ObjectiveTo clarify the impact of Japan’s Clinical Trials Act (CTA), which was enacted in April 2018, on subsequent clinical trial activity through an analysis of Japanese registry data.DesignRetrospective database study.SettingWe extracted information on clinical intervention studies registered between 1 April 2018 and 30 September 2020 in the conventional University Hospital Medical Information Network Clinical Trials Registry (UMIN-CTR) and the new Japan Registry of Clinical Trials (jRCT). We collected and analysed information on registration dates, intervention types, funding, secondary sponsors and use of designated staff in multidisciplinary roles (research planning support, research administration, data management, statistical analysis, monitoring and auditing). The temporal trends in clinical trial activity after CTA enactment were examined.ResultsA total of 577 CTA-compliant specified clinical trials (ie, studies funded by pharmaceutical companies or studies evaluating the efficacy and safety of off-label drugs or devices in humans) were registered in the jRCT. During the same period, 5068 clinical trials were registered in the UMIN-CTR. The number of specific clinical trials increased immediately after the implementation of the CTA and stabilised in late 2019, whereas the number of clinical trials registered in the UMIN-CTR generally declined over time. Specified clinical trials that received industry funding and public grants were more likely to use designated staff in multidisciplinary roles.ConclusionsThe implementation of the CTA has not reduced the number of specified clinical trials, but has reduced the total number of intervention trials. The use of designated staff in multidisciplinary roles is associated with funding, secondary sponsors and multicentre studies. It was inferred that funding was needed to establish research infrastructure systems that support high-quality research.
Langerhans cell histiocytosis (LCH) is the most frequent type of histiocytosis and is characterized by both “inflammation/immune dysregulation” and “neoplastic disorder.” Although overall survival has remarkably improved through consecutive previous studies, the relapse-free survival rate is still only 60% to 70%, even in the latest clinical trials. Relapse of LCH is the most significant risk factor for permanent consequences in the central nervous system, such as diabetes insipidus, anterior pituitary hormone deficiency, and neurodegenerative diseases. In this trial, we evaluated the safety and effectiveness of hydroxyurea and methotrexate in recurrent LCH. Treatments in this study consisted of only orally administered drugs and not intravascularly administered drugs. Hydroxyurea therapy for patients with LCH is expected to be safer, less painful, and more cost-effective than other treatments for LCH. The results of this study could provide new therapeutic alternatives for recurrent LCH.
Methods and analysis:
This study was a non-blinded, multicenter, single-arm study. Recurrent (relapsed) LCH is defined as the appearance of new lesions or the enlargement of preexisting lesions due to LCH. In this study, all patients received hydroxyurea, and if the treatment response was unsatisfactory, methotrexate was added. The duration of treatment was 48 weeks. The primary endpoint was the rate of non-active disease achievement, which was 24 weeks after initiating hydroxyurea administration. No active disease is defined as the resolution of all the signs and symptoms related to LCH.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.