Phase 1 Study of Danvatirsen Alone and Combined With Venetoclax for Patients With Relapsed or Refractory Myelodysplastic Syndromes and Acute Myeloid Leukemia

What this Trial Is About

Researchers are evaluating the safety and effectiveness of Danvatirsen alone and then combined with Venetoclax in patients with relapsed or refractory myelodysplastic syndromes MDS or acute myeloid leukemia AML. This Phase 1 study focuses on patients with intermediate to very high risk MDS or AML who have not responded to frontline therapy, aiming to find a safe and biologically effective dose for future studies. The trial also explores how blocking STAT3, a protein involved in cancer cell survival, affects disease progression and treatment resistance. The study includes two parts one where patients receive Danvatirsen alone and another where Danvatirsen is combined with Venetoclax. Danvatirsen is given as an infusion with increasing doses over 28-day cycles, while Venetoclax is taken orally with a gradual dose increase during the first cycle. Dose levels and safety are carefully monitored to find the maximum tolerated dose. Treatment continues in cycles of about four weeks, with dose adjustments allowed based on side effects. Participants will have regular assessments including bone marrow biopsies and blood tests to monitor response and safety. Evaluations occur between days 21 and 42 of treatment cycles and continue at defined intervals to determine if the therapy is working or if side effects require stopping treatment. After completing the study treatment, patients will be followed every three months for up to three years to track long-term response and survival. Safety monitoring includes frequent checks and follow-up visits if treatment is stopped due to adverse effects.

Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML