A Phase 2 clinical trial published on ClinicalTrials.gov seeks to study a novel cancer drug in patients with indolent systemic mastocytosis (ISM) or myelofibrosis, with data collection expected to conclude soon.
The trial, which is sponsored by Telios Pharmaceuticals and includes several centers across the globe, is studying TL-895, a drug that blocks a class of proteins called receptor tyrosine kinases. Many patients with ISM have mutations in the KIT gene, which codes for one of these tyrosine kinases.
What is a KIT gene mutation?
SM is usually caused by a sporadic mutation in the KIT gene, which codes for a protein called CD117 transmembrane tyrosine kinase. The protein is involved in the growth, survival and migration of mast cells. The most common KIT mutation associated with SM is the D816V mutation, which results in the amino acid aspartic acid being replaced by the amino acid valine in the protein chain.
According to Telios, the study had an estimated primary completion date of June 30, 2025. This is the date when the final participant is examined in order to assess the main study outcome, which in this case is determining an optimal dose of TL-895. The estimated overall study completion date is set for Dec. 31, 2025.
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All patients were randomized to receive either a placebo or TL-895 twice daily for 28 days, in addition to standard supportive care. Participants were given varying doses of the study drug to evaluate the primary outcome. Patients, healthcare providers, investigators and data analysts were all blinded to the participants’ treatment allocation in order to minimize bias.
As a secondary outcome measure, the trial also seeks to investigate whether TL-895 causes changes in patient-reported symptoms after 12 weeks of treatment.
As of May 2024, the investigators expected to enroll approximately 121 participants in the trial. Patients with ISM were required to be at least 18 years of age, have a confirmed diagnosis of ISM and be experiencing moderate to severe symptoms. Patients who had previously received other tyrosine kinase inhibitors such as avapritinib, bezuclastinib and elenestinib were excluded from the study.
Preliminary findings have not yet been published on ClinicalTrials.gov.
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