In short
Clinical trials are investigating Imatinib in several patient groups, mainly people with blood cancers and some other serious conditions. These studies look at how well it works, how safe it is, and what results it can achieve in different phases of research.
Key points
- Clinical trials of Imatinib in the data focus mostly on blood cancers, especially chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia. Other studies look at gastrointestinal stromal tumor, pulmonary arterial hypertension, acute ischemic stroke, and primary breast cancer. The trials range from Phase 1/2 to Phase 4, so they include early safety work and later studies that compare outcomes in larger groups. Many studies measure molecular response, survival, recurrence-free survival, or recovery scores. Some trials test Imatinib alone, while others compare it with other treatments or use it as part of combination therapy.
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. Most studies are in cancer, especially leukemia, but some also test Imatinib in pulmonary arterial hypertension, acute ischemic stroke, and rare cancer settings.
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.
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. Several trials also study Philadelphia chromosome-positive acute lymphoblastic leukemia, which is a type of blood cancer with a specific genetic change.
Other conditions include gastrointestinal stromal tumor, pulmonary arterial hypertension, acute ischemic stroke, and primary breast cancer. There is also a rare-disease study that includes severe sickle cell disease with Imatinib used before gene therapy in some participants.
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.
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. The pediatric trial includes infants, children, and young adults, showing that the studied age range can be very broad depending on the protocol.
Trial phases and study design
The data include Phase 2, Phase 3, Phase 4, and one Phase 1/2 study. 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.
Some studies are randomized, meaning participants are assigned by chance to different treatment groups. 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.
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.
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. 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.
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. In the breast cancer study, the main outcome is whether the tumor changes from estrogen receptor-negative to estrogen receptor-positive after Imatinib treatment.
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. This study measures major molecular response at week 48, which is a common marker of how well the leukemia is controlled.
Several Phase 3 leukemia studies compare Imatinib with ponatinib or with other treatment strategies in Ph+ ALL. These studies focus on molecular response, MRD-negative remission, and event-free or disease-free survival, which are important signs of treatment success.
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.
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.
Patient-friendly terms
Randomized means people are assigned by chance to one of the study groups, so the groups can be compared fairly.
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.
Response means the disease gets smaller, less active, or easier to control after treatment.
Survival outcomes measure how long people live or how long they stay free from a major event like relapse, recurrence, or worsening disease.