Rose Eleanor Keller,1 Sneha Dave,1 Katherine Melton1,21Generation Patient, Indianapolis, IN, USA; 2Department of Nursing Professional Practice, Rhode Island Hospital, Providence, RI, USACorrespondence: Katherine Melton, Department of Nursing Professional Practice, Rhode Island Hospital, 593 Eddy Street, Providence, RI, 02903, USA, Email kmelton@brownhealth.orgAbstract: Clinical trial design and results often do not adequately address the needs of young adult patients (ages 18– 35 years), despite the increasing prevalence of chronic conditions among young adults in the United States. A group of medical professionals, pharmaceutical industry representatives, and young adults with chronic conditions convened in a roundtable discussion hosted by Generation Patient to identify current barriers to young adult clinical trial participation and propose strategies to improve the research process for young adults. The discussion explored the added complexities of young adulthood, including developmental and situational instability and the transition from pediatric to adult care, which can make clinical trial participation more difficult and less appealing; the benefits of increased, proactive collaboration between young adult patients and researchers at every stage of the research process; and practical changes to ensure that data reporting reflects potential outcomes or dispositions unique the young adult patient population, such as arriving at a national consensus around the operational definition of "young adult” and disaggregating safety and efficacy data within finer age brackets. All stakeholders highlighted the need to make medical research more inclusive of young adults so they can be well-equipped to make informed medical decisions.Keywords: patient perspective, roundtable, chronic illness, advocacy
Purpose: To evaluate the efficacy and safety of EnXtra (R) (Alpinia galanga extract) alone, caffeine and its combination with caffeine on mental alertness and fatigue in healthy sleep-restricted adults through acute single-dose and sub-acute 28-day supplementation protocols. Patients and Methods: This randomized, double-blind, placebo-controlled study consisted of two stages: an acute crossover design (n=128) and a sub-acute 28-day parallel design (n=127). In both the stages, participants aged 18-40 years with habitual caffeine intake received one of the following interventions: A. galanga extract (300 mg), caffeine (200 mg), A. galanga extract (300 mg) + caffeine (200 mg), or placebo (300 mg). The primary outcome was mental alertness evaluated using the Jin Fan Attention Network Test (ANT). Secondary outcomes included fatigue (Samn-Perelli Fatigue Scale (SPS)), psychomotor performance (Nine-Hole Peg Test), sustained attention (Continuous Performance Test), and safety parameters. Cognitive assessments were performed at 0, 1, 3, and 5 hours post-dose in controlled environments on each scheduled visit (Days 0, 5, 10, 15, 20, and 48). Results: In both acute and sub-acute phases, A. galanga extract showed statistically significant improvements in alertness compared with placebo at selected time points. The combination group demonstrated greater improvements in alertness compared with placebo in several assessments. Improvements in fatigue scores were also observed with A. galanga extract alone and in combination with caffeine, with statistically significant reductions compared with placebo at certain time points. Trends toward improvements in psychomotor performance (NHPT) and sustained attention (CPT) were observed with A. galanga extract, although these changes were not consistently statistically significant. In the sub-acute phase, caffeine showed improvements in alertness and fatigue primarily at 1 hour post-dose, with limited effects at later time points. Conclusion: Alpinia galanga extract, alone or combined with caffeine, significantly improved mental alertness and reduced fatigue without caffeine-associated rebound effects, with both interventions showing good safety profiles in healthy adults.
Andre Williams,1 Cassandre Horne,1 Katherine Freeman2 1Christine E. Lynn College of Nursing, Florida Atlantic University, Boca Raton, FL, USA; 2Charles E. Schmidt College of Medicine, Florida Atlantic University, Boca Raton, FL, USACorrespondence: Andre Williams, Email andrewilliams@health.fau.eduAbstract: Clinical trials constitute a cornerstone of contemporary medicine and healthcare, as they generate the highest level of empirical evidence regarding the safety and efficacy of pharmacological agents and other therapeutic or preventive interventions. Although strategies to address underrepresentation exist, a critical gap in the literature is the lack of comprehensive, operationalized approaches that integrate study design, social determinants of health (SDOH) data, statistical methodologies, reporting practices, and applications of artificial intelligence and machine learning (AI/ML). To address this gap, the Perspective outlines populations that are typically underrepresented, discusses the ramifications of this underrepresentation, and presents a comprehensive set of strategies to enhance representation and promote generalizability in clinical trials. The five components detailed in the following sections are: (1) defining inclusion and exclusion criteria that balance internal and external validity and reflect the broader affected community; (2) collecting and operationalizing SDOH and demographic data to understand factors associated with treatment outcomes; (3) using statistical adjustment techniques to account for diverse representation in results; (4) ensuring comprehensive clinical trial reporting to support transparency and reproducibility; and (5) leveraging AI/ML to optimize study planning and data analysis.Plain Language Summary: Making Medical Research Work For Everyone: Clinical trials test whether new medicines and treatments are safe and effective. However, many trials do not include enough participants from key groups, such as older adults, racial and ethnic minorities, people with lower incomes, rural residents, and women. When these groups are underrepresented, we cannot be sure if the treatment will work the same way for everyone in real-world settings.This Perspective explains why this underrepresentation happens and why it matters. More importantly, it provides a practical guide to fix the problem. The authors propose five strategies: (1) using real-world health data to design fairer eligibility rules; (2) collecting and analyzing information about social and demographic factors that affect health; (3) using statistical methods to adjust for trial population imbalances; (4) following updated guidelines for transparent reporting of the clinical trial plan and results; and (5) using artificial intelligence carefully to reduce bias, not amplify it.These strategies aim to ensure that future clinical trials include the people who need the treatments most. The goal is better, fairer healthcare for all.Keywords: clinical trial methodology, external validity, underrepresented populations, generalizability, artificial intelligence in clinical research
Kiran Dattatray Pandit,1,* Rajesh Eknath Patil,2,* Abhijeet Ashok Morde,3,* Paras Pravin Patni,3,* Sanjay Vishnu Vaze,4,* Poonam Shrikant Gotal5,* 1Department of General Medicine, Gurukrupa Hospital, Thane, Maharashtra, India; 2Department of General Medicine, New Manak Healthcare Hospital, Navi Mumbai, Maharashtra, India; 3Research & Development, OmniActive Health Technologies, Mumbai, Maharashtra, India; 4Department of Clinical Development, Vedic Lifesciences Pvt. Ltd., Mumbai, Maharashtra, India; 5Department of Science and Communication, Vedic Lifesciences Pvt. Ltd., Mumbai, Maharashtra, India*These authors contributed equally to this workCorrespondence: Abhijeet Ashok Morde, Research & Development, OmniActive Health Technologies, Technology Center, First Floor, A-10, Road 1, Wagle Estate, Thane West, Mumbai, Maharashtra, 400604, India, Tel +91 9594096454, Email a.morde@omniactives.comPurpose: To evaluate the efficacy and safety of EnXtra® (Alpinia galanga extract) alone, caffeine and its combination with caffeine on mental alertness and fatigue in healthy sleep-restricted adults through acute single-dose and sub-acute 28-day supplementation protocols.Patients and Methods: This randomized, double-blind, placebo-controlled study consisted of two stages: an acute crossover design (n=128) and a sub-acute 28-day parallel design (n=127). In both the stages, participants aged 18– 40 years with habitual caffeine intake received one of the following interventions: A. galanga extract (300 mg), caffeine (200 mg), A. galanga extract (300 mg) + caffeine (200 mg), or placebo (300 mg). The primary outcome was mental alertness evaluated using the Jin Fan Attention Network Test (ANT). Secondary outcomes included fatigue (Samn-Perelli Fatigue Scale (SPS)), psychomotor performance (Nine-Hole Peg Test), sustained attention (Continuous Performance Test), and safety parameters. Cognitive assessments were performed at 0, 1, 3, and 5 hours post-dose in controlled environments on each scheduled visit (Days 0, 5, 10, 15, 20, and 48).Results: In both acute and sub-acute phases, A. galanga extract showed statistically significant improvements in alertness compared with placebo at selected time points. The combination group demonstrated greater improvements in alertness compared with placebo in several assessments. Improvements in fatigue scores were also observed with A. galanga extract alone and in combination with caffeine, with statistically significant reductions compared with placebo at certain time points. Trends toward improvements in psychomotor performance (NHPT) and sustained attention (CPT) were observed with A. galanga extract, although these changes were not consistently statistically significant. In the sub-acute phase, caffeine showed improvements in alertness and fatigue primarily at 1 hour post-dose, with limited effects at later time points.Conclusion: Alpinia galanga extract, alone or combined with caffeine, significantly improved mental alertness and reduced fatigue without caffeine-associated rebound effects, with both interventions showing good safety profiles in healthy adults.Keywords: Alpinia galanga, EnXtra®, caffeine, mental alertness, caffeine crash, cognitive enhancement, fatigue
Africa bears a disproportionate share of the global disease burden yet remains underrepresented in global clinical research. Fragmented regulatory systems, variable approval timelines, and limited cross-border coordination have historically constrained multinational trials across the continent. Over the past decade, however, regional medicines regulatory harmonization initiatives and the establishment of the African Medicines Agency (AMA) have begun to transform Africa's regulatory environment. This Commentary reviews progress achieved through regional collaboration, assesses the early role of the AMA, and examines persistent structural and capacity-related barriers. We argue that regulatory harmonization represents a pivotal opportunity to reposition Africa as a central hub for global clinical research. Realizing this potential will require sustained political commitment, investment in regulatory capacity and digital infrastructure, and deeper engagement by global sponsors and funders. Nevertheless, substantial implementation gaps remain, particularly regarding uneven state adoption, sustainable financing, and post-market surveillance capacity. Strengthening Africa's regulatory ecosystem is not only a matter of efficiency, but also an ethical and scientific imperative for producing globally relevant and equitable clinical evidence.
Purpose: This study aimed to explore the clinical efficacy and safety of ProbioSEB CSC3 in modulating immune responses. Patients and Methods: A prospective, interventional, randomized, double-blinded, parallel-group, placebo-controlled clinical study was conducted on 88-healthy subjects, divided into probiotic and placebo groups, who received ProbioSEB CSC3 and Maltodextrin, respectively. The primary endpoints were the total Wisconsin Upper Respiratory Symptom Survey (WURSS) score, overall severity of URTI symptoms, immunological parameters, and serum cortisol levels. The secondary endpoints were vital physical, hematological and biochemical parameters, adverse and/or serious adverse events (AEs/SAEs), tolerability, and Quality of Life (QoL). Results: ProbioSEB CSC3 improved the WURSS and overall severity of the URTI symptom scores post-intervention. A significant improvement in the overall distribution of the illness duration (total symptom days/subject) was observed with probiotic intake compared to placebo (p=0.048,Log rank test). Furthermore, ProbioSEB CSC3 significantly improved immunoglobulin (Ig)-G, Ig-A, interferon-gamma (IFN-gamma), interleukin (IL)-10, C-reactive protein (CRP), and cortisol levels (p<0.01). The overall QoL of the subjects in the probiotic group significantly improved (p<0.01). No AEs or SAEs leading to termination of the study were reported. Conclusion: ProbioSEB CSC3 supplementation positively modulated the immune response and demonstrated efficacy against URTI and/or alleviated associated symptoms. The administered dose was well tolerated and found to be safe. Further clinical studies on larger populations, multiple centers, and prolonged intervention periods are necessary to validate the results and prophylactic role of probiotics in immunomodulation.
Purpose: Athletes often use dietary supplements to enhance their exercise performance. This study investigated the additive effects of Phyllanthus amarus (EB-PA) and whey protein isolate (WPI), compared to WPI alone or placebo on skeletal muscle strength in active males. Participants and Methods: A total of 121 healthy male participants, aged 20 to 35 years and engaged in moderate physical activity, were randomly assigned to one of three groups: placebo group (Microcrystalline cellulose [MCC] capsule and maltodextrin sachet), WPI group (WPI [40 g] and MCC capsule], or EB-PA + WPI group (WPI [40 g] and EB-PA capsule [500 mg]). All participants completed resistance training twice weekly for 30 days. The primary outcome was muscle strength, assessed by 1-repetition maximum (1-RM) for the upper and lower body. Secondary outcomes included muscle endurance, muscle flexibility, muscle mass, and grip strength. Results: At the end of study, combining the EB-PA with WPI showed significantly improved in both upper (18.02%) and lower body (17.62%) 1-RM strength compared to WPI [upper (9.24%) and lower body (9.02%)] as well as placebo [upper (4.39%) and lower body (3.39%)]. The EB-PA + WPI group achieved a 22.89% significant increase in muscle endurance compared to 11.47% increase in the WPI group. Additionally, statistically significant enhancements were observed in muscle flexibility and grip strength in the EB-PA + WPI group versus the other groups (p<0.05). Conclusion: Combination of EB-PA and WPI after 30 days significantly enhanced muscle strength, endurance, flexibility, and grip strength in active males. This suggests the EB-PA enhances muscle strengthening effect of protein supplements. EB-PA was safe throughout the study.
Purpose: This study seeks to examine the efficacy of Cape gooseberries (Physalis peruviana) in regulating blood glucose levels, contributing to diabetes management. By exploring this cost-effective treatment option, this study could inform public health policies, empower communities to use local resources for managing chronic diseases, and encourage further studies on indigenous foods, ultimately enhancing the understanding of their potential to prevent diseases and promote health. Patients and Methods: A 12-week randomized controlled trial will be conducted with 200 diabetic patients recruited from St. Francis Nsambya and Mulago hospital diabetes clinics. The intervention group will consume 80 grams of fresh gooseberry per day in addition to regular diet and the control group will only consume their regular diet. Fasting blood glucose (FBG) will be assessed at baseline and bi-weekly, while the glycated haemoglobin (HbA1c) levels will be assessed at baseline, 6 weeks and 12 weeks. Adherence will be assessed through food intake diaries, bi-weekly group meetings, and Short Message Service (SMS) reminders. Statistical analysis will be conducted using SPSS. Descriptive statistics will summarize baseline characteristics for both the intervention and control arms. Independent t-tests will compare differences between the intervention and control arms. A p-value of <0.05 will be considered statistically significant. The primary outcomes are change in the levels of FBG and HbA1c levels. The secondary outcomes are rates of adherence and reported side effects. Discussion: The study is expected to provide evidence that daily consumption of Cape gooseberries improves FBG and HbA1c in patients with T2DM. Positive results could support the integration of indigenous fruit into the dietary recommendations, offering a potentially less expensive strategy for T2DM management and inform future research and public health interventions.
Background: Portal hypertension (PH) is a consequence of liver fibrosis and can lead to decompensated cirrhosis with complications such as ascites and gastroesophageal hemorrhage, etc. Liver fibrosis and hepatic architecture disorder contribute to the formation and development of PH. There is an unmet need for curing drugs to improve PH and preventing the occurrence of complications. It is not well known whether anti-liver fibrotic medicines can reduce the risk of PH and decompensated cirrhosis due to HBV, which could be validated by clinical trials. Methods/Design: This is a non-blind, non-placebo-controlled, randomized, multicenter clinical trial. One hundred and ninety-two patients with HBV-related cirrhosis with or without mild gastroesophageal varices in the compensatory stage were enrolled in protocol A. The control group received entecavir (ETV), while the experimental group received Fuzheng Huayu Tablets (FZHY) and ETV. One hundred and eighty-four CHB patients with moderate or severe gastroesophageal varices in the compensatory stage of HBV cirrhosis were enrolled in protocol B. The control group received ETV plus carvedilol (CDL), while the experimental group received FZHY, ETV, and CDL. Both protocols were carried out with 96 weeks of treatment and 12 weeks of follow-up. The primary outcome was the incidence of liver decompensation events in cirrhotic patients. The secondary outcomes included grades of gastroesophageal varices (GEV), liver stiffness measurement, and liver functions. The safety of the testing medicines was also observed. Discussion: Through a multi-center, controlled clinical trial, with the main endpoints of liver decompensated events, we aim to evaluate the clinical efficacy and safety of anti-fibrotic product FZHY on PH in patients with HBV-caused cirrhosis. Trial Registration: Clinical Trials.gov, ID: NCT 02945956/02945982. Registered on Oct 26, 2016.
Aims of Research: Expanding global trial access ensures sustainability by incorporating local contexts and prioritizing patient engagement. This research explores how Organizational Empowerment (OE) and incentive management enhance patient involvement in clinical trials, with Contract Research Organizations (CROs) playing a key role. We propose a paradigm shift based on Grothe-Hammer's organizational contributorship, where participation replaces formal membership, enabling active decision-making and engagement. Methods: This qualitative research with holistic case study approach explores the role of incentive management in Organizational Empowerment (OE) within Contract Research Organizations (CROs). Through interviews and analysis, it examines the impact of incentive structures on both employees and patients, assessing their effectiveness in aligning organisational goals with patientcentricity principles. Results: A key outcome of the study is the call for a shift in patient roles, proposing that patients transition from non-contributing to contributing members within the organization. The study demonstrates that patient-centered interventions, including logistical and financial support through third-party organizations, can enhance patient retention, engagement, and diversity in clinical trials. These interventions benefit both patients and the organizations by improving trial efficiency and reducing dropout rates. In advancing the field, this research makes a contribution by integrating organizational theory with clinical research management practices, a perspective that has been underexplored. The application of institutional theory to understand regulatory frameworks and their impact on incentive management reveals how external constraints shape organizational behavior. Conclusion: The clinical trial ecosystem relies on collaboration among stakeholders to enhance patient care, requiring a shift toward a patient-centric model for better treatment adherence and outcomes. This study highlights the role of Organizational Empowerment (OE) and incentive management in fostering inclusivity, advocating for contributorship over traditional membership to strengthen stakeholder engagement in clinical research.
Szymon Musik,1,2 Joanna Sasin-Kurowska,2 Mariusz Panczyk1 1Department of Education and Research in Health Sciences, Medical University of Warsaw, Warsaw, Poland; 2Clinical Data & Insights, Biopharmaceutical Clinical Operations, R&D, AstraZeneca, Warsaw, PolandCorrespondence: Szymon Musik, AstraZeneca Poland, Postępu 14A Street, 02-676, Warsaw, Poland, Tel +48 536-931-331, Email szymon.musik@astrazeneca.comAbstract: Effective clinical data management is fundamental to clinical research and regulatory submissions. Modern clinical trials have increasingly adopted web-based electronic data capture (EDC) systems, which enhance data collection efficiency but introduces challenges in data integration and quality. This scoping review explores the transformative role of artificial intelligence and machine learning in evolving CDM into clinical data science. In the review, we followed the PRISMA-ScR guidelines and analyzed the literature from 2008 to 2025 using Scopus, Web of Science, and PubMed databases. A total of 26 papers were included and categorized into those related to clinical data management, natural language processing, and general artificial intelligence/machine learning adoption in clinical data management. The integration shows promise in enhancing data analysis, automating data cleaning, and predicting critical outcomes. The key emerging trends include risk-based quality monitoring, blockchain technology, remote monitoring, and patient-centric approaches involving wearables and mobile applications. The results clearly indicate a substantial increase in data volume in Phase III trials, underscoring the need for advanced technologies natural language processing offers significant potential in interpreting unstructured text data, thereby improving the clinical data management processes. The review concludes on different artificial intelligence/machine learning techniques like natural language processing, predictive analytics, and automation technologies, and their applications in improving data quality and streamlining clinical data workflows.Keywords: clinical data management, artificial intelligence, machine learning, natural language processing, clinical data science, electronic data capture
Purpose: Anthracycline-induced cardiotoxicity (AIC) is a significant complication in cancer treatment, impacting long-term health outcomes. This study aimed to evaluate interventional clinical trials targeting AIC and identify effective strategies. Methods: We reviewed clinical trials on AIC from International Clinical Trial Registration Platform (ICTRP), focusing on intervention strategies. We assessed the publication status and effectiveness of cardioprotective agents, monitoring techniques, and exercise interventions. Results: A total of 100 trials were identified, with 35% published. Most studies were conducted in the United States, China, and Italy, highlighting geographical disparities. Effective interventions included dexrazoxane for primary prevention, ACEI/ARB combination with beta blockers for long-term cardioprotection. Advanced monitoring techniques, including global longitudinal strain (GLS)-guided echocardiography, cardiac magnetic resonance (CMR) and novel biomarkers (cfDNA, microRNA), showed promise in early AIC detection. Exercise interventions demonstrated significant cardiovascular benefits. Conclusion: Cardioprotective agents, early detection methods, and exercise interventions are key to managing AIC. Dexrazoxane and ACEI/ARB combination with beta blockers are promising. Exercise interventions can improve cardiovascular health and reduce AIC risk. Larger trials with long-term follow-up are essential for refining these strategies.
Purpose: It is crucial to have diverse trial populations to assess the effectiveness of treatments in different patient groups. The purpose of this analysis was to investigate the motivations and barriers to clinical trial participation of potential patients and provide possible solutions to removing these barriers. Patients and Methods: Participants across nine countries, with a variety of ethnic and gender identities, sexual orientations, and socioeconomic backgrounds were included. Potential participants were alerted to the survey via an awareness campaign which included a link to a landing page providing additional information, and the opportunity to sign consent and complete a survey. Survey questions were written to explore how culture, identity, and background influence participant attitudes toward clinical trials. Input into question format was sought from a cross-functional, international team. Results: A total of 3858 participants "true completers" completed all questions in the survey. Of the "true completers" 72.5% of participants said that they would be willing to participate in a clinical trial, but only 23.9% of participants had done so before. The most common barrier to participation was fear of side effects (42.1%) followed by lack of knowledge of clinical trials (23.1%). Financial barriers were also identified, including "potential travel costs" (27.8%) and "a lack of financial compensation apart from travel costs" (24.4%). Survey respondents from minority groups showed a high willingness to participate, with 69.9% of participants who identified as women, 72.7% of LGBTQ+ participants and 96.1% of Black participants expressing an interest in participating in a clinical trial. Conclusion: This survey suggested that insufficient trial enrollment is due to the presence of barriers, rather than an absence of motivation to participate, and should be used to inform new strategies for increasing the diversity of patient populations in clinical trials and making trial participation more widely accessible.
Nearly 3 years after the emergence of COVID-19, it remains one of the world's problems. COVID-19 vaccination is a priority programme for reducing death and severe symptoms. The primary recombinant novel coronavirus ZF2001 vaccine has gone through Phase III clinical trials and demonstrates efficacy against the highly critical COVID-19 globally and in Indonesia (87.6% and 76.0%, respectively). This study aimed to assess immune persistence after three doses of ZF2001. The study monitored and followed up 400 participants 14 days and 6 months after the third dose and investigated immune persistence 6 months after the phase III vaccination (ZF2001). This study was conducted at recruitment locations in Bandung. Participants were divided into the vaccine and placebo groups and entered into the immunogenicity group. The immune persistence of the vaccine was assessed by measuring geometric mean titres (GMT), neutralising antibodies and seropositivity of IgG anti-S-receptor-binding domain (RBD) after 14 days and 6 months. The seropositive antibody rates were nearly the same between 14 days and 6 months. After 6 months, the GMT, seropositivity of IgG anti-S-RBD and neutralising antibodies in the vaccine group decreased significantly, from 6447.63 to 1514.61 with a P-value of 0.001. A booster was considered important after 6 months.
The entire clinical trial process is a perfectly orchestrated team. It took many years before centers started hiring clinical trial coordinators. The centers that decided to do so noted not only positive aspects from the technical side of the study, but also from the side of the participants - which are the patients. The aim of the publication was to collect literature data showing the work of the coordinator in the centers and its impact on increasing the effectiveness of the study. The comparison additionally included the importance of the coordinator's role on patient recruitment and perceptions. The centers analyzed, showed the impact of the coordinator on their center's research at approximately: 99.1% in China (knowledge of patient rights), 80% in Italy (increasing the quality of the clinical trial conducted) and 70% in South Korea (impact on reducing patient withdrawal of consent). Those teams that worked without the support of a coordinator gained less trust among patients, which affected recruitment and retention of participants. The coordinator's influence on study management resulted in better organization of the study. Conclusions reached can support the development of centers and thus clinical trials around the world. Hiring a coordinator not only has an impact on improving the management of the study, but also on increasing the number of patients included, and thus increasing the therapeutic options in medicine. This paper aims to compile existing literature concerning the responsibilities of clinical trial coordinators and subsequently advocate for the value they bring to enhancing the efficacy and efficiency of clinical trials.
In the era of expanded access to effective antiretroviral therapy (ART), the life expectancy of the estimated 1.2 million people with HIV (PWH) in the United States has significantly increased. There is a timely need to develop and evaluate interventions for older PWH to improve their health and functioning. The primary objective of the present work was to describe the pilot trial methodology that aimed to evaluate the feasibility and acceptability of a transdiagnostic cognitive behavioral therapy (CBT) intervention for HIV and Symptom Management - "CHAMP" designed to promote healthy aging by way of decreasing psychological distress, health risk behaviors, and inflammation among older PWH. Ultimately, these data will be used to refine the intervention and study methods, and inform a future efficacy trial.
Aim: The aim of this study is to assess the willingness, motivation, satisfaction levels, and expectations of participants engaged in clinical trials, while also exploring factors that influence compliance from the participants' standpoint. Furthermore, this research aims to enhance the understanding of all stakeholders involved in clinical trials about the needs of participants and to offer evidence -based and pragmatic recommendations for effectively managing clinical trial participants. Methods: An electronic questionnaire covers various aspects of Traditional Chinese Medicine (TCM) clinical trials. The distribution of the electronic questionnaire was facilitated through WeChat groups, WeChat Moments, and individual interactions. Count data was expressed in terms of frequency (or rate), and subsequent analysis was conducted utilizing the standard chi -squared test. A p value <= 0.05 was considered significant. By drawing a Pareto chart, the priority of factors influencing participants' willingness to participate in clinical trials was systematically assessed. Results: 88 participants answered the questionnaire. A decline in education level corresponded to a decreasing comprehension of participants' grasp of "clinical trial" and "clinical trial participant" (p<0.05). The primary reason for participants' reservations about joining clinical trials stems from their inadequate comprehension of clinical trial details. The key factors contributing to participants' reluctance to engage in clinical trials encompass aspects of trial design, the operational procedures, insufficient understanding of clinical trial particulars, concerns about potential harm to their bodies and opposition from family members. Importantly, these areas of participants' hesitation and unwillingness to partake in clinical trials mirror the factors participants' dissatisfaction contributing to. Conclusion: We found four factors significantly influencing participants' willingness to engage and their overall satisfaction: the participant's profile, trial design, trial institution setup, and the qualifications of the researchers. Derived from these critical factors, this paper presents a series of constructive measures.
Background: Multidomain interventions have been shown to be effective in improving cognition, quality of life, reducing neuropsychiatric symptoms and delaying progression of functional impairment or disability in dementia patients. To investigate the multidomain intervention in other populations and diverse cultural and geographical settings, this pilot study will assess the feasibility of a multidomain intervention for older people with dementia in nursing homes in Vietnam.Methods: Participants will be randomized into two equal groups, to receive either a multidomain intervention (intervention group) or regular health advice (control group). The intervention will include physical, cognitive, and social interventions as well as management of metabolic and vascular risk factors. We will hypothesize that the multidomain intervention will be feasible in Vietnam, and participants who receive the intervention will show improvement in quality of life, behaviors, functional ability, cognitive function, sleep, and in reduction of falls, use of healthcare services, and death rate compared to those in the control group during the 6 months intervention period and after the 6 months extended follow-up.Discussion: This is the first study to evaluate the feasibility of a multidomain intervention program for older people with dementia in nursing homes in Vietnam. The results from the trial will inform clinicians and the public of the possibility of comprehensive treatment beyond simply drug treatments for dementia. This paves the way for further studies to evaluate the long-term effects of multidomain interventions in dementia patients. Furthermore, the research results will provide information on the effectiveness of multidomain interventions which will inform policy development on dementia.Trial Registration: The trial is registered with ClinicalTrials.gov identifier: NCT04948450 on 02/07/2021.
Background: Olfactory anomalies are the most common diseases among post-COVID-19 disorders. Only 15% of patients completed their prescribed treatment plans, even though several different treatment strategies were recommended; this had a detrimental effect on the patients' physical, social, and emotional wellbeing.Purpose: The aim of this study was approving intranasal fast-dissolving insulin films as the treatment of choice for anosmia in comparison to the control group, and the innovative treatment for anosmic post-COVID-19 is assessed in terms of the patients' health-related quality of life (HRQoL).Methods: For therapy and evaluation, a randomized clinical trial with forty adult anosmic post-viral patients was performed. The recruited participants were recruited between October 1 and March 8 of 2021 based on predetermined criteria. A validated smell assessment questionnaire concerning the participants' olfactory, physical, and psychological outcomes was given to them. Recruited patients were randomly subdivided into two groups: intervention and control group. Intervention was treated with insulin intranasal films, while control group took plain films (placebo).Results: The physical, emotional, and social health quality of life were significantly (p-value <0.0001) improved after 4 consecutive weeks of treatment with the intervention group compared to the control group. The data were analyzed statistically with the aid of GraphPad Prism 9.1.0.Conclusion: The lowest HRQoLs, which significantly impact their quality of life, are found in post-COVID-19 anosmic patients treated with insulin films. It is advised to employ this new intervention (insulin films) as the main therapy approach and to gather additional industry data for its development and dissemination. Problems with self-hygiene, eating, sense of danger and emotional satisfaction were significantly enhanced with insulin intervention versus placebo.
For many clinical trials, the issue of post-trial access to research treatments is straightforward. Sponsors offer a range of follow-on studies, compassionate use programs or expanded access programs to allow participants to continue accessing beneficial experimental treatments. But there are times when this is not always the case and participants are required to stop beneficial treatments and return to standard care. Guidance states that post-trial access should be made available for "those participants who still need an intervention identified as beneficial". This broad statement has allowed sponsors to make their own interpretation of when an intervention is still "needed" and when it is "beneficial". As a result, there have been a number of situations where participants of clinical trials have been left afterwards with feelings of abandonment. Participants involved in studies with long-term, invasive treatments can be seen as being particularly vulnerable. Optogenetic technology has the potential to offer hope to people with neurological conditions, especially people who may not respond to current approved treatments. Optogenetics typically involves two components: a gene therapy medicinal product (GTMP) that induces long-term expression of light-reactive proteins within cells, and an active implantable device to stimulate the light-sensitised cells. Neither works without the other, hence for long-term patient benefit, both must remain active and may therefore require maintenance or replacement. With the potential life-long consequences of both components and the difficulty of accessing the brain, there is a need to reconsider post-trial guidelines and whether they are suitable to support early phase optogenetic trial participants. This paper considers the ethical and regulatory requirements in place for post-trial access and care in relation to optogenetic treatments of neurological conditions. We propose that a new perspective with wider responsibilities for sponsors is required when it comes to these types of novel therapies.
