24 Participants Needed

BXCL701 for AML or MDS

ES
ES
Overseen ByEric S Winter, MD
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

The goal of this research study is to find the safest and most effective dose of the study drug, BXCL701, for the treatment of Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS).The names of the study drugs involved in this study are/is:* BXCL701

Will I have to stop taking my current medications?

The trial does not specify if you must stop all current medications, but it strongly encourages changing drugs that strongly affect CYP3A4 enzymes if possible. You cannot take certain diabetes medications (gliptins) during the trial.

How is the drug BXCL701 different from other treatments for AML or MDS?

BXCL701 is unique because it targets the multidrug resistance gene (MDR1), which is often overexpressed in AML and MDS, making these cancers resistant to standard treatments. By inhibiting MDR1, BXCL701 may help overcome this resistance and improve treatment effectiveness.12345

Who Is on the Research Team?

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Eric S Winter, MD

Principal Investigator

Dana-Farber Cancer Institute

Are You a Good Fit for This Trial?

Adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS), who have specific blood and organ function levels, can join. Those with HIV or hepatitis must be on effective treatment with an undetectable viral load. Participants should not have other active cancers, CNS leukemia involvement, or recent treatments that could interfere.

Inclusion Criteria

I can take care of myself but might not be able to do heavy physical work.
My organs are functioning well, and I understand the risks and consent.
My condition is either relapsed or refractory AML, or I have a type of MDS with excess blasts-2.

Exclusion Criteria

I have been diagnosed with Acute Promyelocytic Leukemia.
My acute myeloid leukemia has spread to my brain or spinal cord.
I am eligible for this trial regardless of my gender or ethnicity.
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BXCL701 in a dose escalation approach, taking the drug 2x daily during each 28-day cycle for up to 12 cycles

12 months
Disease assessment at baseline, cycle 1, 3, 8, 15, 22, and day 1 of every cycle

Follow-up

Participants are monitored for safety and effectiveness after treatment

up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

  • BXCL701
Trial Overview The trial is testing the safety and effectiveness of a drug called BXCL701 for AML/MDS patients. It aims to determine the best dose by monitoring participants' reactions over time.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Dose Escalation BXCL701Experimental Treatment1 Intervention

BXCL701 is already approved in United States for the following indications:

🇺🇸
Approved in United States as BXCL701 for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Eric Stephen Winer, MD

Lead Sponsor

Trials
1
Recruited
20+

Published Research Related to This Trial

The combination of the MDR1 blocker SDZ PSC-833 with mitoxantrone and etoposide showed tolerable antileukemic activity in patients with relapsed acute myelogenous leukemia, although it required a significant dose reduction of these drugs due to toxicity.
Despite the treatment leading to some clearance of leukemia cells in the blood and bone marrow, no patients achieved complete remission, highlighting the need for further research to improve outcomes in this patient population.
Phase I study of mitoxantrone plus etoposide with multidrug blockade by SDZ PSC-833 in relapsed or refractory acute myelogenous leukemia.Kornblau, SM., Estey, E., Madden, T., et al.[2017]
The study evaluated various methods for detecting the MDR1 gene in acute myelogenous leukemia (AML), finding that RT-PCR was more sensitive than Northern blot analysis, but also more prone to contamination from normal cells.
A modified method for estimating total MDR1 mRNA in leukemic cell populations was developed, which could be useful for large clinical trials and revealed that certain leukemia subtypes, like FAB M3, were mostly MDR1 negative.
Methods for detection of MDR1 mRNA expression on acute myelogenous leukemia cells.Sato, H., Oonishi, T., Wada, C.[2018]
In a study of 74 patients with acute myelogenous leukaemia (AML), high levels of MDR1 gene expression were associated with difficulty in achieving remission and shorter remission durations after treatment.
The findings suggest that elevated MDR1 expression may contribute to the ineffectiveness of anthracycline-based treatments in some AML patients, indicating a potential mechanism for treatment resistance.
MDR1 transcript levels as an indication of resistant disease in acute myelogenous leukaemia.Sato, H., Preisler, H., Day, R., et al.[2019]

Citations

Phase I study of mitoxantrone plus etoposide with multidrug blockade by SDZ PSC-833 in relapsed or refractory acute myelogenous leukemia. [2017]
Methods for detection of MDR1 mRNA expression on acute myelogenous leukemia cells. [2018]
MDR1 transcript levels as an indication of resistant disease in acute myelogenous leukaemia. [2019]
Immunophenotyping and cytogenetics in older adults with acute myeloid leukemia: significance of expression of the multidrug resistance gene-1 (MDR1). [2013]
[Analysis of the multidrug resistance MDR1 gene expression in clinical leukemia with RT-PCR]. [2018]
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