participants
- Cohort 2B (Non-randomized): 1 participant
- Cohort 3A (Non-randomized): 4 participants
- Cohort 3B (Non-randomized): 2 participants
- For "Treatment-Emergent Non-Serious Adverse Events":
- Cohort 2A (Non-randomized): 26 participants
- Randomized Extension of Cohort 2A (3 Arms): C2A-I: 13 participants
- Randomized Extension of Cohort 2A (3 Arms): C2A-II: 2 participants
- Randomized Extension of Cohort 2A (3 Arms): C2A-III: 4 participants
- Cohort 2B (Non-randomized): 7 participants
What this means
The posting of these trial results provides safety data for the combination of Nivolumab and EO2401 in patients with metastatic adrenocortical carcinoma, pheochromocytoma, and paraganglioma. The trial's termination indicates that further development of this specific combination in this context may not be proceeding as initially planned. The detailed counts of treatment-emergent serious and non-serious adverse events offer insights into the safety profile observed during the study, which is crucial for understanding the tolerability of the investigational vaccine EO2401 when combined with Nivolumab.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04187404, titled "A Novel Therapeutic Vaccine (EO2401) in Metastatic Adrenocortical Carcinoma, or Malignant Pheochromocytoma/Paraganglioma," were posted on 2026-01-30 on clinicaltrials.gov.