24 Participants Needed

Danvatirsen + Venetoclax for MDS & AML

Recruiting at 1 trial location
AS
Overseen ByAditi Shastri
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Montefiore Medical Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a Phase 1 study investigating the safety and efficacy of Danvatirsen as a monotherapy followed by combination with Venetoclax in patients with relapsed/refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).Funding Source: FDA OOPD

Do I need to stop my current medications to join the trial?

The trial protocol does not specify if you need to stop your current medications. However, you cannot be on any concurrent anticancer treatment or non-permitted medications as noted in the protocol appendix.

What makes the drug Danvatirsen + Venetoclax unique for treating MDS and AML?

Danvatirsen + Venetoclax is unique because it combines Danvatirsen, which is not commonly used for MDS or AML, with Venetoclax, a drug that targets BCL2 proteins to help kill cancer cells. This combination may offer a novel approach compared to standard treatments that typically involve Venetoclax with other agents like hypomethylating agents or low-dose cytarabine.12345

What data supports the effectiveness of the drug combination Danvatirsen and Venetoclax for MDS and AML?

Research shows that Venetoclax, when combined with other drugs, is effective in treating acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), especially in patients who have relapsed or are high-risk. This suggests that combining Venetoclax with other treatments, like Danvatirsen, could potentially be beneficial for these conditions.12467

Who Is on the Research Team?

AS

Aditi Shastri

Principal Investigator

Montefiore Medical Center

Are You a Good Fit for This Trial?

Adults diagnosed with relapsed/refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML), who have tried conventional therapies without success, can join this trial. They must be in relatively good health otherwise, with a decent performance status and proper liver and kidney function. Pregnant women, those with certain other cancers or severe heart issues, active infections, CNS leukemia, or drug abuse cannot participate.

Inclusion Criteria

My AML or MDS has not responded to standard treatments, including at least 6 cycles of specific therapy.
I must have a bone marrow biopsy after joining the trial.
My white blood cell count is below 25,000, possibly after treatment.
See 5 more

Exclusion Criteria

I have been diagnosed with Acute Promyelocytic Leukemia.
I had CNS leukemia but my latest tests show no active disease.
I am not taking any medications that are not allowed in the study.
See 11 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Danvatirsen Monotherapy

Participants receive Danvatirsen as monotherapy in a dose-escalation study to determine the Maximum Tolerated Dose (MTD).

4 weeks per cycle
Weekly infusions

Danvatirsen + Venetoclax Combination

Participants receive a combination of Danvatirsen and Venetoclax to evaluate safety and efficacy.

4 weeks per cycle
Daily oral Venetoclax, weekly Danvatirsen infusions

Follow-up

Participants are monitored for safety and effectiveness after treatment, with response evaluations every 3 months.

Up to 3 years

What Are the Treatments Tested in This Trial?

Interventions

  • Danvatirsen
  • Venetoclax
Trial Overview The study is testing Danvatirsen alone first and then in combination with Venetoclax to see how safe and effective they are for treating MDS/AML that hasn't responded to standard treatments. It's an early-phase trial which means it's one of the first times these drugs are being tested together in humans.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Danvatirsen MonotherapyExperimental Treatment1 Intervention
Group II: Danvatirsen + Venetoclax Combination TherapyExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Montefiore Medical Center

Lead Sponsor

Trials
468
Recruited
599,000+

Flamingo Therapeutics NV

Industry Sponsor

Trials
2
Recruited
110+

M.D. Anderson Cancer Center

Collaborator

Trials
3,107
Recruited
1,813,000+

Published Research Related to This Trial

In a review of 13 studies involving 1059 patients, the combination of venetoclax (VEN) with hypomethylating agents (HMAs) showed a high overall complete remission (CR) rate of 62% for newly diagnosed acute myeloid leukemia (ND-AML) and 61% for myelodysplastic syndrome (MDS), indicating its efficacy in these conditions.
However, the treatment was associated with significant adverse effects, particularly grade 3-4 events like cytopenia and infections, highlighting the need for careful monitoring of patients for potential complications.
The efficacy and adverse events of venetoclax in combination with hypomethylating agents treatment for patients with acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis.Liu, B., Guo, Y., Deng, L., et al.[2021]
In a study of 60 patients with high-risk MDS and AML, venetoclax demonstrated a 35% overall response rate, indicating its efficacy as a treatment option for these conditions.
However, the treatment was associated with significant hematologic toxicity, with 86.7% of patients experiencing grade 2 or higher side effects, and infections, particularly pneumonia, being the most common nonhematologic complication.
A Real-life Turkish Experience of Venetoclax Treatment in High-risk Myelodysplastic Syndrome and Acute Myeloid Leukemia.Gemici, A., Ozkalemkas, F., Dogu, MH., et al.[2022]
In a meta-analysis of 10 studies involving 243 patients, Venetoclax combined with hypomethylating agents (HMAs) showed a 32% complete response rate and an overall response rate of 48% for relapsed acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) after transplantation.
The treatment demonstrated a 6-month survival rate of 42% and a 1-year survival rate of 23%, indicating a moderate benefit for patients post-transplantation, suggesting that this combination therapy could be a viable option for managing relapse in AML/MDS.
Efficacy and safety of venetoclax combined with hypomethylating agents for relapse of acute myeloid leukemia and myelodysplastic syndrome post allogeneic hematopoietic stem cell transplantation: a systematic review and meta-analysis.Du, Y., Li, C., Zhao, Z., et al.[2023]

Citations

The efficacy and adverse events of venetoclax in combination with hypomethylating agents treatment for patients with acute myeloid leukemia and myelodysplastic syndrome: a systematic review and meta-analysis. [2021]
A Real-life Turkish Experience of Venetoclax Treatment in High-risk Myelodysplastic Syndrome and Acute Myeloid Leukemia. [2022]
Efficacy and safety of venetoclax combined with hypomethylating agents for relapse of acute myeloid leukemia and myelodysplastic syndrome post allogeneic hematopoietic stem cell transplantation: a systematic review and meta-analysis. [2023]
The efficacy and safety of venetoclax and azacytidine combination treatment in patients with acute myeloid leukemia and myelodysplastic syndrome: systematic review and meta-analysis. [2023]
A retrospective comparison of salvage intensive chemotherapy versus venetoclax-combined regimen in patients with relapsed/refractory acute myeloid leukemia (AML). [2022]
Outpatient initiation of venetoclax in patients with acute myeloid leukemia. [2023]
Venetoclax combination therapy in acute myeloid leukemia and myelodysplastic syndromes. [2023]
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