Trial results for the Phase 1 study (NCT03358719) investigating nivolumab in combination with DEC-205/NY-ESO-1 fusion protein CDX-1401, poly ICLC, and decitabine for myelodysplastic syndrome or acute myeloid leukemia were posted on ClinicalTrials.gov on 2026-02-27. The study reported 0.0% dose-limiting toxicities among participants.
Background
Nivolumab is an immunotherapy drug. This study investigated its use in combination with other agents for patients with myelodysplastic syndrome or acute myeloid leukemia. The trial explored a regimen including DEC-205/NY-ESO-1 fusion protein CDX-1401, a vaccine designed to target cancer cells, poly ICLC to stimulate the immune system, and the chemotherapy drug decitabine.
Trial design
The Phase 1 study (NCT03358719) was a completed trial that enrolled 8 participants. It investigated the combination of DEC-205/NY-ESO-1 fusion protein CDX-1401, poly ICLC, decitabine, and nivolumab in patients diagnosed with myelodysplastic syndrome, acute myeloid leukemia, chronic myelomonocytic leukemia, or high risk myelodysplastic syndrome, including those with blasts 30 percent or less of bone marrow nucleated cells. The trial aimed to study the side effects of this multi-agent regimen.
Key results
The trial's key measurements focused on safety and biological responses. For the outcome of 'Proportion or Participants Experiencing a Dose-limiting Toxicity', the study reported 0.0 percentage of participants in the treatment group (CDX-1401, Poly ICLC, Decitabine, Nivolumab).
Regarding 'Immune Cell Profile' measurements, various percentages of CD45⁺ leukocytes were observed, including a mean of 0.0030201667 percentage of CD45⁺ leukocytes (Standard Deviation: 0.0050996289), a mean of 0.152785 percentage of CD45⁺ leukocytes (Standard Deviation: 0.1900236337), and a mean of 0.1246616667 percentage of CD45⁺ leukocytes (Standard Deviation: 0.1795932594). Other immune cell profile measurements included a mean of 0.0038176667 percentage of CD45⁺ leukocytes (Standard Deviation: 0.0054418615), a mean of 0.1421666667 percentage of CD45⁺ leukocytes (Standard Deviation: 0.1900236337), and a mean of 0.0837116667 percentage of CD45⁺ leukocytes (Standard Deviation: 0.1795932594).
Measurements for 'Peripheral Blood and Bone Marrow Cells Responses' included mean percentages of methylated cytosines: 84.5 percentage of methylated cytosines (Standard Deviation: 4.9), 81 percentage of methylated cytosines (Standard Deviation: 7.8), 70.2 percentage of methylated cytosines (Standard Deviation: 9.3), 73.5 percentage of methylated cytosines (Standard Deviation: 0.71), and 76.6 percentage of methylated cytosines (Standard Deviation: 6.8).
What this means
The finding of 0.0% dose-limiting toxicities in this Phase 1 study suggests that the combination of nivolumab with DEC-205/NY-ESO-1 fusion protein CDX-1401, poly ICLC, and decitabine was tolerable in the small cohort of patients with myelodysplastic syndrome or acute myeloid leukemia. While this trial primarily assessed safety and initial biological responses, the absence of dose-limiting toxicities supports further investigation into the efficacy of this multi-agent regimen in larger studies for these challenging hematologic malignancies. The immune cell and methylation response data provide preliminary insights into the biological activity of the treatment.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT03358719, titled 'DEC-205/NY-ESO-1 Fusion Protein CDX-1401, Poly ICLC, Decitabine, and Nivolumab in Treating Patients With Myelodysplastic Syndrome or Acute M', were posted on 2026-02-27 on clinicaltrials.gov.