scholarly journals Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic

2021 ◽  
Vol 12 ◽  
Author(s):  
Meredith K. James ◽  
Kristy Rose ◽  
Lindsay N. Alfano ◽  
Natalie F. Reash ◽  
Michelle Eagle ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Physical Therapists ◽  
Rapid Development ◽  
Clinical Care ◽  
Neuromuscular Disorders ◽  
Mitigation Strategies ◽  
Study Protocols ◽  
Travel Restrictions ◽  
Clinical Outcome Assessments ◽  
Investigational Agents

Clinical outcome assessments of function or strength, assessed by physical therapists, are commonly used as primary endpoints in clinical trials, natural history studies and within clinics for individuals with neuromuscular disorders. These evaluations not only inform the efficacy of investigational agents in clinical trials, but also importantly track disease trajectory to prospectively advise need for equipment, home and work modifications, and other assistive devices. The COVID-19 pandemic had a global impact on the safety and feasibility of in-person visits and assessments, necessitating rapid development of mitigation strategies to ensure ongoing collection of key clinical trial endpoints and access to expert clinical care despite travel restrictions. Physical therapists who are expert in neuromuscular disorders working across clinics, countries, and clinical trials developed initial guidelines and methods for the suitability and feasibility of performing remote evaluations. A number of Sponsors introduced amendments to their study protocols to enable remote evaluations, supported by live video streaming of the assessment to their local clinical evaluators. Similarly, application of these techniques to clinical telemedicine enabled objective evaluations for use in payer discussions, equipment procurement, and general access to expert physical therapy services. Here we report on our methodology for adapting current practices to remote testing and considerations for remote evaluations.

Interest, attitudes, and participation in clinical trials among lymphoma patients with online access

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e19514-e19514
Author(s):  
K. Schwartz ◽  
Keyword(s):  
Clinical Trials ◽  
Study Design ◽  
Treatment Decision ◽  
Participation Rates ◽  
Regular Treatment ◽  
Study Enrollment ◽  
Study Protocols ◽  
Survey Results ◽  
Online Access ◽  
Investigational Agents

e19514 Background: There is an increasing number of investigational agents for lymphomas and a limited patient pool, approximately 5% of available patients. Thus, identifying and addressing obstacles to study enrollment is vital to making progress. Methods: This analysis was made from a non-random population of 251 lymphoma patients with online access to our survey. Results: In this cohort, 43% reported High Interest in clinical trials, 50% have Considered Trials, and 27% Have Participated. Patients who considered studies (126) ranked the following reasons for declining as significant: Randomization (70), Insurance (49), Study Risks / Toxicities (42), Travel and Lodging (30), Eligibility (28), Tests and Procedures (12), and a perception that Regular Treatment is superior (6). See Table 1 for associations with consideration of clinical trials and participation. Conclusions: In this cohort, interest in clinical trials and participation rates were much higher than generally cited. As expected, the discussion of clinical trials with the patient's oncologist was associated with the highest consideration (85%) and participation (53%) rates, suggesting a need to increase awareness of study protocols among treating physicians so that this discussion can become more routine. Patient issues and perceptions regarding randomization, study risk, eligibility, and tests and procedures suggest an opportunity to improve enrollment in clinical trials by focusing on these aspects of study design, specifically, attending to the rationale of the protocol as a treatment decision. [Table: see text] No significant financial relationships to disclose.

Strategies for managing paediatric patients in immunoglobulin clinical trials

2019 ◽  
Vol 6 (3) ◽  
pp. 113-116
Author(s):  
Brenda Reid
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Study Protocol ◽  
Clinical Care ◽  
Paediatric Patients ◽  
Study Protocols ◽  
Federal Food ◽  
Immunoglobulin Treatment ◽  
Health Canada ◽  
Considerable Strain

The continued demand for immunoglobulin treatment for multiple indications has placed considerable strain on the supply of immunoglobulin product. Reliance on a few manufacturers can significantly impact the availability of product. In addition, patient tolerability may vary from one product to another necessitating a choice of products to find the best treatment for an individual patient. Therefore, it is important to conduct clinical trials with new immunoglobulin products to ensure that there is adequate supply and choice of products available on the market. This is particularly important for immunodeficient patients who require treatment with immunoglobulins for life. A requirement for licensing by the Federal Food and Drug Administration and Health Canada is that every immunoglobulin licensing study includes some paediatric patients. Enrolling paediatric subjects in immunoglobulin clinical trials can be challenging due to the need for both consent and assent for enrolment, as well as the increased demands that the study protocol places on the child and family over their usual clinical care. Therefore, it is necessary to utilize strategies that make the demands of the protocol more tolerable for children, and to ensure that the study documentation reflects the unique needs of paediatric patients (Denhoff et al. 2015). Statement of novelty: This paper discusses strategies to facilitate enrolment and adherence to immunoglobulin study protocols that are unique to paediatric patients.

scholarly journals Digital Biomarkers for Neuromuscular Disorders: A Systematic Scoping Review

Diagnostics ◽  
2021 ◽  
Vol 11 (7) ◽  
pp. 1275
Author(s):  
Bo-Young Youn ◽  
Youme Ko ◽  
Seunghwan Moon ◽  
Jinhee Lee ◽  
Seung-Gyu Ko ◽  
...  
Keyword(s):  
Animal Study ◽  
Scoping Review ◽  
Observational Studies ◽  
Muscular Atrophy ◽  
Neurological Diseases ◽  
Rapid Development ◽  
Clinical Care ◽  
Neuromuscular Disorders ◽  
Mice Model ◽  
Digital Biomarkers

Biomarkers play a vital role in clinical care. They enable early diagnosis and treatment by identifying a patient’s condition and disease course and act as an outcome measure that accurately evaluates the efficacy of a new treatment or drug. Due to the rapid development of digital technologies, digital biomarkers are expected to grow tremendously. In the era of change, this scoping review was conducted to see which digital biomarkers are progressing in neuromuscular disorders, a diverse and broad-range disease group among the neurological diseases, to discover available evidence for their feasibility and reliability. Thus, a total of 10 studies were examined: 9 observational studies and 1 animal study. Of the observational studies, studies were conducted with amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD), and spinal muscular atrophy (SMA) patients. Non-peer reviewed poster presentations were not considered, as the articles may lead to erroneous results. The only animal study included in the present review investigated the mice model of ALS for detecting rest disturbances using a non-invasive digital biomarker.

Current RNA-based Therapeutics in Clinical Trials

2019 ◽  
Vol 19 (3) ◽  
pp. 172-196 ◽  
Author(s):  
Ling-Yan Zhou ◽  
Zhou Qin ◽  
Yang-Hui Zhu ◽  
Zhi-Yao He ◽  
Ting Xu
Keyword(s):  
Clinical Trials ◽  
Rapid Development ◽  
Genetic Diseases ◽  
Three Dimensional ◽  
Therapeutic Effects ◽  
Messenger Rnas ◽  
Small Interfering Rnas ◽  
Rna Modifications ◽  
Guide Rna ◽  
Endogenous Genes

Long-term research on various types of RNAs has led to further understanding of diverse mechanisms, which eventually resulted in the rapid development of RNA-based therapeutics as powerful tools in clinical disease treatment. Some of the developing RNA drugs obey the antisense mechanisms including antisense oligonucleotides, small interfering RNAs, microRNAs, small activating RNAs, and ribozymes. These types of RNAs could be utilized to inhibit/activate gene expression or change splicing to provide functional proteins. In the meantime, some others based on different mechanisms like modified messenger RNAs could replace the dysfunctional endogenous genes to manage some genetic diseases, and aptamers with special three-dimensional structures could bind to specific targets in a high-affinity manner. In addition, the recent most popular CRISPR-Cas technology, consisting of a crucial single guide RNA, could edit DNA directly to generate therapeutic effects. The desired results from recent clinical trials indicated the great potential of RNA-based drugs in the treatment of various diseases, but further studies on improving delivery materials and RNA modifications are required for the novel RNA-based drugs to translate to the clinic. This review focused on the advances and clinical studies of current RNA-based therapeutics, analyzed their challenges and prospects.

scholarly journals Interventions to Ameliorate the Psychosocial Effects of the COVID-19 Pandemic on Children—A Systematic Review

2021 ◽  
Vol 18 (5) ◽  
pp. 2361
Author(s):  
Katharina Boldt ◽  
Michaela Coenen ◽  
Ani Movsisyan ◽  
Stephan Voss ◽  
Eva Rehfuess ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trials ◽  
Health Management ◽  
Psychosocial Interventions ◽  
Well Being ◽  
World Health ◽  
Research Database ◽  
Psychosocial Effects ◽  
Global Database ◽  
Study Protocols

The aim of this study was to identify interventions targeting children and their caregivers to reduce psychosocial problems in the course of the COVID-19 pandemic and comparable outbreaks. The review was performed using systematic literature searches in MEDLINE, Embase, PsycINFO and COVID-19-specific databases, including the CDC COVID-19 Research Database, the World Health Organisation (WHO) Global Database on COVID-19 Research and the Cochrane COVID-19 Study Register, ClinicalTrials.gov, the EU Clinical Trials Register and the German Clinical Trials Register (DRKS) up to 25th September 2020. The search yielded 6657 unique citations. After title/abstract and full text screening, 11 study protocols reporting on trials planned in China, the US, Canada, the UK, and Hungary during the COVID-19 pandemic were included. Four interventions targeted children ≥10 years directly, seven system-based interventions targeted the parents and caregivers of younger children and adolescents. Outcome measures encompassed mainly anxiety and depressive symptoms, different dimensions of stress or psychosocial well-being, and quality of supportive relationships. In conclusion, this systematic review revealed a paucity of studies on psychosocial interventions for children during the COVID-19 pandemic. Further research should be encouraged in light of the expected demand for child mental health management.

scholarly journals International teleproctoring in neurointerventional surgery and its potential impact on clinical trials in the era of COVID-19: legal and technical considerations

2020 ◽  
pp. neurintsurg-2020-017053
Author(s):  
Emanuele Orru' ◽  
Miklos Marosfoi ◽  
Neil V Patel ◽  
Alexander L Coon ◽  
Christoph Wald ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
High Resolution ◽  
Situational Awareness ◽  
Flow Diverter ◽  
Multiple Sources ◽  
Remote Supervision ◽  
Angiography Suite ◽  
The Us ◽  
Travel Restrictions

BackgroundExisting travel restrictions limit the mobility of proctors, significantly delaying clinical trials and the introduction of new neurointerventional devices. We aim to describe in detail technical and legal considerations regarding international teleproctoring, a tool that could waive the need for in-person supervision during procedures.MethodsInternational teleproctoring was chosen to provide remote supervision during the first three intracranial aneurysm treatments with a new flow diverter (currently subject of a clinical trial) in the US. Real-time, high-resolution transmission software streamed audiovisual data to a proctor located in Canada. The software allowed the transmission of images in a de-identified, HIPAA-compliant manner.ResultsAll three flow diverters were implanted as desired by operator and proctor and without complication. The proctor could swap between images from multiple sources and reported complete spatial and situational awareness, without any significant lag or delay in communication. Procedural times and radiologic dose were similar to those of uncomplicated, routine flow diversion cases at our institution.ConclusionsInternational teleproctoring was successfully implemented in our clinical practice. Its first use provided important insights for establishing this tool in our field. With no clear horizon for lifting the current travel restrictions, teleproctoring has the potential to remove the need for proctor presence in the angiography suite, thereby allowing the field to advance through the continuation of trials and the introduction of new devices in clinical practice. In order for this tool to be used safely and effectively, highly reliable connection and high-resolution equipment is necessary, and multiple legal nuances have to be considered.

scholarly journals COVD-07. THE IMPACT OF COVID-19 ON PATIENTS AND CAREGIVERS AFFECTED BY BRAIN TUMORS: THE PATIENT NAVIGATOR PERSPECTIVE

2020 ◽  
Vol 22 (Supplement_2) ◽  
pp. ii22-ii22
Author(s):  
Kyle Walsh
Keyword(s):  
Clinical Trials ◽  
Brain Tumor ◽  
Clinical Care ◽  
Structured Interview ◽  
Patient Navigator ◽  
Structured Interviews ◽  
Social Distancing ◽  
Tumor Patient ◽  
Patient Navigators ◽  
The Impact

Abstract BACKGROUND Preliminary evidence indicates that glioma patients are at higher risk for COVID-19 complications due to systemic immunosuppression. Interruptions in cancer care may exacerbate patient and caregiver anxiety, but surveying patients/caregivers about their COVID-19 experiences is often limited by attainable sample sizes and over-reliance upon single-institution experiences. METHODS To explore how COVID-19 is impacting brain tumor patients/caregivers across the U.S., we performed semi-structured interviews with brain tumor patient navigators employed by two different 501(c)3 nonprofit organizations. A semi-structured interview guide was used, utilizing prompts and open-ended questions to facilitate dialogue. A core set of COVID-19 topics were covered, including: financial issues, coping strategies, geographic variability, variability by tumor grade/histology, disruptions in care continuity, accessing clinical trials, psychosocial issues, and end-of-life care. Interviews were audio-recorded, transcribed, and organized by discussion topic to identify emerging themes. Inductive sub-coding was completed using the constant comparison method, within and between transcripts. RESULTS/CONCLUSIONS Ten patient navigators were interviewed between April 15th and May 8th, with interviews lasting approximately one hour (range 38-77minutes). Navigators reported having contact with 183 unique brain tumor families during the pandemic (range 7–38 families per navigator). High concordance emerged across narratives, revealing important considerations for the neuro-oncology workforce. The most prominent theme was increased caregiver burden, attributed to maintaining social distancing by reducing visits from home-health aides and friends/family. A related theme that applied to both patients and caregivers was increased social isolation due to social distancing, suspension of in-person support groups, and church/temple closures. Accessing clinical trials was a recurrent issue, exacerbated by patients’ increasing unwillingness to travel. Glioblastoma patients, especially those with recurrent tumors, expressed greater reluctance to travel. Access to standard-of-care treatment was rarely interrupted, but reduced access to supportive services – especially physical and occupational therapy – was identified as an emerging COVID-related deficiency in clinical care.

Efficacy and Safety of AZD1222, BNT162b2 and mRNA-1273 vaccines against SARS-CoV-2

2021 ◽  
Vol 5 (1) ◽  
pp. 791-796
Author(s):  
Ilir Alimehmeti
Keyword(s):  
Clinical Trials ◽  
Diagnostic Tests ◽  
Web Of Science ◽  
Rapid Development ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Vaccine Candidates ◽  
Genetic Sequence ◽  
Phase Iii Clinical Trials ◽  
Clinical Trial Results

SARS-CoV-2, the beta coronavirus causing COVID-19, was isolated and categorizes as a novel one on January 7th, 2020 in China.[1] To date, official reports depict that SARS-CoV-2 has already infected 88.828.387 persons and caused 1.926.625 deaths worldwide.[2] On January 12th, 2020, China officials made public its genetic sequence, thus paving the way towards the research and development of diagnostic tests and vaccines. With regard to vaccination, e large number of clinical trials were designed and are currently undergoing, of which 189 are listed in ClinicalTrials.gov. [3] However, up to date, only three vaccines have published their respective phase III clinical trial results in peer-reviewed medical journals. [4-6] Vaccines are needed to reduce the morbidity and mortality associated with Covid-19, and multiple vaccine platforms as AZD1222 (AstraZeneca) [4], BNT162b2 (Pfizer/BioNTech) [5] and mRNA-1273 (Moderna) have been involved in the rapid development of vaccine candidates. Methodology: In this review, PubMed, Embase, Web of Science, Scopus, medRxiv, and bioRxiv were systematically scrutinized for peer-reviewed and preprint articles on phase III clinical trials of vaccines against SARS-CoV-2. In total, only three peer-reviewed papers fulfilling the search criteria were identified. Conclusions; All vaccine candidates should publish in peer-reviewed journals their efficacy and safety well before requesting approval to the national or international authorities…

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