Table of contents
- Trial overview
- Who can participate
- Study phases and design
- What researchers measure
- Trial status and size
Trial overview
The source data includes two authorised clinical trials investigating Siremadlin.[1][2]
One is an open-label, multi-center, Phase 3 rollover protocol designed to collect long-term safety data.[1] The other is a Phase 2 trial in advanced or metastatic cancer that studies treatment matched to the molecular alterations found in a patient’s tumor.[2]
Who can participate
The Phase 3 rollover study is designed to accept patients with varied disease origins, depending on the parent protocol.[1] In simple terms, this means the exact patient group depends on the earlier study that the person came from.
The Phase 2 study includes people with advanced / metastatic cancers.[2] It also focuses on cohorts based on tumor molecular features, so not every cancer type is treated the same way in the study.[2]
Study phases and design
The rollover safety study is a Phase 3 interventional trial and is open label and multi-center.[1] Open label means both the study team and the patient know what treatment is being given, and multi-center means the trial runs at more than one site.[1]
The cancer-matching study is a Phase 2 interventional trial.[2] Phase 2 studies are usually used to see whether a treatment has activity in a specific group and to continue safety monitoring.[2]
What researchers measure
In the Phase 3 rollover study, the main outcome is the frequency and severity of SAEs/AEs.[1] SAE means serious adverse event, and AE means adverse event; both are unwanted medical problems that happen during a study, but serious events are more severe.[1]
The brief summary says this study is meant to evaluate long-term safety data, especially SAEs and AEs.[1]
In the Phase 2 study, the main outcome is the progression free rate after 3 months of treatment.[2] This means researchers check how many patients have not had their cancer get worse after 12 weeks.[2]
The brief summary for the Phase 2 study says the goal is to evaluate the activity of selected study drugs for each cohort based on molecular alterations or characteristics of the patient’s tumor.[2]
Trial status and size
Both trials are listed as Authorised, meaning they have been approved to run.[1][2]
The Phase 3 rollover study lists an enrollment of 139 patients.[1] The Phase 2 study lists an enrollment of 455 patients.[2]
The trial data also shows that the Phase 3 study includes treatments such as Jakavi, HDM201, and Farydak in its intervention list.[1] The Phase 2 study lists HDM201 and several other targeted cancer drugs, including alectinib, ribociclib, trametinib, dabrafenib, regorafenib, avapritinib, and cabozantinib.[2]
