Ianalumab + TPO-RA for ITP

(VAY2EXPLORE Trial)

This trial tests a new combination treatment for individuals with primary immune thrombocytopenia (ITP) or primary Evans syndrome (ES), conditions where the immune system attacks blood cells. The study aims to evaluate the effectiveness of ianalumab when combined with another drug that boosts platelet levels. Eligible participants include those who have tried one to four treatments for ITP without lasting success and are currently on a stable dose of a specific platelet-boosting drug. Participants will receive ianalumab with a thrombopoietin receptor agonist (TPO-RA), a drug that stimulates platelet production, chosen by their doctor. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

The trial requires that participants stay on a stable dose of their current thrombopoietin receptor agonist (TPO-RA) for at least 14 days before starting ianalumab. Other medications are not specifically mentioned, so it's best to discuss with the trial team.

Research has shown that ianalumab is generally safe for people with primary immune thrombocytopenia (ITP). Earlier studies reported good safety results for ianalumab, with most side effects being manageable and related to the treatment's mechanism. Common side effects included infections and infusion-related reactions, but these were usually not severe.

For thrombopoietin receptor agonists (TPO-RAs), studies have demonstrated their general safety. Most patients experienced a good increase in platelet levels, and serious side effects were rare. Although a few cases of blood clots occurred, they were uncommon. TPO-RAs have been used for many years, and their safety is well-established.

Researchers are excited about ianalumab combined with a thrombopoietin receptor agonist (TPO-RA) for treating primary immune thrombocytopenia (ITP) because it targets the condition uniquely. Unlike standard treatments that focus primarily on platelet production, ianalumab works by targeting and modulating the immune system, potentially offering a more comprehensive approach. This dual-action strategy, combining immune modulation with enhanced platelet production, could provide a more effective treatment for patients who haven't responded well to existing therapies. Additionally, using an investigator's choice of TPO-RA allows for personalized treatment, which could further improve patient outcomes.

Research has shown that combining ianalumab with a thrombopoietin receptor agonist (TPO-RA) may effectively treat immune thrombocytopenia (ITP). In earlier studies, nearly half of the patients responded positively, with more than half maintaining stable platelet levels. This combination significantly delayed the need for additional treatment, with patients experiencing an average of 13 months without treatment failure. Additionally, it improved disease control by 45%, allowing patients to manage their condition 2.8 times longer than those not using this treatment. In this trial, all participants in both the main cohort for ITP and the exploratory cohort for Evans syndrome (ES) will receive ianalumab plus an investigator's choice of TPO-RA. For Evans syndrome, TPO-RAs have proven very effective, but they require careful monitoring to avoid potential risks.⁶⁷⁸⁹¹⁰