Table of Contents
Trial overview
This clinical research is a Phase 2 study of IADADEMSTAT in adults with essential thrombocythemia, a blood disorder marked by too many platelets.[1] The trial is authorised and is designed to test both whether the study treatment may help and whether it can be used safely in this patient group.[1]
The study is interventional, which means researchers give the treatment to participants and then measure the results.[1] The brief summary says the trial aims to reduce the percentage of adult ET patients with abnormal platelet counts and to evaluate safety and tolerability.[1]
Who can participate
The trial is for adult patients with essential thrombocythemia who are resistant or intolerant to hydroxyurea.[1] Resistant means the usual treatment did not control the disease well enough, and intolerant means the patient could not continue it because of problems or side effects.
This focus helps the study look at a group of patients who still need better treatment options.[1] No other participation details are given in the source data.
What the study measures
The main outcome is the percentage of patients who reach a platelet count of ≤400×10^9/L without later thrombotic events while keeping normal platelet counts during up to 24 weeks of treatment.[1] In simple terms, the study wants to know if platelet levels can be brought down to a safer range and stay there.
The study also measures the occurrence and severity of treatment-emergent adverse events, which are health problems that begin or worsen after treatment starts.[1] These events are graded using the Common Terminology Criteria for Adverse Events, version 5.0, a standard way to describe how serious side effects are.[1]
Trial phase and size
This is a Phase 2 trial, which usually means the study is looking for early signs that the treatment may work while continuing to monitor safety.[1] The planned enrollment is 36 participants, so this is a small study rather than a large confirmatory trial.[1]
Because the study is Phase 2, the results may help decide whether larger studies should be done later.[1] The trial data do not provide results yet, only the research plan.[1]
Safety measures
Safety is one of the main parts of the study, alongside possible benefit.[1] Researchers will track occurrence and severity of adverse events, meaning they will record whether side effects happen and how serious they are.[1]
The trial also looks for thrombotic events, which are blood clots.[1] This is important in essential thrombocythemia because the disease itself can be linked with clotting problems.[1]
