Abstract / Summary
Abstract Background Thalassemia is a prevalent inherited hematologic disorder imposing substantial health burdens, with unmet clinical needs for effective anemia-relieving therapies. Despite the rapid advancement of targeted drug development, systematic characterization of the global clinical trial landscape remains insufficient. Objective To examine the characteristics and publication status of clinical trials involving anemia-relieving medications for thalassemia registered on Clinicaltrials.gov. Methods Data of interventional clinical trials of anemia-relieving medications for thalassemia, registered on Clinicaltrials.gov, were collected. Key aspects such as the general characteristics, study design features, treatment strategies, outcome measures, and publication status were analyzed. Results Fifty-three clinical trials were included. Of these, 45.28% were completed. However, only 24.53% reported results. Most trials targeted adults/older adults, with 50.94% of the studies conducted in Asia. Among the trials analyzed, 47.17% were Phase 2 studies, and 37.74% employed randomization. Additionally, 47.17% used a parallel assignment intervention model, while 75.47% had no blinding. The most commonly studied drug was fetal hemoglobin inducers. Recently, research into novel therapeutic agents has risen considerably. While primary outcome measures varied across studies, hemoglobin level changes were the most commonly utilized measure. Conclusions Most interventional trials on anemia-relieving medications for thalassemia were non-randomized, non-blinded, and employed parallel assignment, primarily involving adult and older adult patients. While a significant portion of the research focused on fetal hemoglobin inducers, there has been a rising interest in erythroid maturation agents and pyruvate kinase-R agonists.