A new clinical trial published on ClinicalTrials.gov is evaluating whether combining avapritinib with decitabine can offer better disease management for systemic mastocytosis (SM) with an associated hematologic neoplasm (SM-AHN).
SM-AHN is a complex disease that presents significant treatment challenges. While targeted drugs such as avapritinib help control mastocytosis, the associated blood cancer often continues to worsen. Researchers hope that by addressing both aspects of the disease simultaneously, patients will experience improved outcomes.
This study, listed under ClinicalTrials.gov ID NCT06327685, is focused on determining the safety and effectiveness of this dual-drug approach. It is sponsored by the H. Lee Moffitt Cancer Center and Research Institute.
Avapritinib, an oral tyrosine kinase inhibitor, specifically targets mast cell proliferation, while decitabine, a chemotherapy drug, is designed to treat various blood cancers. However, concerns about potential overlapping toxicities mean that careful monitoring is necessary. This phase I study will assess safety, establish appropriate dosage levels and monitor overall response rates.
To qualify for the trial, patients must meet specific eligibility criteria, including a confirmed diagnosis of SM-AHN according to the World Health Organization’s 2022 classification. Participants must also demonstrate adequate organ function and be able to adhere to study protocols. This study excludes individuals with certain pre-existing conditions, including prior disease progression on either avapritinib or decitabine, severe cardiac issues or recent major surgery.
This trial is designed in two phases: a dose-escalation phase to determine safe dosage levels and a dose-expansion phase to evaluate the drug combination’s broader effects. Patients with higher platelet counts will receive both drugs from the beginning, while those with lower counts will start with decitabine alone before transitioning to the combination treatment. This study will monitor response rates and potential side effects over a 24-month period.
Read more about treatment and care of SM
The ultimate goal is to determine whether this combination therapy can provide better disease control than existing single-drug treatments. If successful, this approach could lead to a more effective treatment option for patients with SM-AHN, a condition that currently has limited options. While risks remain, the potential to improve survival and quality of life makes this study an important step forward in treating this rare and difficult disease.
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