Table of Contents

• Trial overview
• Main conditions studied
• Who can participate
• Trial phases and study design
• Main outcomes measured
• Notable Imatinib studies
• Patient-friendly terms

Trial overview

Across the provided data, Imatinib is being studied in many interventional trials, meaning researchers are giving a planned treatment and measuring the results.^[1] Most studies are in cancer, especially leukemia, but some also test Imatinib in pulmonary arterial hypertension, acute ischemic stroke, and rare cancer settings.^[2][3]

The trials include both comparison studies and add-on studies. In some trials, Imatinib is compared with another treatment, while in others it is used with chemotherapy or other drugs to see whether the combination works better.^[4][5]

Main conditions studied

The most common condition in the data is chronic myeloid leukemia and related forms such as chronic phase CML and chronic myelogenous leukemia.^{[6][7] Several trials also study Philadelphia chromosome-positive acute lymphoblastic leukemia, which is a type of blood cancer with a specific genetic change.[8][9]}

Other conditions include gastrointestinal stromal tumor, pulmonary arterial hypertension, acute ischemic stroke, and primary breast cancer.^{[10][11][5] There is also a rare-disease study that includes severe sickle cell disease with Imatinib used before gene therapy in some participants.[12]}

Who can participate

Eligibility depends on the trial. Some studies are for adults with newly diagnosed disease, such as adults with Ph+ CML-CP or adults with Ph+ ALL, while others include older adults aged 55 and over.^[6][7]

Some trials focus on people who already received Imatinib or another tyrosine kinase inhibitor and did not reach a deep molecular response, while other studies include people in deep molecular remission who may try treatment withdrawal.^{[7][6] The pediatric trial includes infants, children, and young adults, showing that the studied age range can be very broad depending on the protocol.[13]}

Trial phases and study design

The data include Phase 2, Phase 3, Phase 4, and one Phase 1/2 study.^{[1][11][12] Phase 2 trials often look at early signs of benefit and safety, while Phase 3 trials compare treatments in larger groups and measure key outcomes such as response or survival.[4][6]}

Some studies are randomized, meaning participants are assigned by chance to different treatment groups.^{[5][10] One stroke study is randomized, double-blind, and placebo-controlled, which means neither the patient nor the study team knows who receives the active treatment or placebo during the trial.[5]}

Main outcomes measured

The trials measure different results depending on the disease. In leukemia studies, common outcomes include major molecular response, molecular remission, MRD-negative complete remission, disease-free survival, and event-free survival.^[6][8][9][6]

In the gastrointestinal stromal tumor studies, the key outcomes are progression-free survival and recurrence-free survival, which show how long the disease stays controlled or does not return.^{[10][14] In the stroke study, the main outcome is the change in the modified Rankin Scale at 3 months, which measures how much disability a person has after the stroke.[5]}

In pulmonary arterial hypertension, the studies look at changes in pulmonary vascular resistance, 6-minute walk distance, NT-proBNP, time to clinical worsening, and heart ultrasound measurements of right ventricular function.^{[11][15] In the breast cancer study, the main outcome is whether the tumor changes from estrogen receptor-negative to estrogen receptor-positive after Imatinib treatment.[16]}

Notable Imatinib studies

One large Phase 3 study compares asciminib with other tyrosine kinase inhibitors in newly diagnosed Ph+ CML-CP and includes an analysis for the group where Imatinib was the selected treatment before randomization.^[1] This study measures major molecular response at week 48, which is a common marker of how well the leukemia is controlled.^[1]

Several Phase 3 leukemia studies compare Imatinib with ponatinib or with other treatment strategies in Ph+ ALL.^{[8][9][8] These studies focus on molecular response, MRD-negative remission, and event-free or disease-free survival, which are important signs of treatment success.[8][9][8]}

In gastrointestinal stromal tumor, one Phase 3 study tests whether continuing adjuvant Imatinib for 2 more years after 3 years of treatment improves recurrence-free survival, while another Phase 2 study tests whether restarting Imatinib with atezolizumab helps people with unresectable advanced disease after standard treatment failure.^[14][10]

There is also a completed Phase 4 inhaled Imatinib program in pulmonary arterial hypertension, which first studied dose finding and then long-term safety and longer-term effects on exercise capacity and heart-related markers.^[11][15]

Patient-friendly terms

Randomized means people are assigned by chance to one of the study groups, so the groups can be compared fairly.^[5]

Placebo means a look-alike treatment that does not contain the active study drug. It helps researchers see whether the real treatment works better than no active treatment.^[5]

Response means the disease gets smaller, less active, or easier to control after treatment.^[1][8]

Survival outcomes measure how long people live or how long they stay free from a major event like relapse, recurrence, or worsening disease.^[6][14]