Table of Contents
- Clinical trials overview
- Trials in MDS and AML after prior treatment failure
- Trial in myelofibrosis after JAK-inhibitor treatment
- Trial in transfusion-dependent low-risk MDS
- Main endpoints and what they mean
- Who the trials are for
Clinical trials overview
The available studies on Imetelstat Sodium are testing it in people with blood cancers and related bone marrow disorders.[1][2][3]
The trial set includes one withdrawn Phase 2 study, two authorised Phase 2 studies with the same MDS/AML focus, one authorised Phase 3 study in myelofibrosis, and one authorised Phase 4 study in lower-risk MDS.[1][2][3][4]
Trials in MDS and AML after prior treatment failure
Two Phase 2 studies evaluate efficacy and safety in patients with AML or MDS who are failing or refractory to hypomethylating agent (HMA)-based treatment.[1][2]
These studies are interventional, which means participants receive study treatment rather than only being observed.[1][2]
Both trials plan to enroll 46 participants and use a primary outcome of overall response rate after 4 months of treatment.[1][2]
The response is measured using combined criteria for MDS and AML, based on IWG 2018 criteria for MDS and European LeukemiaNet criteria for AML.[1][2]
One of these Phase 2 studies is withdrawn, while the other is authorised.[1][2]
Trial in myelofibrosis after JAK-inhibitor treatment
The Phase 3 study in myelofibrosis compares Imetelstat Sodium with best available therapy (BAT) in people with intermediate-2 or high-risk disease that is relapsed or refractory to JAK-inhibitor treatment.[3]
This study is larger than the Phase 2 trials and plans to enroll 354 participants.[3]
The main endpoint is overall survival, which means the time from randomization until death from any cause.[3]
This endpoint helps show whether one treatment helps people live longer than the other treatment strategy.[3]
Trial in transfusion-dependent low-risk MDS
The Phase 4 study looks at subjects with IPSS low or intermediate-1 risk MDS who are transfusion dependent and relapsed or refractory to ESA treatment.[4]
This study includes a comparison with placebo in one part of the trial and also has an extension phase for longer follow-up.[4]
The trial plans to enroll 224 participants.[4]
The main efficacy endpoint is red blood cell transfusion independence lasting at least 8 weeks, meaning no red blood cell transfusions during a continuous 8-week period.[4]
The extension phase also looks at long-term safety, overall survival, and disease progression, including progression to AML.[4]
Main endpoints and what they mean
Overall response rate is the percentage of people whose disease improves based on study rules.[1][2]
Overall survival measures how long participants live after starting the study treatment.[3]
Red blood cell transfusion independence measures time without needing red blood cell transfusions, which is important for people with transfusion-dependent MDS.[4]
Some studies also track long-term safety and disease progression, which helps researchers understand whether the treatment may help control the disease over time.[4]
Who the trials are for
The trial populations are adults with specific blood cancers or bone marrow disorders, especially those whose disease has not responded to earlier treatment.[1][2][3][4]
Some studies focus on people with AML or MDS after HMA-based treatment failure, while others focus on myelofibrosis after JAK-inhibitor treatment or on transfusion-dependent lower-risk MDS after ESA treatment.[1][2][3][4]
These differences show that the trials are not general studies for all patients with blood cancer, but targeted studies for specific groups with unmet treatment needs.[1][2][3][4]
